Search Results
within category "Children's Health"
Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
Optimizing Individual Nutrition in Preterm Very Low Birth Weight Infants
In preterm infants fed human milk, milk needs to be fortified to meet nutrient
recommendations. Fortification can be 1) standard, 2) individualized (adjusted based on daily
human milk nutrient analysis and milk volume), or 3) optimized (adjusted based on growth rate
and serum analyses).
The first specific aim will determine whether individualized and optimized nutrition during
hospitalization results in improved growth in the neonatal intensive care unit (NICU) in
extremely low gestational age (GA) neonates (ELGANs, <29 weeks) and in small for GA (SGA,
birth weight <10th percentile for GA) preterm infants compared with optimized nutrition.
The second specific aim will determine whether individualized and optimized nutrition in the
NICU improves neurodevelopmental outcomes (acquisition of development milestones) and reduces
the risk of disproportionate growth (i.e., excess fat) in the NICU and findings suggestive of
metabolic syndrome in the first 3 years of life.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Luc Brion
85893
All
up to 7 Days old
N/A
This study is NOT accepting healthy volunteers
NCT02372136
STU 102014-056
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Inclusion Criteria:
• Preterm infants <29 weeks GA and SGA infants <35 weeks GA born at Parkland Health and
Hospital System
• Maternal plan to breastfeed or to use milk from the donor milk bank
• From birth to 1 week of life
Exclusion Criteria:
• Patients on comfort care only
• Patients with major congenital abnormalities
• Patients who are too unstable for the first 7 days to have an accurate length
measurement
Determining an Optimal Weaning Method of Nasal Continuous Positive Airway Pressure in Preterm Neonates
The purpose of this study is to determine an optimal strategy to wean nasal continuous
positive airway pressure (NCPAP) in preterm babies. The investigators hypothesize that babies
that are taken off NCPAP at lower settings will need fewer total days on NCPAP than those
babies taken off at higher settings.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Venkatakrishna Kakkilaya
125855
All
up to 32 Weeks old
N/A
This study is NOT accepting healthy volunteers
NCT02064712
STU 112013-076
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Inclusion Criteria:
• Neonates ≤32wks GA requiring NCPAP in the delivery room or NICU for increasing
respiratory distress or post-extubation from mechanical ventilation.
Exclusion Criteria:
• Neonates requiring NCPAP for less than 48 hours
• Congenital anomalies
• Need for surgery
• Transfer to a different facility
• Grade 3-4 intraventricular hemorrhage
Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
Forty-five subjects receiving no dietary therapy with a proven G1D diagnosis will be
enrolled. To evaluate the effect of C7 supplementation of a regular diet on a EEG activity in
addition to IQ, language, working memory, processing speed, emotional and behavioral
functioning, ataxia, and other neuropsychological and neurological performance indices in
children and adults genetically diagnosed with G1D receiving a regular diet at enrollment.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Juan Pascual
85158
All
24 Months to 35 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03181399
STU 122016-013
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Inclusion Criteria:
• Diagnosis of glucose transporter type I deficiency (G1D), confirmed by clinical
genotyping at a CLIA-certified laboratory or by PET scan.
• Stable diet on either a modified atkins diet or on no dietary therapy (i.e., no
dietary therapy for 1 month).
• Males and females 24 months to 35 years old, inclusive.
Exclusion Criteria:
• Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
• Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome,
Crohn's disease, or colitis that could increase the subject's risk of developing
diarrhea or stomach pain.
• Subjects with a BMI (body mass index) greater than or equal to 30.
• Subjects currently on dietary therapy (i.e., ketogenic diet, medium chain triglyceride
supplemented diets, Atkins diet, low glycemic index diet).
• Subjects with no evidence of abnormal EEG (spike wave discharges) in the last 12
months.
• Women who are pregnant or breast-feeding may not participate. Women who plan to become
pregnant during the course of the study, or who are unwilling to use birth control to
prevent pregnancy (including abstinence) may not participate. Females age 10 and over
will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects
will be asked to agree to abstinence or another form of birth control for the duration
of the study.
• Allergy/sensitivity to C7.
• Previous use of triheptanoin in the past 1 month. Subjects who participate in Protocol
1 of this study are thus eligible.
• Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain
disorder (such as Alzheimer's disease) that would confound assessment of cognitive
changes, in the opinion of the investigator.
• Active drug or alcohol use or dependence that, in the opinion of the investigator,
would interfere with adherence to study requirements.
• Inability or unwillingness of subject or legal guardian/representative to give written
informed consent, or assent for children age 10-17.
• Addition of a new antiseizure drug in the previous 3 months.
Drug: Triheptanoin
Glucose Transporter Type 1 Deficiency Syndrome, Epilepsy, GLUT1DS1, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Protein Type 1 Deficiency Syndrome
Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including Central Nervous System Tumors
Phase 1 of this study, utilizing a rolling 6 design, will be conducted to determine a maximum
tolerated dose (MTD) and recommended Phase 2 dose (RP2D), and to describe the toxicities of
lenvatinib administered in combination with everolimus once daily to pediatric participants
with recurrent/refractory solid tumors. Phase 2, utilizing Simon's optimal 2-stage design,
will be conducted to estimate the antitumor activity of lenvatinib in combination with
everolimus in pediatric participants with selected recurrent/refractory solid tumors
including Ewing sarcoma/peripheral primitive neuroectodermal tumor (pPNET), rhabdomyosarcoma,
and high grade glioma (HGG) using objective response rate (ORR) at Week 16 as the outcome
measure.
Inclusion Criteria
• ≥2 years and <18 years of age for enrolment in Phase 1 or ≥2 years and ≤21 years of
age for enrolment in Phase 2.
• Recurrent or refractory solid tumors
• Phase 1: All solid tumors (measurable or evaluable disease), including primary
central nervous system (CNS) tumors; exclusion of hepatoblastoma and lymphomas.
Participants with diffuse intrinsic pontine glioma, optic pathway glioma, or
pineal tumors with elevated tumor markers (alpha-fetoprotein [AFP] and beta-human
chorionic gonadotropin [ß-hCG][or human chorionic gonadotropin [hCG])do not
require histological or cytological confirmation of diagnosis
• Phase 2: Ewing sarcoma/peripheral primitive neuroectodermal tumor (pPNET),
Rhabdomyosarcoma, High Grade Glioma (HGG) (all must have measurable disease);
exclusion of Diffuse Intrinsic Pontine Glioma
• Histologically or cytologically confirmed diagnosis
• Measurable disease that meets the following criteria (Phase 2):
1. RECIST 1.1 (for all tumor types except HGG): At least 1 lesion of ≥1.0 cm in the
longest diameter for a non lymph node or ≥1.5 cm in the short-axis diameter for a
lymph node which is serially measurable according to RECIST 1.1 using computed
tomography /magnetic resonance imaging (CT/MRI)
2. Response Assessment in Neuro-Oncology (RANO) for high grade glioma (HGG): At
least one lesion must be measurable as defined as a bi dimensionally contrast
enhancing lesion with clearly defined margins by CT or MRI scan, with a minimal
diameter of 1 cm, and visible on 2 axial slices which are preferably at most 5 mm
apart with 0 mm skip
Lesions that have had external beam radiotherapy (EBRT) or locoregional therapies such as
radiofrequency (RF) ablation must show evidence of progressive disease based on RECIST 1.1
to be deemed a target lesion
• Karnofsky performance score ≥50 for participants>16 year of age and Lansky play score
≥50 for participants ≤16 years of age. Neurologic deficits in participants with CNS
tumors must have been relatively stable for at least 7 days prior to study enrollment.
Participants who are unable to walk because of paralysis, but who are up in a
wheelchair, will be considered ambulatory for the purpose of assessing the performance
score
• Prior Therapy
• Participants must have fully recovered from the acute toxic effects of all prior
anti-cancer therapy
• Cytotoxic chemotherapy or other chemotherapy known to be myelosuppressive: ≥21
days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days
if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (eg, not associated with
reduced platelet or absolute neutrophil counts): ≥7 days after the last dose of
agent
• Monoclonal antibodies: ≥21 days or 3 half-lives (whichever is shorter) of the
antibody must have elapsed after the last dose of a monoclonal antibody
(including checkpoint inhibitors). Toxicity related to prior antibody therapy
must be recovered to Grade ≤1
• Corticosteroids: If used to modify immune adverse events related to prior
therapy, ≥14 days must have elapsed since last dose of corticosteroid.
Participants receiving corticosteroids, who have not been on a stable or
decreasing dose of corticosteroid for at least 7 days prior to enrollment, are
not eligible
• Hematopoietic growth factors: ≥14 days after the last dose of a long-acting
growth factor or 7 days for short-acting growth factor. For agents that have
known adverse events occurring beyond 7 days after administration, this period
must be extended beyond the time during which adverse events are known to occur
• Interleukins, interferons, and cytokines (other than hematopoietic growth
factors): ≥21 days after the completion of interleukins, interferons or cytokines
(other than hematopoietic growth factors)
• Stem cell infusions (with or without total body irradiation): Allogeneic
(non-autologous) bone marrow or stem cell transplant, or any stem cell infusion
including donor leukocytes infusion or boost infusion: ≥84 days after infusion
and no evidence of graft versus host disease; Autologous stem cell infusion
including boost infusion: ≥42 days
• Cellular Therapy: ≥42 days after the completion of any type of cellular therapy
(eg, modified T cells, natural killer cells, dendritic cells, etc)
• Radiotherapy (XRT)/External Beam Irradiation including Protons: ≥14 days after
local XRT; ≥150 days after total body irradiation, craniospinal XRT or if
radiation to ≥50% of the pelvis; ≥42 days if other substantial bone marrow
radiation.
• Radiopharmaceutical therapy: ≥42 days after systemically administered therapy.
• Vascular endothelial growth factor (VEGF)/VEGF receptor (VEGFR)-targeted or
mammalian target of rapamycin (mTOR)-targeted therapies: Must not have received
prior exposure to lenvatinib; May have previously progressed on an mTOR
inhibitor; No more than 2 prior VEGF/VEGFR-targeted therapies (For Phase 2 only);
Must not have received prior VEGF/VEGFR-targeted therapy in combination with an
mTOR inhibitor (For Phase 2 only)
• Adequate bone marrow function for participants with solid tumors without known bone
marrow involvement
• Adequate bone marrow function for participants with known bone marrow metastatic
disease
• Adequate renal function
• Adequate liver function
• Adequate cardiac function
• Adequate neurologic function
• Adequate blood pressure (BP) control with or without antihypertensive medications
• Adequate coagulation
• Adequate pancreatic function
• Adequate metabolic function
• Adequate glycemic control
• Participants must have a minimum body surface area (BSA) of 0.6 m^2 at study entry.
Exclusion Criteria
• Participants who have had or are planning to have the following invasive procedures
• Major surgical procedure, laparoscopic procedure, open biopsy or significant
traumatic injury within 28 days prior to enrolment
• Central line placement or subcutaneous port placement is not considered major
surgery. External central lines must be placed at least 3 days prior to
enrollment and subcutaneous ports must be placed at least 7 days prior to
enrollment
• Fine needle aspirate within 7 days prior to enrolment
• Surgical or other wounds must be adequately healed prior to enrolment
• For purposes of this study, bone marrow aspirate and biopsy are not considered
surgical procedures and therefore are permitted within 14 days prior to start of
protocol therapy
• Participants who have non-healing wound, unhealed or incompletely healed fracture, or
a compound (open) bone fracture at the time of enrolment
• Participants having an active infection requiring systemic therapy.
• Participants with a known history of active hepatitis B (defined as hepatitis B
surface antigen reactive or hepatitis B virus- deoxyribonucleic [DNA] detected) or
known active hepatitis C virus (HCV, defined as HCV- Ribonucleic acid [RNA] detected).
Note: No testing for hepatitis B and hepatitis C is required unless mandated by the
local health authority.
• Known to be human immunodeficiency virus (HIV) positive. Note: HIV testing is required
at screening only when mandated by the local health authority
• Clinical evidence of nephrotic syndrome prior to enrolment
• Gastrointestinal bleeding or active hemoptysis (bright red blood of at least half
teaspoon) within 21 days prior to enrolment
• Thrombotic/ thromboembolic event requiring systemic anticoagulation within 90 days
prior to enrollment
• Evidence of new intracranial hemorrhage of more than punctate size on MRI assessment
obtained within 28 days prior to study enrollment for Participants with HGG
• Diagnosis of lymphoma
• Radiographic evidence of major blood vessel invasion/infiltration.
• Evidence of untreated CNS metastases (exception: participants with primary CNS tumors
and leptomeningeal disease)
• Participants who are currently receiving enzyme-inducing anticonvulsants
• Participants chronically receiving strong cytochrome P450 3A4 (CYP3A4)/P-glycoprotein
(P-gp) inhibitors or inducers within 7 days prior to study enrollment
• Females who are breastfeeding or pregnant. For females of childbearing potential, a
negative screening pregnancy test must be obtained within 72 hours before the first
dose of study drug
Drug: Lenvatinib, Drug: Everolimus
Sarcoma, Recurrent and Refractory Solid Tumors, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Kaposis sarcoma, Small Intestine, Soft Tissue
pediatrics, central nervous system tumors, lenvatinib, E7080, everolimus, Ewing sarcoma/peripheral primitive neuroectodermal tumor, rhabdomyosarcoma, high grade glioma, solid tumors
Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)
This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment
that is directed by genetic testing works in pediatric patients with solid tumors,
non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one
line of standard systemic therapy and/or for which no standard treatment exists that has been
shown to prolong survival. Genetic tests look at the unique genetic material (genes) of
patients' tumor cells. Patients with genetic changes or abnormalities (mutations) may benefit
more from treatment which targets their tumor's particular genetic mutation, and may help
doctors plan better treatment for patients with solid tumors or non-Hodgkin lymphomas.
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients with recurrent or
refractory solid tumors, including non-Hodgkin lymphomas, histiocytoses (e.g.
langerhans cell histiocytosis [LCH], juvenile xanthogranuloma [JXG], histiocytic
sarcoma), and central nervous system (CNS) tumors are eligible; patients must have had
histologic verification of malignancy at original diagnosis or relapse except in
patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with
pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers
including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG); in cases where
patient enrolls prior to histologic confirmation of recurrent disease, patient is
ineligible and should be withdrawn from study if histology fails to confirm
recurrence; please note: Patients with Hodgkin lymphoma and plexiform neurofibroma are
not eligible
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must have an
formalin-fixed paraffin-embedded (FFPE) tumor sample available for MATCH study testing
from a biopsy or surgery that was performed at any point after initial tumor
recurrence/progression, or be planned to have a procedure to obtain such a sample that
is considered to be of potential benefit by the treating clinicians; a tumor sample
from a clinically performed diagnostic (pre-treatment) biopsy will be acceptable for
enrollment onto Pediatric MATCH only for children with high-grade gliomas of the
brainstem (diffuse intrinsic pontine gliomas) or thalamus
• Please note: Samples that have been decalcified using standardly utilized
acid-based decalcification methods are not generally suitable for MATCH study
testing; the nucleic acids will have been degraded in the decalcification process
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Karnofsky >= 50% for patients >
16 years of age and Lansky >= 50 for patients =< 16 years of age); note: neurologic
deficits in patients with CNS tumors must have been stable for at least 7 days prior
to study enrollment; patients who are unable to walk because of paralysis, but who are
up in a wheelchair, will be considered ambulatory for the purpose of assessing the
performance score
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must have
radiographically measurable disease; measurable disease based on imaging obtained less
than or equal to 56 days prior to enrollment; patients with neuroblastoma who do not
have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable
disease are eligible; measurable disease in patients with central nervous system (CNS)
involvement is defined as tumor that is measurable in two perpendicular diameters on
magnetic resonance imaging (MRI) and visible on more than one slice
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or
positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression
post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for
Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: NOTE: patient does not need to meet all
subprotocol criteria at time of enrollment onto the APEC1621SC screening protocol, but
will need to meet all criteria prior to enrollment on any assigned treatment
subprotocol. Patients must be enrolled onto a subprotocol within 8 weeks (56 days) of
treatment assignment
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Karnofsky >= 50% for patients > 16 years
of age and Lansky >= 50 for patients =< 16 years of age); Note: neurologic deficits in
patients with CNS tumors must have been stable for at least 7 days prior to study
enrollment; patients who are unable to walk because of paralysis, but who are up in a
wheelchair, will be considered ambulatory for the purpose of assessing the performance
score
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of treatment with subprotocol
specified therapy, the patients must have radiographically measurable disease;
patients with neuroblastoma who do not have measurable disease but have MIBG+
evaluable are eligible; measurable disease in patients with CNS involvement is defined
as tumor that is measurable in two perpendicular diameters on MRI and visible on more
than one slice
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or
positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression
post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for
RECIST 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of enrollment onto a
subprotocol, the following general criteria for initiation of therapy will be
required:
• Patients must have fully recovered from the acute toxic effects of all prior
anticancer therapy and must meet the following minimum duration from prior
anticancer directed therapy prior to enrollment to the subprotocol; if after the
required timeframe, the numerical eligibility criteria are met, e.g. blood count
criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anticancer agents known to be
myelosuppressive: for agents not listed, the duration of this interval must
be discussed with the study chair and the study-assigned research
coordinator prior to enrollment >= 21 days after the last dose of cytotoxic
or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anticancer agents not known to be myelosuppressive (e.g. not associated with
reduced platelet or absolute neutrophil counts [ANC]): >= 7 days after the
last dose of agent; for agents not listed, the duration of this interval
must be discussed with the study chair and the study-assigned research
coordinator prior to enrollment
• Antibodies: >= 21 days must have elapsed from infusion of last dose of
antibody, and toxicity related to prior antibody therapy must be recovered
to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior
therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a
long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth
factor; for agents that have known adverse events occurring beyond 7 days
after administration, this period must be extended beyond the time during
which adverse events are known to occur; the duration of this interval must
be discussed with the study chair and the study-assigned research
coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth
factors): >= 21 days after the completion of interleukins, interferon or
cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any
stem cell infusion including donor lymphocyte infusion (DLI) or boost
infusion: >= 84 days after infusion and no evidence of graft versus
host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular
therapy (e.g. modified T cells, NK cells, dendritic cells, etc.)
• X-ray therapy (XRT)/External Beam Irradiation including Protons: >= 14 days
after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to
>= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM)
radiation; note: radiation may not be delivered to "measurable disease"
tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42
days after systemically administered radiopharmaceutical therapy
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: For patients with solid tumors without
known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving
platelet transfusions for at least 7 days prior to enrollment)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients with known bone marrow
metastatic disease will be eligible for study provided they meet the blood counts (may
receive transfusions provided they are not known to be refractory to red cell or
platelet transfusions); these patients will not be evaluable for hematologic toxicity
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Creatinine clearance or radioisotope
glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or a serum creatinine based on
age/gender as follows:
• Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
• Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
• Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
• Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
• Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
• Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Bilirubin (sum of conjugated +
unconjugated) =< 1.5 x upper limit of normal (ULN) for age
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Serum glutamate pyruvate transaminase
(SGPT) (alanine transferase [ALT]) =< 135 U/L (for the purpose of this study, the ULN
for SGPT is 45 U/L)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients must be able to swallow intact
capsules/tablets, unless otherwise specified in the subprotocol to which they are
assigned
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Agent specific limitations on prior
therapy will be included with specific treatment subprotocols
Exclusion Criteria:
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Pregnant or breast-feeding women will not
be entered on this study due to risks of fetal and teratogenic adverse events as seen
in animal/human studies, or because there is currently no available information
regarding human fetal or teratogenic toxicities; pregnancy tests must be obtained in
females who are post-menarchal; males or females of reproductive potential may not
participate unless they have agreed to use an effective contraceptive method
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Concomitant medications
• Corticosteroids: at the time of consent and enrollment to regimen specific
subprotocols, patients receiving corticosteroids who have not been on a stable or
decreasing dose of corticosteroid for at least 7 days prior to enrollment to the
subprotocol will not be eligible; if used to modify immune adverse events related
to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients must meet criteria for prior therapy at the time
of consent and enrollment to a subprotocol; other investigational agents may not
be administered to patients while they are receiving study drug as part of a
subprotocol
• Anticancer agents: patients must meet criteria for prior therapy at the time of
consent and enrollment to a subprotocol; other investigational agents may not be
administered to patients while they are receiving study drug as part of a
subprotocol
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine,
tacrolimus or other agents to prevent graft-versus-host disease post bone marrow
transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have an uncontrolled
infection are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have had a prior solid organ
transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Additional agent specific criteria will
be included with specific treatment subprotocols
CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes (CHEC-SC)
This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat
chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and
currently receiving commercially approved Cystic Fibrosis transmembrane conductance regulator
(CFTR) modulator therapies.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Meghana Sathe
68730
All
4 Months and over
N/A
This study is NOT accepting healthy volunteers
NCT03350828
STU 012018-076
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Inclusion Criteria:
• Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative
• Enrolled in the Cystic Fibrosis Foundation Patient Registry (CFFPR)
• Male or female ≥ 4 months of age on day of study visit
• Documentation of a CF diagnosis as evidenced by one or more clinical features
consistent with the CF phenotype and one or more of the following criteria:
• Sweat chloride equal to or greater than 60 milliequivalent (mEq)/liter by
quantitative pilocarpine iontophoresis test (QPIT)
• Two well-characterized mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene
• Current treatment with a prescribed commercially approved CFTR modulator for at least
3 months prior to enrollment
• Able to perform the testing and procedures required for this study, as judged by the
investigator
Exclusion Criteria:
• Presence of a condition or abnormality that, in the opinion of the Investigator, would
compromise the safety of the patient or the quality of the data
• Currently enrolled in an investigational trial (including open-label follow-on studies
and Expedited Access Pathway (EAP)) of an agent expected to have an impact on sweat
chloride (refer to current list provided on study website)
Cycled Phototherapy: A Safer Effective Treatment for Small Premature Infants?
Cycled (intermittent) phototherapy will be compared to continuous (uninterrupted)
phototherapy in the treatment of hyperbilirubinemia (newborn jaundice) in extremely low birth
weight newborns in a pilot randomized controlled trial.
Hypothesis: Cycled phototherapy (PT) will provide the same benefits as continuous
phototherapy in extremely low birth weight (ELBW) infants without the risks that have been
associated with continuous phototherapy.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Myra Wyckoff
19272
All
up to 24 Hours old
N/A
This study is NOT accepting healthy volunteers
NCT01944696
STU-2019-0854
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Inclusion Criteria:
• birth weight 401-1000 grams
• age less than or equal to 24 hours
Exclusion Criteria:
• hemolytic disease
• major anomaly
• overt nonbacterial infection
Other: phototherapy
Hyperbilirubinemia, Premature Newborns, Extremely Low Birth Weight, Other Skin
Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
This phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide,
or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active
surveillance may help doctors to monitor subjects with low risk germ cell tumors after their
tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and
cisplatin, work in different ways to stop the growth of tumor cells, either by killing the
cells, by stopping them from dividing, or by stopping them from spreading.
• There is no age limit for the low risk stratum (stage I ovarian immature teratoma and
stage I non-seminoma or seminoma malignant GCT [all sites])
• Standard risk 1: Patient must be < 11 years of age at enrollment
• Standard risk 2: Patients must be >= 11 and < 25 years of age at enrollment
• Patients enrolling on one of the low risk arms must be newly diagnosed with a stage I
germ cell tumor; for the standard risk arms, patients must be newly diagnosed with
metastatic germ cell tumor (stage II or higher); histologic confirmation of a primary
extracranial germ cell tumor in any of the categories outlined below is required of
all patients at enrollment except for those who were initially diagnosed with stage I
non-seminoma malignant GCT and later recur during observation post surgery off study;
for these patients, if elevated tumor markers rise to > 5 x upper limit of normal
(ULN) on at least 2 measurements taken at least 1 week apart, a diagnostic biopsy is
not required for enrollment
• Low risk stage I immature teratoma (IT); site: ovarian; stage: Children's Oncology
Group (COG) stage I, Federation of Gynecology and Obstetrics (FIGO) stage IA and IB;
grade: 2 or 3; histology: pure immature teratoma (may contain microscopic foci of yolk
sac tumor), mixed immature and mature teratoma, (no pathological evidence of MGCT);
tumor markers: alpha-FP =< 1,000 ng/mL, beta-HCG institutional normal; all ages
• Low risk stage I non-seminoma MGCT; site: ovarian, testicular, or extragonadal; stage:
COG stage I, FIGO stage IA and IB, American Joint Committee on Cancer (AJCC)
testicular stage IA, IB and IS; histology: must contain at least one of the following:
yolk sac tumor, embryonal carcinoma, or choriocarcinoma (pure or mixed); all ages
• Low risk stage I seminoma-MGCT; site: testicular; stage: COG stage I; AJCC testicular
stage IA IB, and IS; histology: must contain at least one of the following: may
contain immature/mature teratoma; may NOT contain yolk sac tumor, embryonal carcinoma,
or choriocarcinoma; all ages
• Standard risk 1 (SR1); site: ovarian, testicular, or extragonadal; stage: COG stage
II-IV, FIGO stage IC, FIGO stage II-IV (International Germ Cell Consensus
Classification [IGCCC] criteria DO NOT apply); histology: must contain at least one of
the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) <
11
• Standard risk 2 (SR2)
• Site: ovarian; stage: COG stage II and III, FIGO stage IC, II and III; histology:
must contain at least one of the following: yolk sac tumor, embryonal carcinoma,
or choriocarcinoma; age (years) >= 11 and < 25
• Site: testicular; stage: COG stage II-IV, AJCC stage II, III, IGCCC good risk;
histology: must contain at least one of the following: yolk sac tumor, embryonal
carcinoma, or choriocarcinoma; tumor markers: must be IGCCC good risk; post op:
alpha-FP < 1,000 ng/mL, beta-HCG < 5,000 IU/mL and lactate dehydrogenase (LDH) <
3.0 x normal; age (years) >= 11 and < 25
• Site: extragonadal; stage: COG stage II; histology: must contain at least one of
the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age
(years) >= 11 and < 25
• Notes:
• IGCCC criteria only apply to SR2 patients with a testicular primary tumor
• Use post-op tumor marker levels to determine IGCCC risk group
• Stage 1 seminoma patients are not eligible for the standard risk arms of the
study
• For the low risk stage I non-seminoma MGCT and the standard risk arms, components
of yolk sac tumor, embryonal carcinoma, or choriocarcinoma can be mixed with
other forms of GCT, such as seminoma or mature or immature teratoma; if yolk sac
tumor is the only malignant component present, then it must be deemed by the
pathologist to be greater than a "microscopic component" of yolk sac tumor
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, 2 or 3; use Karnofsky for patients > 16 years of age and
Lansky for patients =< 16 years of age
• Organ function requirements apply ONLY to patients who will receive chemotherapy (SR1
and SR2 patients)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2
• A serum creatinine based on age/gender as follows: (mg/dL)
• 1 month to < 6 months male: 0.4 female: 0.4
• 6 months to < 1 year male: 0.5 female: 0.5
• 1 to < 2 years male: 0.6 female: 0.6
• 2 to < 6 years male: 0.8 female: 0.8
• 6 to < 10 years male: 1 female: 1
• 10 to < 13 years male: 1.2 female: 1.2
• 13 to < 16 years: male: 1.5 female: 1.4
• >= 16 years male: 1.7 female: 1.4
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or
serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x
upper limit of normal (ULN) for age (for the purpose of this study, the ULN for SGPT
is 45 U/L)
• Peripheral absolute neutrophil count (ANC) >= 1,000/mm^3
• Platelet count >= 100,000/mm^3
• Patients enrolling on the standard risk arms must be medically fit to receive protocol
treatment and with no contraindications to protocol treatment
• Eligibility criteria to participate in the pilot study of the AYA-Hears instrument
(patient reported outcomes [PROs] of ototoxicity) Note: participants in group 1 will
not receive AGCT1531 protocol-directed therapy; all other AYA-HEARS patients must be
enrolled on the AGCT1531 SR2 arm in order to participate
• >= 11 and < 25 years old at enrollment
• Able to fluently speak and read English
• Has received prior cisplatin- or carboplatin-based chemotherapy regimen for malignancy
including diagnoses other than germ cell tumor
• Followed for cancer or survivorship care at one of the following institutions:
• Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
• Dana Farber/Harvard Cancer Center
• Hospital for Sick Children
• Children's Hospital of Eastern Ontario
• Oregon Health and Science University
• Seattle Children's Hospital
• Yale University
Exclusion Criteria:
• Patients with any diagnoses not listed including:
• Stage I testicular cancer patients who have undergone primary RPLND
(retroperitoneal lymph node dissection)
• Pure dysgerminoma
• Pure mature teratoma
• Pure immature teratoma COG stage I, grade I
• Pure immature teratoma COG stage I, grade 2,3 with alpha-fetoprotein (AFP) >=
1000 ng/mL
• Pure immature teratoma COG stage II •IV or FIGO stage IC to IV
• "Poor risk" GCT (age >= 11 years old and COG stage IV ovarian, COG stage III or
IV EG, or IGCCC intermediate or poor risk testicular), or
• Primary central nervous system (CNS) germ cell tumor
• Germ cell tumor with somatic malignant transformation
• Spermatocytic seminoma
• Patients must have had no prior systemic therapy for the current cancer diagnosis
• Patients must have had no prior radiation therapy with the exception of CNS
irradiation of brain metastases; (this exception only applies to SR1 patients; any
patients over age 11 with distant metastases to brain [stage IV disease] would be
considered poor risk and therefore not eligible for this trial)
• Patients with significant, pre-existing co-morbid respiratory disease that
contraindicate the use of bleomycin, are ineligible for the standard risk arms of the
trial
• Female patients who are pregnant since fetal toxicities and teratogenic effects have
been noted for several of the study drugs; a pregnancy test is required for female
patients of childbearing potential; (this criteria applies ONLY to patients who will
receive chemotherapy [SR1 and SR2 patients])
• Lactating females who plan to breastfeed their infants; (this criteria applies ONLY to
patients who will receive chemotherapy [SR1 and SR2 patients])
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation; (this
criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2
patients])
Childhood Extracranial Germ Cell Tumor, Malignant Germ Cell Tumor, Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Stage I Ovarian Choriocarcinoma, Stage II Ovarian Choriocarcinoma, Stage III Ovarian Choriocarcinoma, Stage IV Ovarian Choriocarcinoma, Testicular Mixed Choriocarcinoma and Embryonal Carcinoma, Testicular Mixed Choriocarcinoma and Teratoma, Testicular Mixed Choriocarcinoma and Yolk Sac Tumor, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7, Stage I Testicular Yolk Sac Tumor AJCC v6 and v7, Stage II Testicular Choriocarcinoma AJCC v6 and v7, Stage II Testicular Embryonal Carcinoma AJCC v6 and v7, Stage II Testicular Yolk Sac Tumor AJCC v6 and v7, Stage III Testicular Choriocarcinoma AJCC v6 and v7, Stage III Testicular Embryonal Carcinoma AJCC v6 and v7, Stage III Testicular Yolk Sac Tumor AJCC v6 and v7, Malignant Ovarian Teratoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Teratoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7, Other Female Genital, Other Male Genital, Ovary, Unknown Sites, Stage I Testicular Seminoma AJCC v6 and v7
UT Southwestern; Children’s Health; Parkland Health & Hospital System
A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors (LIBRETTO-121)
This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric
participants with an activating rearranged during transfection (RET) alteration and an
advanced solid or primary CNS tumor.
• Advanced or metastatic solid or primary CNS tumor which has failed standard of care
therapies
• Evidence of an activating RET gene alteration in the tumor and/or blood
• Measurable or non-measurable disease
• Karnofsky (participants 16 years and older) or Lansky (participants younger than 16)
performance score of at least 50
• Participant with primary CNS tumors or cerebral metastases must be neurologically
stable for 7 days prior and must not have required increasing doses of steroids within
the last 7 days
• Adequate hematologic, hepatic and renal function.
• Ability to receive study drug therapy orally or via gastric access
• Willingness of men and women of reproductive potential to observe conventional and
effective birth control
Exclusion Criteria:
• Major surgery within two weeks prior to planned start of LOXO-292
• Clinically significant, uncontrolled cardiac, cardiovascular disease or history of
myocardial infarction within 6 months prior to planned start of LOXO-292
• Active uncontrolled systemic bacterial, viral, fungal or parasitic infection
• Clinically significant active malabsorption syndrome
• Pregnancy or lactation
• Uncontrolled symptomatic hyperthyroidism or hypothyroidism (i.e. the participant
required a modification to current thyroid medication in the 7 days before start of
LOXO-292)
• Uncontrolled symptomatic hypercalcemia or hypocalcemia
• Known hypersensitivity to any of the components of the investigational agent, LOXO-292
or Ora-Sweet® SF and OraPlus®, for participants who will receive LOXO-292 suspension
• Prior treatment with a selective RET inhibitor(s) (including investigational selective
RET inhibitor[s])
The investigators want to study if lower doses of chemotherapy will help babies with SCID to
achieve good immunity with less short and long-term risks of complications after
transplantation. This trial identifies babies with types of immune deficiencies that are most
likely to succeed with this approach and offers them transplant early in life before they get
severe infections or later if their infections are under control. It includes only patients
receiving unrelated or mismatched related donor transplants.
The study will test if patients receiving transplant using either a low dose busulfan or a
medium dose busulfan will have immune recovery of both T and B cells, measured by the ability
to respond to immunizations after transplant. The exact regimen depends on the subtype of
SCID the patient has. Donors used for transplant must be unrelated or half-matched related
(haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance
of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T
cells removed, using a newer, experimental approach of a well-established technology. Once
the stem cell transplant is completed, patients will be followed for 3 years. Approximately
9-18 months after the transplant, vaccinations will be administered, and a blood test
measuring whether your child's body has responded to the vaccine will be collected.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Victor Aquino
10208
All
up to 2 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03619551
STU-2018-0210
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
1. Infants with SCID, either typical or leaky or Omenn syndrome.
1. Typical SCID is defined as either of the following
• Absence or very low number of T cells (CD3+ T cells <300/microliter AND no or
very low T cell function (<10% of lower limit of normal) as measured by response
to phytohemagglutinin OR
• Presence of maternally derived T cells
2. Leaky SCID is defined as the following
• Absence of maternally derived T cells
• AND either one or both of the following (i, ii): i) <50% of lower limit of normal T
cell function as measured by response to PHA OR <30% of lower limit of normal T cell
function as measured by response to CD3 ii) Absent or <10% of lower limit of normal
proliferative responses to candida and tetanus toxoid antigens (must document post
vaccination or exposure for this criterion to apply)
• AND at least two of the following (i through iii): i) CD3 T cells < 1500/microliter
ii) >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are
CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of
age) AND/OR are oligoclonal T iii) Low TRECs and/or the percentage of CD4+/45RA+/CD31+
or CD4+/45RA+/CD62L+ cells is below the lower level of normal.
3. Omenn syndrome • Generalized skin rash
• Maternal lymphocytes tested for and not detected.
• >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are
CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (<2 years of
age)
• Absent or low (up to 30% lower limit of normal (LLN)) T cell proliferation to
antigens (Candida, tetanus) to which the patient has been exposed IF:
Proliferation to antigen was not performed, but at least 4 of the following 8
supportive criteria, at least one of which must be among those marked with an
asterisk (*) below are present, the patient is eligible as Omenn Syndrome.
1. Hepatomegaly
2. Splenomegaly
3. Lymphadenopathy
4. Elevated IgE
5. Elevated absolute eosinophil count
6. *Oligoclonal T cells measured by CDR3 length or flow cytometry (upload
report)
7. *Proliferation to PHA is reduced to < 50% of lower limit of normal (LLN) or
SI < 30
8. *Low TRECs and/or percentage of CD4+/RA+ CD31+ or CD4+/RA+ CD62L+ cells
below the lower level of normal
2. Documented mutation in one of the following SCID-related genes
a. Cytokine receptor defects (IL2RG, JAK3) b. T cell receptor rearrangement defects (RAG1,
RAG2) 3. No available genotypically matched related donor (sibling) 4. Availability of a
suitable donor and graft source
1. Haploidentical related mobilized peripheral blood cells
2. 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor
mobilized peripheral blood cells 5. Age 0 to 2 years at enrollment
Note: to ensure appropriate hepatic metabolism, age at time of busulfan start:
For IL2RG/JAK3: 8 weeks For RAG1/RAG2: 12 weeks
6. Adequate organ function defined as:
1. Cardiac:
Left ventricular ejection fraction (LVEF) at rest ≥ 40% or, shortening fraction (SF) ≥
26% by echocardiogram.
2. Hepatic:
Total bilirubin < 3.0 x the upper limit of normal (ULN) for age (patients who have
been diagnosed with Gilbert's Disease are allowed to exceed this limit) and AST and
ALT < 5.0 x ULN for age.
3. Renal:
GFR estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated
GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine
clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2.
4. Pulmonary No need for supplemental oxygen and O2 saturation > 92% on room air at sea
level (with lower levels allowed at higher elevations per established center standard
of care).
Exclusion Criteria:
1. Presence of any serious life-threatening or opportunistic infection at time of
enrollment and prior to the initiation of the preparative regimen. Serious infections
as defined below that occur after enrollment must be reported immediately to the Study
Coordinating Center, and enrollment will be put on hold until the infection resolves.
Ideally enrolled subjects will not have had any infection. If patients have
experienced infections, these must have resolved by the following definitions:
a. Bacterial i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat
culture(s) from same site must be negative and patient has completed appropriate
course of antibacterial therapy (typically at least 10 days).
ii. Tissue-based clinical infection (e.g. cellulitis): Complete resolution of clinical
signs (e.g. erythema, tenderness, etc.) and patient has completed appropriate course
of antibacterial therapy (typically at least 10 days).
iii. Pneumonia, organism not identified by bronchoalveolar lavage: Complete resolution
of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed
appropriate course of antibacterial therapy (typically at least 10 days). If possible,
radiographic resolution should also be demonstrated.
b. Fungal i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat
culture(s) from same site is negative and patient has completed appropriate course of
antifungal therapy (typically at least 14 days). The patient may be continued on
antifungal prophylaxis following completion of the treatment course.
c. Pneumocystis i. Complete resolution of clinical signs (e.g. tachypnea, oxygen
requirement, etc.) and patient has completed appropriate course of therapy (typically
at least 21 days). If possible, radiographic resolution should also be demonstrated.
The patient may be continued on prophylaxis following completion of the treatment
course.
d. Viral i. Viral PCRs from previously documented sites (blood, nasopharynx, CSF) must
be re-tested and are negative.
ii. If re-sampling a site is not clinically feasible (i.e. BAL fluid): Complete
resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.). If possible,
radiographic resolution should also be demonstrated.
2. Patients with HIV or HTLV I/II infection will be excluded.
Drug: Busulfan, Device: Cell processing for TCRαβ+/CD19+ depletion
Age Patients must be ≥ 1 and ≤30 years of age.
Diagnosis
1. Relapsed or refractory AML with ≥5% blasts (by morphology) in the bone marrow.
• 1st or greater relapse, OR
• Failed to go into remission (i.e. refractory) after first or greater relapse, OR
• Failed to go into remission from original diagnosis after two or more induction
attempts.
2. Relapsed or refractory AML with ≤ 5% blasts (by morphology) and MRD positive disease
(M1/MRD+): Two serial marrows demonstrating stable or rising MRD ≥ 0.1 % (i.e. not
declining). MRD will be determined by multiparameter flow cytometry using
AML-associated phenotype markers, or real-time quantitative PCR for AML-associated
genetic lesions
3. Patients may have CNS 1 or 2 or other sites of extramedullary disease. No cranial
irradiation is allowed during the protocol therapy.
4. Patients with secondary AML are eligible.
5. Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are
excluded.
6. Patients with Down Syndrome will be eligible and will be included as an observation
cohort
Performance Level Karnofsky > 50% for patients > 16 years of age and Lansky > 50% for
patients ≤ 16 years of age.
Prior Therapy Patients must have fully recovered from the acute toxic effects of all prior
chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
A. Myelosuppressive chemotherapy
1. Prior chemotherapy Patients must have fully recovered from the acute toxic effects of
all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
At least 14 days must have elapsed since the completion of the cytotoxic therapy,
except Intrathecal chemotherapy.
2. Cytoreduction with hydroxyurea Hydroxyurea can be initiated and continued for up to 24
hours prior to the start of day 1 nivolumab and azacytidine. It is recommended to use
hydroxyurea in patients with significant leukocytosis (WBC > 50,000/L) to control
blast count before initiation of systemic protocol therapy.
B. Hematopoietic stem cell transplant: Patients who have experienced their relapse after a
HSCT are eligible provided they have no evidence of active GVHD, no past history of grade 3
or greater GVHD, and are at least 100 days post-transplant at the time of enrollment.
Patients should be off immune suppression for at least 2 weeks (excluding physiologic
replacement steroids).
C. Hematopoietic growth factors: It must have been at least 7 days since the completion of
therapy with GCSF or other growth factors at the time of enrollment. It must have been at
least 14 days since the completion of therapy with pegfilgrastim (Neulasta®).
D. Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic
agent. For agents that have known adverse events occurring beyond 7 days after
administration, this period must be extended beyond the time during which adverse events
are known to occur. The duration of this interval must be discussed with the study chair
E. Monoclonal antibodies: At least 3 half-lives of the antibody must have elapsed after the
last dose of monoclonal antibody. (i.e. Gemtuzumab = 36 days)
F. Immunotherapy: At least 42 days after the completion of any type of immunotherapy, e.g.
tumor vaccines or CAR T-cells.
G. XRT: XRT is prohibited during protocol therapy. No washout period is necessary for
radiation given to non-CNS chloromas; ≥ 90 days must have elapsed if prior TBI or
craniospinal XRT.
Renal and hepatic function
Patients must have adequate renal and hepatic functions as indicated by the following
laboratory values:
A. Adequate renal function defined as: Patient must have a calculated creatinine clearance
or radioisotope GFR ≥ 70ml/min/1.73m2 OR a normal serum creatinine based on age/gender in
the chart below:
B. Adequate Liver Function Defined as: Direct bilirubin < 1.5 x upper limit of normal (ULN)
for age or normal, AND alanine transaminase (ALT) < 5 x ULN for age. The hepatic
requirements are waived for patients with known or suspected liver involvement by leukemia.
This must be reviewed by and approved by the study chair or vice chair.
Adequate Cardiac Function Defined as: Shortening fraction of ≥ 27% OR ejection fraction of
≥ 50%.
Reproductive Function A. Female patients of childbearing potential must have a negative
urine or serum pregnancy test confirmed within 24 hours prior to first dose.
B. Female patients with infants must agree not to breastfeed their infants while on this
study.
C. Male and female patients of child-bearing potential must agree to use an effective
method of contraception approved by the investigator during the study and for a minimum of
7 months after study treatment. Women of childbearing potential (WOCBP) receiving nivolumab
will be instructed to adhere to contraception for a period of 5 months after the last dose
of nivolumab. Men receiving nivolumab and who are sexually active with WOCBP will be
instructed to adhere to contraception for a period of 7 months after the last dose of
nivolumab.
Informed Consent Patients and/or their parents or legal guardians must be capable of
understanding the investigational nature, potential risks and benefits of the study. All
patients and/or their parents or legal guardians must sign a written informed consent. Age
appropriate assent will be obtained per institutional guidelines. To allow non-English
speaking patients to participate in this study, bilingual health services will be provided
in the appropriate language when feasible.
Protocol Approval All institutional, FDA, and OHRP requirements for human studies must be
met.
Exclusion Criteria:
Patients will be excluded if they have a known allergy or hypersensitivity to nivolumab or
AZA used in the study.
Patients will be excluded if they have a systemic fungal, bacterial, viral or other
infection that is exhibiting ongoing signs/symptoms related to the infection without
improvement despite appropriate antibiotics or other treatment. The patient needs to be off
pressors and have negative blood cultures for 48 hours.
Patients will be excluded if there is a plan to administer non-protocol chemotherapy,
radiation therapy, or immunotherapy during the study period.
Patients will be excluded if they have significant concurrent disease, illness, psychiatric
disorder or social issue that would compromise patient safety or compliance with the
protocol treatment or procedures, interfere with consent, study participation, follow up,
or interpretation of study results.
Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are
excluded.
Patients with a known history of severe interstitial lung disease or severe pneumonitis or
active pneumonitis that is uncontrolled in the opinion of the treating physician.
Patients who have previously been treated with nivolumab will be excluded.
Patients with a known history of any of the following autoimmune diseases are excluded: (a)
patients with a history of inflammatory bowel disease (including Crohn's disease and
ulcerative colitis) (b) patients with a history of rheumatoid arthritis, systemic
progressive sclerosis [scleroderma], Systemic Lupus Erythematosus, autoimmune vasculitis
[e.g., Wegener's Granulomatosis]).
Patients with organ allografts (such as renal transplant) are excluded.
Patients with known Human Immunodeficiency Virus seropositivity will be excluded.
Known to be positive for hepatitis B by surface antigen expression. Known to have active
hepatitis C infection (positive by polymerase chain reaction or on antiviral therapy for
hepatitis C within the last 6 months).
Pregnant or breastfeeding.
Acute promyelocytic leukemia (APL).
CNS 3 disease.
Patients who have experienced their relapse after a HSCT and are less than 100 days
post-transplant at the time of enrollment, have active GVHD at time of enrollment, have
past history of grade 3 or greater GVHD, Patients on immune suppression (excluding
physiologic replacement steroids).
Larotrectinib in Treating Patients With Previously Untreated TRK Fusion Solid Tumors and TRK Fusion Relapsed Acute Leukemia
This phase II trial studies the side effects and how well larotrectinib works in treating
patients with previously untreated TRK fusion solid tumors and TRK fusion acute leukemia that
has come back. Larotrectinib may stop the growth of cancer cells with TRK fusions by blocking
the TRK enzymes needed for cell growth.
• COHORT A: Patients must have a histologic diagnosis of infantile fibrosarcoma with an
NTRK1, NTRK2, or NTRK3 fusion identified in a Clinical Laboratory Improvement
Act/College of American Pathologists (CLIA/CAP) certified laboratory. Fusions may be
identified by fluorescence in situ hybridization (FISH) or molecular techniques
(reverse transcriptase-polymerase chain reaction [RT-PCR] using primers flanking the
fusion junction or next generation sequencing). For fusions identified by FISH, an
ETV6 rearrangement is sufficient for eligibility in Cohort A. Identification of the
upstream TRK fusion partner is not required.
• COHORT B: Patients must have a histologic diagnosis of any solid tumor other than
infantile fibrosarcoma, including central nervous system (CNS) tumors but excluding
high grade gliomas. An NTRK1, NTRK2, or NTRK3 fusion must be identified in a CLIA/CAP
certified laboratory. Fusions may be identified by FISH or molecular techniques
(RT-PCR using primers flanking the fusion junction or next generation sequencing). For
fusions identified by FISH, there must be an identified rearrangement in NTRK1, NTRK2,
or NTRK3 (e.g., an ETV6 rearrangement is not sufficient for eligibility) unless the
patient has a diagnosis of congenital mesoblastic nephroma in which case an ETV6
rearrangement is sufficient for eligibility. Identification of the upstream TRK fusion
partner is not required.
• COHORT C: Patients must have a histologic diagnosis of relapsed or refractory acute
leukemia with an NTRK1, NTRK2, or NTRK3 fusion identified in a CLIA/CAP certified
laboratory. Fusions may be identified by FISH or molecular techniques (RT-PCR using
primers flanking the fusion junction or next generation sequencing). For fusions
identified by FISH, there must be an identified rearrangement in NTRK1, NTRK2, or
NTRK3 (e.g., an ETV6 rearrangement is not sufficient for eligibility). Identification
of the upstream TRK fusion partner is not required.
• SOLID TUMORS (COHORTS A AND B): Patients must have measurable disease. Patients must
have disease that cannot be completely resected without a predicted functional,
neurologic, or significant cosmetic deficit in the opinion of the investigator.
• LEUKEMIA (COHORT C): Patients must have >= 5% blasts in the bone marrow.
Extramedullary disease is permitted.
• Patients must have a Lansky or Karnofsky performance status score of >= 50,
corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2. Use
Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age.
NOTE: Neurologic deficits in patients with CNS tumors must have been stable for at
least 7 days prior to study enrollment. Patients who are unable to walk because of
paralysis, but who are up in a wheelchair, will be considered ambulatory for the
purpose of assessing the performance score.
• COHORTS A AND B: No prior anti-cancer therapy, including radiotherapy, other than
surgical resection is permitted.
• Patients who experience recurrence after surgery alone and no other anti-cancer
therapy will be eligible.
• If not eligible due to prior anticancer therapy, patients may be eligible for the
larotrectinib arm of Pediatric MATCH (APEC1621A) or treatment with commercial
larotrectinib off study.
• COHORT C: Patients with relapsed leukemia (Cohort C) must have fully recovered from
the acute toxic effects of all prior anti-cancer therapy and must meet the following
minimum duration from prior anti-cancer directed therapy prior to enrollment. If after
the required timeframe, the numerical eligibility criteria are met, e.g. blood count
criteria, the patient is considered to have recovered adequately.
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive.
The duration of this interval must be discussed with the study chair and the
study-assigned research coordinator prior to enrollment.
• A waiting period prior to enrollment is not required for patients receiving
standard cytotoxic maintenance chemotherapy (i.e., corticosteroid,
vincristine, thioguanine [6MP], and/or methotrexate).
• A waiting period is not required for patients receiving a single dose of
intrathecal methotrexate, hydrocortisone, and/or cytarabine within 7 days
prior to enrollment
• >= 14 days must have elapsed after the completion of other cytotoxic
therapy, with the exception of hydroxyurea, for patients not receiving
standard maintenance therapy. Additionally, patients must have fully
recovered from all acute toxic effects of prior therapy.
• Note: Cytoreduction with hydroxyurea must be discontinued >= 24 hours
prior to the start of protocol therapy.
• Anti-cancer agents not known to be myelosuppressive (e.g., not associated with
reduced platelet or absolute neutrophil [ANC] counts): >= 7 days after the last
dose of agent. The duration of this interval must be discussed with the study
chair and the study-assigned research coordinator prior to enrollment.
• Anti-cancer agents that are antibodies: >= 21 days must have elapsed from
infusion of last dose of antibody, and toxicity related to prior antibody therapy
must be recovered to grade =< 1. There is an exception for blinatumomab
infusions, for which patients must have been off for at least 3 days and all drug
related toxicity must have resolved to grade 2 or lower as outlined in the
inclusion/exclusion criteria.
• Corticosteroids: If used to modify immune adverse events related to prior
therapy, >= 14 days must have elapsed since last dose of corticosteroid. A
waiting period prior to enrollment is not required for patients receiving
corticosteroid for leukemia therapy/cytoreduction.
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting
growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For
agents that have known adverse events occurring beyond 7 days after
administration, this period must be extended beyond the time during which adverse
events are known to occur. The duration of this interval must be discussed with
the study chair and the study-assigned research coordinator.
• Interleukins, interferons and cytokines (other than hematopoietic growth
factors): >= 21 days after the completion of interleukins, interferon or
cytokines (other than hematopoietic growth factors )
• Stem cell infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem
cell infusion including donor lymphocyte infusion (DLI) or boost infusion:
>= 84 days after infusion and no evidence of graft versus host disease
(GVHD).
• Autologous stem cell infusion including boost infusion: >= 42 days.
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy
(e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days
after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >=
50% of the pelvis; >= 42 days if other substantial BM radiation.
• Radiopharmaceutical therapy (e.g., radiolabeled antibody): >= 42 days after
systemically administered radiopharmaceutical therapy.
• Patients must not have received prior exposure to TRK inhibitors (including
larotrectinib, LOXO-195, entrectinib, lorlatinib, crizotinib, or lestaurtinib).
• For patients with solid tumors without known bone marrow involvement: Peripheral
absolute neutrophil count (ANC) >= 1000/mm^3 (within 7 days prior to enrollment)
• For patients with solid tumors without known bone marrow involvement: Platelet count
>= 100,000/mm^3 (transfusion independent, defined as not receiving platelet
transfusions for at least 7 days prior to enrollment)
• For patients with solid tumors without known bone marrow involvement: Hemoglobin >=
8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions).
• Patients with solid tumors with known bone marrow metastatic disease will be eligible
for study provided they meet the blood counts above (may receive transfusions provided
they are not known to be refractory to red cell or platelet transfusions). These
patients will not be evaluable for hematologic toxicity.
• For patients with leukemia: Platelet count >= 20,000/mm^3 (within 7 days prior to
enrollment) (may receive platelet transfusions; must not be known to be refractory to
red cell or platelet transfusion)
• For patients with leukemia: Hemoglobin >= 8.0 g/dL at baseline (within 7 days prior to
enrollment) (may receive RBC transfusions; must not be known to be refractory to red
cell or platelet transfusion)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows (within 7 days
prior to enrollment):
• 1 month to < 6 months (male 0.4 mg/dL, female 0.4 mg/dL)
• 6 months to < 1 year (male 0.5 mg/dL, female 0.5 mg/dL)
• 1 to < 2 years (male 0.6 mg/dL, female 0.6 mg/dL)
• 2 to < 6 years (male 0.8 mg/dL, female 0.8 mg/dL)
• 6 to < 10 years (male 1 mg/dL, female 1 mg/dL)
• 10 to < 13 years (male 1.2 mg/dL, female 1.2 mg/dL)
• 13 to < 16 years (male 1.5 mg/dL, female 1.4 mg/dL)
• >= 16 years (male 1.7 mg/dL, female 1.4 mg/dL)
• For patients < 1 month of age, serum creatinine levels must be < 1.5 x the
treating institution's creatinine upper limit of normal (ULN) for patients <
1 month of age or the creatinine clearance or radioisotope GFR must be >= 70
mL/min/1.73 m^2.
• Patients with solid tumors: Bilirubin (sum of conjugated + unconjugated) =< 1.5 x
upper limit of normal (ULN) for age (within 7 days prior to enrollment). After
approval of the study chair or designee, infants with a higher total bilirubin due to
physiologic or breast milk jaundice are eligible if the conjugated (direct) bilirubin
is =< 2 mg/dL
• Patients with solid tumors: Serum glutamate pyruvate transaminase (SGPT) (alanine
aminotransferase [ALT]) =< 135 U/L (within 7 days prior to enrollment). For the
purpose of this study, the ULN for SGPT is 45 U/L
• Patients with solid tumors: Serum albumin >= 2 g/dL (within 7 days prior to
enrollment).
• Patients with leukemias: Conjugated (direct) bilirubin =< 1.5 x upper limit of normal
(ULN) for age (within 7 days prior to enrollment).
• Patients with leukemias: SGPT (ALT) =< 225 U/L (within 7 days prior to enrollment).
For the purpose of this study, the ULN for SGPT is 45 U/L
• Patients with leukemias: Serum albumin >= 2 g/dL (within 7 days prior to enrollment).
• Patients with seizure disorder may be enrolled if on a stable antiepileptic regimen
for >= 14 days and well controlled.
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE]
version [v] 5) except tendon reflex decreased resulting from prior therapy must be =<
grade 2.
Exclusion Criteria:
• Pregnant or breast-feeding women will not be entered on this study due to risks of
fetal and teratogenic adverse events as seen in animal/human studies, OR because there
is yet no available information regarding human fetal or teratogenic toxicities.
Pregnancy tests must be obtained in girls who are post-menarchal. Female patients of
reproductive potential may not participate unless they have agreed to use a highly
effective contraceptive method for the duration of study therapy and for at least one
month after the final dose of larotrectinib. Males of reproductive potential with a
non-pregnant female partner of child-bearing potential must use a highly effective
contraception for the duration of the study and for at least one month after the final
dose of larotrectinib. Because of the unknown risk of larotrectinib in nursing
infants, nursing women should discontinue breastfeeding during treatment with
larotrectinib and for 3 days following the final dose.
• Patients with solid tumors, including CNS tumors, requiring corticosteroids who have
not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to
enrollment are not eligible. Patients with leukemia may receive systemic
corticosteroids for cytoreduction up to 24 hours prior to the start of protocol
therapy. If used to modify immune adverse events related to prior therapy, >= 14 days
must have elapsed since last dose of corticosteroid.
• Patients who are currently receiving another investigational drug are not eligible.
• Patients who are currently receiving other anti-cancer agents are not eligible [except
leukemia patients receiving corticosteroids or hydroxyurea, which may be continued
until 24 hours prior to start of protocol therapy]. Patients with leukemia should
receive a single dose of intrathecal cytarabine, hydrocortisone, and/or methotrexate
within 7 days prior to Day 1 of Cycle 1 at the time of the baseline lumbar puncture.
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent
graft-versus-host disease post bone marrow transplant are not eligible for this trial.
• Patients currently receiving a strong CYP3A4 inducer or inhibitor are not eligible.
Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to
enrollment to the end of the study. Note: CYP3A4 inducing anti-epileptic drugs and
dexamethasone for CNS tumors or metastases, on a stable dose, are allowed.
• Patients with malabsorption syndrome or other conditions that significantly limit
enteral absorption are not eligible.
• Patients who are unable to swallow capsules or liquid and do not have gastric access
via a nasogastric or gastrostomy tube are not eligible.
• Patients who have an uncontrolled infection are not eligible.
• Patients who have received prior solid organ transplantation are not eligible.
• Patients who in the opinion of the investigator may not be able to comply with the
safety monitoring requirements of the study are not eligible.
• Patients with high grade gliomas (HGG) are not eligible.
• All patients and/or their parents or legal guardians must sign a written informed
consent.
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met.
Drug: Larotrectinib Sulfate
Central Nervous System Neoplasm, Infantile Fibrosarcoma, Solid Neoplasm, Recurrent Acute Leukemia, Refractory Acute Leukemia
Exclusive Human Milk Feeding in Infants With Single Ventricle Physiology
A randomized, blinded, controlled trial to evaluate growth velocity and clinical outcomes in
infants with single ventricle physiology fed an exclusive human milk diet prior to, and
throughout the post-operative period following, surgical repair. Human milk is defined as
expressed human milk or donor milk and its derivatives, human milk-based fortifier and human
milk caloric fortifier.
The study hypothesis is that infants fed an exclusive human milk diet will have short and
long term benefits, with improved wound healing, growth, and neurodevelopmental outcomes
while reducing episodes of feeding intolerance and necrotizing enterocolitis (NEC).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Erin Gordon
155770
All
up to 7 Days old
N/A
This study is NOT accepting healthy volunteers
NCT02860702
STU 072016-089
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Inclusion Criteria:
1. Term infants (≥37 and 0/7 weeks gestational age) ≤ 7 days old with a diagnosis of
single ventricle physiology who are thought to require a single ventricle repair at
the time of enrollment.
2. Infant feeding was NPO or consisted of 100% human milk diet prior to enrollment
3. Parent(s) willing to sign informed consent.
4. Parent(s) willing to comply with study follow-up procedures.
5. Require surgical palliation within the first 1 month of life.
Exclusion Criteria:
1. Term infants >7 days old at the time of diagnosis.
2. <37 weeks gestation
3. Infants requiring cardio-pulmonary resuscitation prior to surgical repair.
4. Outborn infants who received enteral nutrition at the other institution prior to
surgical repair. If it is uncertain if infant received even 1 bottle or a small amount
of formula, infants will be excluded.
5. Major congenital abnormalities that could significantly affect survival such as:
1. Confirmed or suspected major genetic abnormalities (lethal or with extremely low
probability for survival).
2. Chromosomal abnormalities: Trisomies (13, 18, 21 etc.) deletions or
translocations (Turner/Williams Syndrome, DiGeorge, to name a few)
3. Major organ system abnormalities not related to a genetic syndrome that are
lethal or have extremely low probability for survival (i.e, bilateral kidney
intrinsic disease, pulmonary hypoplasia, Central Nervous System (CNS)
malformations: Arnold Chiari, myelomeningoceles, hydranencephaly, schizencephaly,
holoprosencephaly))
4. Heterotaxia
5. Metabolic disorders affecting growth: homocystinuria, methylmalonic acidemias,
propionic acidemias, urea cycle defects
6. Evidence of intracerebral hemorrhage (IVH) ≥ Grade 3
7. Any comorbidity or significant clinical event prior to enrollment, deemed by the
Investigator as likely to affect survival.
8. Requires Extracorporeal Membrane Oxygenation (ECMO) pre-operatively
9. Legally Authorized Representative(s) unwilling to comply with an exclusive human milk
diet either in the form of mother's milk, human milk-based human milk fortifier, human
milk based caloric fortifier or donor human milk during the initial hospitalization
period and through the 30 day feeding period after surgical repair or hospital
discharge, whichever comes first.
Other: Human Milk Derived Fortifier, Other: Human/Bovine Milk
The purpose of this study is to determine the optimal range of insertion depths for a
nasopharyngeal probe in anesthetized pediatric patients as a function of age.
Call 214-648-5005 studyfinder@utsouthwestern.edu
John Zhong
69695
All
up to 12 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02963285
STU 072016-059
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Inclusion Criteria:
1. Elective non-cardiac surgery in children to last at least 1.5 hours;
2. Supine position anticipated;
3. General endotracheal anesthesia.
Exclusion Criteria:
1. Nasopharyngeal disease (e.g. sinusitis), upper airway abnormalities, or planned oral
or facial surgery;
2. History of genetic or congenital anomalies leading to facial dimorphism;
3. History of recent substantive epistaxis or suspected bleeding disorder;
4. Therapeutic-dose anti-coagulation;
5. Contraindication to esophageal temperature probe insertion (i.e., esophageal varices,
congenital anomalies).
Network Of Clinical Research Studies On Craniosynostosis, Skull Malformations With Premature Fusion Of Skull Bones
Craniosynostosis (CS) is a common malformation occurring in ~4 per 10,000 live births in
which the sutures between skull bones close too early, causing long-term problems with brain
and skull growth. Infants with CS typically require extensive surgical treatment and may
experience many perioperative complications, including hemorrhage and re-synostosis. Even
with successful surgery, children can experience developmental and learning disabilities or
vision problems. Most often, CS appears as isolated nonsyndromic CS (NSC). Of the several
subtypes of CS, unilateral or bilateral fusion of the coronal suture is the second most
common form of CS accounting for 20-30% of all NSC cases. The etiology of coronal NSC (cNSC)
is not well understood, although the published literature suggests that it is a
multifactorial condition. About 5-14% of coronal craniosynostosis patients have a positive
family history, with a specific genetic etiology identified in >25% of cNSC cases, suggesting
a strong genetic component in the pathogenesis of this birth defect. The causes for cNSC and
its phenotypic heterogeneity remain largely unknown. An international team of investigators
will generate large genomic and gene expression datasets on samples from patients with cNSC.
State-of-the-art imaging, genetic, and developmental and systems biology approaches will be
used to quantitatively model novel pathways and networks involved in the development of cNSC.
Novel variant-, gene- and network-level analyses will be performed on the genomic data
obtained from cNSC cases, their relatives, and controls to identify novel variants and
genetic regions associated with cNCS. Quantitative, analytical, and functional validations of
these predictions will provide insights into the etiology and possible therapeutic targets
for CS and potentially other bone-related disorders.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Alex Kane
120974
All
up to 80 Years old
This study is also accepting healthy volunteers
NCT03025763
STU 022015-001
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Inclusion Criteria:
• Cases with diagnosis of coronal
• Unaffected relatives of cases
• Unaffected controls including those who may have undergone clinically indicated
craniofacial surgery for trauma or conditions other than craniosynostosis or bone
disease. These individuals will be recruited at some of the other collaborating
institutions, but not at Mount Sinai.
Individuals of any racial or ethnic group with the established or suspected clinical
diagnosis of coronal, nonsyndromic craniosynostosis will be included in this study.
Unaffected relatives, such as their biological parents and/or sibs, will also be included
to contribute medical information and samples as negative controls for our study.
Exclusion Criteria:
• Those who fit the criteria, but who choose not to participate
• Those who do not meet the criteria.
• Other than children, no vulnerable individuals will be recruited, such as intellectual
impaired individuals or prisoners.
Does Wearing Tetra-Grip Improve Arm Function in Children Diagnosed With Neonatal Brachial Plexus Palsy?
This study evaluates the effect of wearing a tetra-grip on the affected arm of children with
neonatal brachial plexus palsy. Half of the participants will have tetra-grip applied to the
arm, while the other half will not have it applied to the arm.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Robert Rinaldi
160870
All
3 Years to 7 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03647761
STU 022018-069
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Inclusion Criteria:
• 3 •7 years old
• male or female
• non-dominant upper extremity brachial plexus injury
Exclusion Criteria:
• Botox injections to the affected extremity within the past 3 months
• Severe muscle contractures of affected extremity that restricts functional use of the
arm and hand
• Concurrent cerebral palsy
Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy (SIMPLIFY)
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator
(CFTR) modulator therapies in treating CF, it is still largely unknown whether or not other
chronic therapies can be safely stopped. The SIMPLIFY study is being done to test whether or
not it is safe to stop taking inhaled hypertonic saline or Pulmozyme® (dornase alfa) in those
people that are also taking Trikafta™.
Trikafta (elexacaftor/tezacaftor/ivacaftor) is a combination CFTR modulator therapy that was
approved by the Food and Drug Administration for people with CF who have at least one F508del
mutation. The three drugs that make up Trikafta work together to allow many more chloride
ions to move into and out of the cells, improving the balance of salt and water in the lungs.
These changes result in better clearance of mucus from the lungs and improvements in lung
function.
Inhaled hypertonic saline and Pulmozyme (dornase alfa) also improve clearance of mucus from
the lungs to support lung function and have been available to people with CF for many years.
Both therapies are considered to be relatively burdensome and it is not known whether either
therapy can improve or maintain lung function above what is already gained through Trikafta
use.
The goal of the SIMPLIFY study is to get information about whether or not it is safe to stop
either inhaled hypertonic saline or Pulmozyme (dornase alfa) by testing if there is a change
in lung function in subjects with cystic fibrosis (CF) who are assigned to stop their chronic
medication (either hypertonic saline or Pulmozyme) as compared to those who are assigned to
keep taking their medication while continuing to take Trikafta.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Raksha Jain
19733
All
12 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04378153
STU-2020-0246
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Inclusion Criteria:
• Diagnosis of CF.
• Age ≥ 12 years at the Screening Visit.
• Forced expiratory volume in 1 second (FEV1) ≥ 70 % predicted at the Screening Visit if
< 18 years old, and ≥ 60 % predicted at Screening Visit if ≥ 18 years old.
• Clinically stable with no significant changes in health status within the 7 days prior
to and including the Screening Visit.
• Current treatment with elexacaftor/tezacaftor/ivacaftor (ETI) for at least the 90 days
prior to and including the Screening Visit and willing to continue daily use for the
duration of the study.
• Currently taking hypertonic saline (at least 3%) and/or dornase alfa for at least the
90 days prior to and including the Screening Visit and willing to continue daily use
for the 2-week screening period.
Exclusion Criteria:
• Active smoking or vaping.
• Use of an investigational drug within 28 days prior to and including the Screening
Visit.
• Changes to chronic therapy (e.g., ibuprofen, azithromycin, inhaled tobramycin,
aztreonam lysine) within 28 days prior to and including the Screening Visit. This
includes new airway clearance routines.
• Acute use of antibiotics (oral, inhaled or IV) or acute use of systemic
corticosteroids for respiratory tract symptoms within 7 days prior to and including
the Screening Visit.
• Chronic use of systemic corticosteroids at a dose equivalent to ≥ 10mg per day of
prednisone within 28 days prior to and including the Screening Visit.
• Antibiotic treatment for nontuberculous mycobacteria (NTM) within 28 days prior to and
including the Screening Visit.
Other: Discontinuation of hypertonic saline (HS), Other: Continuation of hypertonic saline (HS), Other: Discontinuation of dornase alfa (dnase), Other: Continuation of dornase alfa (dnase)
The Hydrocortisone and Extubation study will test the safety and efficacy of a 10 day course
of hydrocortisone for infants who are less than 30 weeks estimated gestational age and who
are intubated at 14-28 days of life. Infants will be randomized to receive hydrocortisone or
placebo. This study will determine if hydrocortisone improves infants'survival without
moderate or severe BPD and will be associated with improvement in survival without moderate
or severe neurodevelopmental impairment at 22 - 26 months corrected age.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Roy Heyne
13172
All
up to 30 Weeks old
Phase 3
This study is NOT accepting healthy volunteers
NCT01353313
STU 082017-069
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Inclusion Criteria:
• infants <30 weeks estimated gestational age
• inborn at an NRN site or were admitted to an NRN site before 72 hours postnatal age
• have received at least 7days of mechanical ventilation;
• are receiving mechanical ventilation through an endotracheal tube .
Exclusion Criteria:
• Major congenital anomalies
• Decision to limit support
• Indomethacin or ibuprofen treatment within 48 hours of study drug
• Previous corticosteroid treatment for BPD
• Received hydrocortisone for 14 or more cumulative days
• Received hydrocortisone within 7 days of study entry
Drug: Hydrocortisone, Drug: Placebo
Infant, Newborn, Bronchopulmonary Dysplasia, Infant, Small for Gestational Age, Infant, Premature, Infant, Very Low Birth Weight, Lung/Thoracic
NICHD Neonatal Research Network, Extremely Low Birth Weight (ELBW), Very Low Birth Weight (VLBW), Prematurity, Mechanical ventilation, Intubation, Neurodevelopmental impairment
Children’s Health; Parkland Health & Hospital System
Donor Milk vs. Formula in Extremely Low Birth Weight (ELBW) Infants
The Milk Trial seeks to determine the effect on neurodevelopmental outcomes at age 22-26
months of donor human milk as compared to preterm infant formula as the in-hospital diet for
infants whose mothers choose not to provide breast milk or are able to provide only a minimal
amount. Infants will be randomized to receive donor breast milk or formula during their
hospital stay. Infant's will be followed until they reach 22-26 months of age.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Myra Wyckoff
19272
All
up to 21 Days old
Phase 3
This study is NOT accepting healthy volunteers
NCT01534481
STU 022012-028
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Inclusion Criteria:
• Gestational age less than 29 weeks.
• Admitted to the NICU at less than or equal to 72 hours of life
• Survived at least 12 hours
Exclusion Criteria:
• Chromosomal anomalies
• Cyanotic congenital heart disease
• Diagnosed intrauterine infection
• Other congenital disorders known to impair neurodevelopment
• NEC or IP prior to seeking consent
• Decision documented to limit intensive care therapies
• Congenital disorders that may affect feeding
Feeding Group Eligibility:
• Sole Diet Group: Infants will be eligible for the sole diet feeding protocol if the
mother declines to provide breast milk for the baby.
• Supplemental Diet (minimal maternal milk) Group: Infants whose mothers initially
choose to provide breast milk and begin pumping will be re-screened for eligibility at
least weekly until the infant is 21 days old. If the mother stops expressing milk at
any point prior to the infant's 21st day of life, her infant will be eligible for
randomization. In addition, those whose mothers are providing less than 20% of the
infant's dietary needs (averaged over past 5 days) when the infant reaches 21 days of
age will be eligible for randomization at this point. No infant will be randomized
after reaching 21 days.
Biological: Donor Milk, Dietary Supplement: Preterm Formula
Infant, Newborn, Infant, Small for Gestational Age, Infant, Extremely Low Birth Weight
NICHD Neonatal Research Network, Extremely Low Birth Weight (ELBW), Prematurity, Neurodevelopmental Impairment, Donor Breast Milk, Preterm Formula
Children’s Health; Parkland Health & Hospital System
Individuals with a past diagnosis of severe combined immune deficiency (including many cases
of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and
marrow transplant, gene therapy, or enzyme replacement in the past may be eligible for this
study. The purpose of study is to look backwards at what has already been done in the. Over
800 patients with SCID are expected to be enrolled, making this one of the largest studies
ever to describe outcomes for patients with SCID treated at many different hospitals around
North America.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Victor Aquino
10208
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01346150
STU 032011-168
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Inclusion Criteria:
Strata A, B, and C (Part 1 •Retrospective Study)-
• Individuals with Severe Combined Immune Deficiency (SCID) diagnosis who:
--were treated at a location participating in this consortium from 1968 until present,
and
--are not enrolled in RDCRN PIDTC-6901 (ClinicalTrials.gov ID: NCT01186913).
• Subjects who received HCT/GT/ERT prior to the present date are eligible for the
retrospective study. The enrollment criteria for subjects who died prior to definitive
therapy are the same as for Strata A, B and C.
Stratum A, Typical SCID:
• Individuals who meet the following inclusion criteria and who received HCT are
eligible for enrollment into Stratum A of the study:
• Absence or very low number of T cells (CD3 T cells < 300/microliter), and no or
very low T cell function (< 10% of lower limit of normal) as measured by response
to phytohemagglutinin (PHA) or cells of maternal origin present.
• If maternal cells are present but the patient does not meet criteria for very low
T cell function as defined, the assigned reviewers for the potential subject, and
if necessary, the full PID-SCID RP will review the laboratory report to determine
if criteria of maternal engraftment are met for Protocol 6902.
• Laboratory report of testing for maternal engraftment is required, for evaluation
by the PID-SCID RP.
Stratum B, Leaky SCID, Omenn Syndrome, Reticular Dysgenesis:
Individuals who meet the following criteria are eligible for enrollment into Stratum B of
the study:
Leaky SCID-
• Maternal lymphocytes tested for and not detected and,
• Either one or both of the following (a,b):
a) < 50% of lower limit of normal T cell function (as measured by response to PHA OR <
50% of lower limit of normal T cell function as measured by response to CD3/CD28
antibody, b) Absent or < 30% lower limit of normal proliferative responses to candida
and tetanus toxoid antigens postvaccination or exposure,
• AND at least one of the following (a through e):
1. Reduced number of CD3 T cells,
2. > 80% of CD3+ or CD4 T cells are CD45RO+,
• AND/OR >80% of CD3+ or CD4+ T cells are,CD62L negative,
• AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of age),
• AND/OR are oligoclonal T cells. c) Hypomorphic mutation in IL2RG in a male, or
homozygous hypomorphic mutation or compound heterozygosity with at least one
hypomorphic mutation in an autosomal SCID-causing gene.
d) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is
below the lower limit of normal.
e) Functional testing in vitro supporting impaired, but not absent, activity of the
mutant protein,
• AND does not meet criteria for Omenn Syndrome,
• AND does not have known selective loss of lymphocytes, Ataxia- Telangiectasia, or
congenital heart defect associated with lymphopenia, unless a SCID genotype is
also present.
Omenn Syndrome (OS):
• Generalized skin rash,
• Maternal engraftment tested for and not detected,
• Absent or low (up to 30% of normal) T cell proliferation to antigens to which the
patient has been exposed.
• If the proliferation to antigen was not performed, but at least 4 of the following 10
supportive criteria, at least one of which must be among those marked with an asterisk
(*) are present, the patient is eligible: hepatomegaly; splenomegaly; lymphadenopathy;
elevated IgE; elevated absolute eosinophil count; *oligoclonal T cells measured by
CDR3 length or flow cytometry >80% of CD4+ T cells are CD45RO+ ;*proliferation to PHA
is reduced <50% of lower limit of normal or SI <30; *proliferative response in mixed
leukocyte reaction is reduced to increment cpm < 20% or SI <20; hypomorphic mutation
to SCID causing gene; low TRECs and/or percentage of CD 4+/ RA+/CD31+; or
CD4+/RA+/CD62L+ cells below the lower limit of normal.
Reticular Dysgenesis (RD):
• Absence or very low number of T cells (CD3 T cells <300/microliter),
• No or very low (<10% of lower limit of normal) T cell function (as measured by
response to phytohemagglutinin (PHA),
• Severe congenital neutropenia (absolute neutrophil count <200/microliter),
• AND at least one of the following:
• Sensorineural deafness and/or absence of granulopoiesis at bone marrow
examination and/or a deleterious AK2 mutation,
• absence of granulopoiesis on bone marrow examination; a pathogenic mutation in
the adenylate kinase 2 (AK2) gene identified.
Stratum C, SCID with Non-HCT Treatments:
-Individuals who meet the following criteria and were treated with PEG-ADA or gene therapy
with autologous modified cells are eligible for enrollment into Stratum C (SCID with
non-HCT treatments) of the study-
•Any SCID patient previously treated with a thymus transplant (includes intention to treat
with HCT, as well as PEG-ADA ERT or gene therapy).
Strata A, B, and C (Part 2 •Cross-Sectional Study):
Patient inclusion criteria for the cross sectional study: Eligibility for Strata A, B and C
are the same as for the retrospective study except that all the patients in the
cross-sectional study are currently surviving and are at least 2 years post the most recent
class of therapy.
Exclusion Criteria:
Parts 1 and 2 •Retrospective and Cross-Sectional Studies -
• Lack of appropriate testing to rule out HIV infection after 1997 (p24 antigen or more
sensitive) or other cause of secondary immunodeficiency,
• Presence of DiGeorge syndrome,
• Most patients with other PIDs such as nucleoside phosphorylase deficiency, ZAP70
deficiency, CD40 ligand deficiency, NEMO deficiency, XLP, cartilage hair hypoplasia or
ataxia telangiectasia will not meet the inclusion criteria for Stratum A, B, or C
above; however, a patient with one of the above may meet the inclusion criteria for
Stratum B and if so will be included-
• MHC Class I and MHC Class II antigen deficiency are excluded,
• Metabolic conditions that imitate SCID or related disorders such as folate
transporter deficiency, severe zinc deficiency, transcobalamin deficiency.
A Study to Compare the Long-term Outcomes After Two Different Anaesthetics (TREX)
There is considerable evidence that most general anaesthetics modulate brain development in
animal studies. The impact is greater with longer durations of exposure and in younger
animals. There is great controversy over whether or not these animal data are relevant to
human clinical scenarios.
The changes seen in preclinical studies are greatest with GABA agonists and NMDA antagonists
such as volatile anaesthetics (eg sevoflurane), propofol, midazolam, ketamine, and nitrous
oxide. There is less evidence for an effect with opioid (such as remifentanil) or with alpha
2 agonists (such as dexmedetomidine).
Some, but not all, human cohort studies show an association between exposure to anaesthesia
in infancy or early childhood and later changes in cognitive tests, school performance or
risk of developing neurodevelopmental disorders. The evidence is weak due to possible
confounding.
A recent well designed cohort study (the PANDA study) comparing young children that had
hernia repair to their siblings found no evidence for a difference in a range of detailed
neuropsychological tests. In that study most children were exposed to up to two hours of
anaesthesia. The only trial (the GAS trial) has compared children having hernia repair under
regional or general anesthesia and has found no evidence for a difference in neurodevelopment
when tested at two years of age. The GAS and PANDA studies confirm the animal data that short
exposure is unlikely to cause any neurodevelopmental impact.
The impact of longer exposures is still unknown. In humans the strongest evidence for an
association between surgery and poor neurodevelopmental outcome is in infants having major
surgery. However, this is also the group where confounding is most likely.
The aim of our study is to see if a new combination of anaesthetic drugs results in a better
long-term developmental outcome than the current standard of care for children having
anaesthesia expected to last 2 hours or longer.
Children will be randomised to receive either a low dose
sevoflurane/remifentanil/dexmedetomidine or standard dose sevoflurane anaesthetic.
They will receive a neurodevelopmental assessment at 3 years of age to assess global
cognitive function.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Peter Szmuk
80418
All
up to 2 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03089905
STU 052017-065
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Inclusion Criteria:
• Younger than 2 years (chronological age)
• Scheduled for anaesthesia that is expected to last at least 2 hours (and/or total
operating room time is scheduled to be at least 2.5 hours)
• Has a legally acceptable representative capable of understanding the informed consent
document and providing consent on the participant's behalf.
Exclusion Criteria:
• Known neurologic, chromosomal or congenital anomaly which is likely to be associated
with poor neurobehavioural outcome
• Existing diagnosis of behavioural or neurodevelopmental disability
• Prematurity (defined as < 36 weeks gestational age at birth)
• Birth weight less than 2 kg.
• Congenital cardiac disease requiring surgery
• Intracranial neurosurgery and intracranial craniofacial surgery (isolated cleft lip is
not an exclusion)
• Previous cumulative exposure to general anaesthesia exceeding 2 hours
• Planned future cumulative exposure to anaesthesia exceeding 2 hours before the age of
3 years.
• Any specific contra-indication to any aspect of the protocol
• Previous adverse reaction to any anaesthetic
• Circumstances likely to make long term follow-up impossible
• Living in a household where the primary language spoken at home is not a language in
which we can administer the Wechsler Preschool and Primary School Intelligence Scale
• Planned postoperative sedation with any agent except opioids (e.g. benzodiazepines,
dexmedetomidine, ketamine, barbiturates, propofol, clonidine, chloral hydrate, and
other non-opioid sedatives). For example if such sedation is planned for
post-operative ventilation
RATIONALE
The accrual of data from the laboratory and from epidemiologic and prevention trials has
improved the understanding of the etiology and pathogenesis of type 1 diabetes mellitus
(T1DM). Genetic and immunologic factors play a key role in the development of T1DM, and
characterization of the early metabolic abnormalities in T1DM is steadily increasing.
However, information regarding the natural history of T1DM remains incomplete. The TrialNet
Natural History Study of the Development of T1DM (Pathway to Prevention Study) has been
designed to clarify this picture, and in so doing, will contribute to the development and
implementation of studies aimed at prevention of and early treatment in T1DM.
Purpose:
TrialNet is an international network dedicated to the study, prevention, and early treatment
of type 1 diabetes. TrialNet sites are located throughout the United States, Canada,
Finland, United Kingdom, Italy, Germany, Sweden, Australia, and New Zealand. TrialNet is
dedicated to testing new approaches to the prevention of and early intervention for type 1
diabetes.
The goal of the TrialNet Natural History Study of the Development of Type 1 Diabetes is to
enhance our understanding of the demographic, immunologic, and metabolic characteristics of
individuals at risk for developing type 1 diabetes.
The Natural History Study will screen relatives of people with type 1 diabetes to identify
those at risk for developing the disease. Relatives of people with type 1 diabetes have
about a 5% percent chance of being positive for the antibodies associated with diabetes.
TrialNet will identify adults and children at risk for developing diabetes by testing for
the presence of these antibodies in the blood. A positive antibody test is an early
indication that damage to insulin-secreting cells may have begun. If this test is positive,
additional testing will be offered to determine the likelihood that a person may develop
diabetes. Individuals with antibodies will be offered the opportunity for further testing to
determine their risk of developing diabetes over the next 5 years and to receive close
monitoring for the development of diabetes.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Perrin White
17917
All
1 Year to 45 Years old
N/A
This study is also accepting healthy volunteers
NCT00097292
STU 042011-074
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Inclusion Criteria:
• Individuals 1 to 45 years old who have an immediate family member with type 1
diabetes (such as a child, parent, or sibling)
• Individuals 1-20 years old who have an extended family member with type 1 diabetes
(such as a cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)
Exclusion Criteria:
To be eligible a person must not:
• Have diabetes already
• Have a previous history of being treated with insulin or oral diabetes medications.
• Currently be using systemic immunosuppressive agents (topical and inhaled agents are
acceptable)
• Have any known serious diseases
Diabetes Mellitus, Type 1, Pancreas
"at risk" for developing type 1 diabetes, T1DM, T1D, juvenile diabetes, Type 1 Diabetes TrialNet, TrialNet
UT Southwestern; Children’s Health; Parkland Health & Hospital System
STaph Aureus Resistance-Treat Early and Repeat (STAR-TER) (STAR-TER)
To evaluate the micro-biologic efficacy and safety of a streamlined treatment for early onset
methicillin-resistant staphylococcus aureus (MRSA) in patients with cystic fibrosis.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Preeti Sharma
117060
All
2 Years to 45 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03489629
STU 022018-089
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Inclusion Criteria:
1. Male or female ≥ 2 and ≤ 45 years of age at the Screening Visit.
2. Documentation of a CF diagnosis as evidenced by one or more clinical features
consistent with the CF phenotype and one or more of the following criteria:
1. sweat chloride ≥ 60 milliequivalents/liter by quantitative pilocarpine
iontophoresis test (QPIT)
2. two well-characterized mutations in the cystic fibrosis transmembrane conductive
regulator (CFTR) gene
3. abnormal nasal potential difference(NPD) (change in NPD in response to a low
chloride solution and isoproteronol of less than -5 mV)
3. First OR early MRSA colonization defined as:
1. First MRSA colonization: first documented isolation of MRSA from respiratory
tract occurred ≤ 6 months prior to screening
2. Early MRSA colonization: MRSA was previously isolated from the respiratory tract
≤ 2 times over the past 3.5 years, but this was followed by at least 1 year of
documented negative cultures for MRSA
4. MRSA is available to the central laboratory •either the incident MRSA isolate from
the clinic visit or the subject is MRSA positive at the screening visit
5. Clinically stable with no significant changes in health status within the 14 days
prior to screening
6. Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative and ability for subject to comply with the requirements
of the study
Exclusion Criteria:
1. Received antibiotics with activity against MRSA within 28 days prior to screening
2. Use of an investigational agent within 28 days prior to screening
3. For subjects ≥ 6 years of age: FEV1 at screening < 25% of predicted for age based on
the Wang (males < 18 years, females < 16 years) or Hankinson (males ≥ 18 years,
females ≥ 16 years) standardized equations
4. MRSA from the screening culture or the most recent clinical care visit within 6 months
prior to screening resistant to TMP/SMX
5. History of intolerance to topical chlorhexidine or mupirocin
6. History of intolerance to both TMP/SMX and minocycline
7. < 8 years of age and allergic or intolerant to TMP/SMX
8. ≥ 8 years of age and allergic or intolerant to TMP/SMX and MRSA isolate (from
screening or clinical care visit)is resistant to minocycline
9. For females of child bearing potential: pregnant, breastfeeding, or unwilling to use
barrier contraception through Day 42 of the study
10. Subjects with history of abnormal renal function will need screening labs showing
normal function Abnormal renal function is defined as estimated creatinine clearance
<50 mL/min using the:
1. Bedside Schwartz Equation for subjects <18 years of age, and
2. Levey Glomerular filtration rate (GFR) Equation for subjects ≥ 18 years of age.
11. Subjects with a history of abnormal liver function will need to have screening labs
showing normal transaminases. Liver dysfunction is defined as ≥3x upper limit of
normal (ULN), of serum aspartate transaminase (AST) or serum alanine transaminase
(ALT) or abnormal synthetic function
12. History of solid organ or hematological transplantation
13. Presence of a condition or abnormality that in the opinion of the Investigator would
compromise the safety of the patient or the quality of the data.
Estimate the risks and benefits of active treatment versus expectant management of a
symptomatic patent ductus arteriosus (sPDA) in premature infants.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Mambarambath Jaleel
85918
All
up to 21 Days old
Phase 3
This study is NOT accepting healthy volunteers
NCT03456336
STU-2019-0784
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Inclusion Criteria:
• Postnatal age 48 hours -21 days
• Infant 22 0/7 to 28 6/7 weeks gestation at birth
• sPDA, as defined as:
1. Mild, Moderate, or Severe Clinical Criteria with Small or Moderate size PDA on
echocardiogram
2. Mild or Moderate Clinical Criteria with Large PDA on echocardiogram
Exclusion Criteria:
• Cardiopulmonary compromise
• Known congenital heart disease (besides atrial septal defect or ventricular septal
defect)
• Known pulmonary malformation (e.g. congenital lobar emphysema, congenital pulmonary
adenomatous malformation)
• Any condition which, in the opinion of the investigator, would preclude enrollment
Other: Active Treatment, Other: Expectant Management
Optimizing (Longer, Deeper) Cooling for Neonatal Hypoxic-Ischemic Encephalopathy(HIE)
The Optimizing Cooling trial will compare four whole-body cooling treatments for infants born
at 36 weeks gestational age or later with hypoxic-ischemic encephalopathy: (1) cooling for 72
hours to 33.5°C; (2) cooling for 120 hours to 33.5°C; (3) cooling for 72 hours to 32.0°C; and
(4) cooling for 120 hours to 32.0°C. The objective of this study is to evaluate whether
whole-body cooling initiated at less than 6 hours of age and continued for 120 hours and/or a
depth at 32.0°C in will reduce death and disability at 18-22 months corrected age.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Lina Chalak
35027
All
up to 6 Hours old
N/A
This study is NOT accepting healthy volunteers
NCT01192776
STU 082010-342
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Inclusion Criteria:
Eligibility will be determined in a stepped process:
1. All infants with a gestational age ≥ 36 weeks will be screened for study entry if they
are admitted to the NICU with a diagnosis of fetal acidosis, perinatal asphyxia,
neonatal depression or encephalopathy.
2. Infants will be eligible if:
• They have a pH ≤ 7.0 or a base deficit ≥ 16m mEq/ L on umbilical cord or any
postnatal sample within 1 hour of age.
• If, during this interval, they have a pH between 7.01 and 7.15, a base deficit is
between 10 and 15.9 mEq/L, or a blood gas is not available, AND they have an
acute perinatal event AND either a 10-minute Apgar score ≤ 5 or assisted
ventilation initiated at birth and continued for at least 10 minutes.
3. Once these criteria are met, eligible infants will have a standardized neurological
examination performed by a certified physician examiner. Infants will be candidates
for the study when encephalopathy or seizures are present. For this study,
encephalopathy is defined as the presence of 1 or more signs in 3 of the following 6
categories:
• Level of consciousness: lethargy, stupor or coma;
• Spontaneous activity: decreased, absent;
• Posture: distal flexion, decerebrate;
• tone: hypotonia, flaccid or hypertonia, rigid;
• Primitive reflexes: a) suck, weak, absent; b) Moro, incomplete, flaccid;
• Autonomic nervous system: a) pupils: constricted, unequal, skew deviation or non
reactive to light; b) heart rate: bradycardia, variable heart rate or c)
respiration: periodic breathing, apnea.
Eligible infants from multiple births will be enrolled in the same arm of the study.
Exclusion Criteria:
• Inability to randomize by 6 hours of age
• Major congenital abnormality
• Major chromosomal abnormality (including Trisomy 21),
• Severe growth restriction (≤ 1800gm birth weight),
• Infant is moribund and will not receive any further aggressive treatment,
• Refusal of consent by parent
• Refusal of consent by attending neonatologist
• Infants with a core temperature < 33.5°C for > 1 hour at the time of screening by the
research team would not be eligible for the study.
Procedure: Whole-body Cooling
Infant, Newborn, Hypoxia, Brain, Hypoxia-Ischemia, Brain, Encephalopathy, Hypoxic-Ischemic, Hypoxic-Ischemic Encephalopathy, Ischemic-Hypoxic Encephalopathy, Brain and Nervous System
This observational study tests the feasibility of enrolling subjects and obtaining an
amplitude-integrated electroencephalogram (aEEG) within the first 72 hours of life, a second
aEEG recording between 72-168 hours of life, and weekly thereafter up to 36 weeks
post-menstrual age. It will enroll 85-100 infants between 401-1,000 grams birth weight OR
between 23 0/7 and 28 6/7 weeks gestational age born at the 7 participating NICHD Neonatal
Research Network sites.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Lina Chalak
35027
All
up to 72 Hours old
N/A
This study is NOT accepting healthy volunteers
NCT00873847
STU 032012-028
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Inclusion Criteria:
• Inborn infants
• Between 401 grams and 1,000 grams inclusive birth weight OR between 23 0/7 and 28 6/7
weeks inclusive gestational age
• Decision to provide full intensive care support
• Less than 72 hours of age
Exclusion Criteria:
• Non-intact skin at the central or parietal regions of scalp
• Presence of known or suspected congenital anomalies, including:
• Congenital central nervous system malformations
• Chromosomal anomalies or multiple congenital anomalies
• Complex congenital heart disease
• Inborn error of metabolism
• Acidosis (pH < 6.8 for > 2 hours)
• Persistent bradycardia [HR < 100 bpm] associated with hypoxia for > 2 hours
Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Electroencephalography
NICHD Neonatal Research Network, Very Low Birth Weight (VLBW), Extremely Low Birth Weight (ELBW), Prematurity, Amplitude-integrated Electroencephalography (aEEG)
This is a prospective, non-randomized, non-blinded observational study. The overarching goal
is to discover new disease-associated genes in children, while establishing a specific focus
on disorders where molecular characterization is most likely to lead to novel therapies. This
study will merge detailed phenotypic characterization of patients presenting to the Pediatric
Genetics and Metabolism Division in the Department of Pediatrics/Children's Medical Center at
Dallas and collaborating clinics with Next-Generation sequencing techniques to identify
disease-producing mutations. The primary objective of the study is to identify novel
pathogenic mutations in children with rare Mendelian disorders. A secondary objective of the
study is to establish normative ranges of a large number of metabolites from healthy newborns
and older children.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Ralph DeBerardinis
99018
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02650622
STU 112014-001
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Inclusion criteria of Cohort 1- Newborn:
• Subjects aged 1-2 days
• Subjects with gestational age 37-42 weeks
• Subjects with stable clinical status (admitted to normal newborn nursery)
Inclusion criteria of Cohort 2 •Older children:
• Subjects aged 0-18 years
Inclusion criteria of Cohort 3 •Diseased children:
Subjects (no age limit) with ANY phenotype as below:
• Confirmed metabolic or genetic diseases
• Suspected metabolic or genetic diseases
• Episodic metabolic decompensation (e.g. hypoglycemia, hyperammonemia, metabolic
acidosis)
• Developmental regression
• Major congenital malformation
• Other unexplained symptoms of potential genetic origin
Exclusion criteria of Cohort 1 •Newborn:
• Subjects with gestational age <37 weeks or >42 weeks
• Subjects with overt signs of metabolic dysfunction, distress or genetic diseases
including hypoglycemia, hyperglycemia, sepsis/shock, hypoxemia, or major congenital
malformation
• Subjects with mothers whose pregnancies were complicated by gestational diabetes,
gestational hyperglycemia, gestational hypertension, preeclampsia, or any other major
disorders.
Exclusion criteria of Cohort 2 •Older children:
• Subjects with confirmed metabolic or genetic diseases
• Subjects with suspected metabolic or genetic diseases
• Subjects with episodic metabolic decompensation (e.g. hypoglycemia, hyperammonemia,
metabolic acidosis)
• Subjects with developmental regression
• Subjects with major congenital malformation
Exclusion criteria of Cohort 3 •Diseased children No.
Procedure: Skin Biopsy
Genetic Diseases, Metabolic Diseases, Other
Metabolism, Genetics, Metabolomics, Genomics
UT Southwestern; Children’s Health; Parkland Health & Hospital System
STeroids to REduce Systemic Inflammation After Infant Heart Surgery (STRESS)
This study's objective is to determine the pharmacokinetics (PK)/pharmacodynamics (PD),
safety and efficacy of methylprednisolone in infants undergoing heart surgery with
cardiopulmonary bypass. This is a prospective, double blind, multi-center, placebo-controlled
safety and efficacy study. Blood samples will be collected from a subset of enrolled study
participants to evaluate multiple dose methylprednisolone PK/PD. Participants will be
randomized in a 1:1 fashion to intravenous methylprednisolone versus placebo. Study
drug/placebo will be administered 8 to 12 hours before the anticipated start time of surgery
and in the operating room at the time of initiation of cardiopulmonary bypass. Patients will
be followed for primary and secondary outcomes for the duration of their hospitalization.
Serious study drug-related adverse events will be collected for 7 days after the last dose of
study drug.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Ryan Butts
169606
All
up to 12 Months old
Phase 3
This study is NOT accepting healthy volunteers
NCT03229538
STU 072017-052
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Inclusion Criteria:
• Age < 1 year at the time of surgery
• Undergoing heart surgery with CPB as part of standard clinical care
• Availability and willingness of the parent/legally authorized representative to
provide written informed consent
Exclusion Criteria:
• < 37 weeks adjusted gestational age at time of surgery
• Any oral or intravenous steroid treatment within two days of surgery
• Any patient receiving any of the following medications within 2 days of surgery:
Amphoteracin B, aminoglutethimide, anticholesterases, warfarin, P450 3A4 inducers including
(but not limited to) carbamazepine, phenobarbital, phenytoin, rifampin, bosentan and
nafcillin or P450 3A4 inhibitors including (but not limited to) clarithromycin,
voriconazole, itraconazole, ketoconazole, ciprofloxacin, diltiazem, fluconazole,
erythromycin and verapamil.
• Infection contraindicating steroid use
• Preoperative mechanical circulatory support or active resuscitation at the time of
randomization
• Emergent surgery precluding steroid administration 8-12 hours before surgery
Drug: Methylprednisolone, Drug: Isotonic saline
Congenital Heart Disease in Children, Inflammatory Response, Cardiovascular
The investigators propose an open label, non-blinded, single center randomized controlled
feasibility study to find the optimal initial HFNC flow rate in children less than 12 months
old with clinically diagnosed moderate to severe bronchiolitis. This feasibility study is
projected to take 6 months over the Winter/Spring of 2020-2021. The study is consisted of 3
arms, comparing HFNC therapy at 1 L/kg/min, 1.5 L/kg/min, and 2 L/kg/min (20 L/min max).
Moderate to severe bronchiolitis is defined by RDAI of 6 or more.15
The primary outcome is treatment response to HFNC therapy defined by RDAI/Respiratory
Assessment Change Score (RACS) ≥ 4 at 4 hours of therapy. Secondary outcome measures comprise
of treatment failure requiring an escalation of care during the first 24 hours of HFNC
therapy, duration of HFNC and simple nasal cannula therapy, duration of simple nasal cannula
therapy, hospital and PICU length of stay (LOS), time to treatment failure, and adverse
events.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Amy Cheng
185763
All
up to 12 Months old
N/A
This study is NOT accepting healthy volunteers
NCT04517344
STU-2020-0816
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Inclusion Criteria:
• Patients less than 12 months of age with: (1) clinical signs of bronchiolitis defined
by AAP and (2) respiratory distress defined by RDAI score 6 or greater.
Exclusion Criteria:
• Infants who required immediate need for respiratory support such as non-invasive
positive pressure ventilation (NIPPV) or invasive ventilation, or with congenital
heart disease, immunocompromise, upper airway obstruction, chronic lung disease,
bronchopulmonary dysplasia, infants on home oxygen therapy basilar skull fracture,
facial traumas, craniofacial malformations, and infants admitted to the neonatal or
cardiac ICUs. Patients who have received bronchodilator or steroid treatments are not
excluded as previous studies have not shown these treatments to be effective in
bronchiolitis management.
Other: Initial Flow Rate
Bronchiolitis
Bronchiolitis, HFNC, High Flow Nasal Cannula, Infants
Randomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants (ValEAR)
The overall goal of this study is to determine the clinical benefit and safety of antiviral
therapy for asymptomatic congenital cytomegalovirus (cCMV) infected hearing-impaired infants.
We will conduct a multi-center double-blind randomized placebo-controlled trial to determine
whether hearing-impaired infants with asymptomatic cCMV have better hearing and language
outcomes if they receive valganciclovir antiviral treatment. We will also determine the
safety of antiviral valganciclovir therapy for asymptomatic cCMV-infected hearing impaired
infants. This study will be unique in that the cohort enrolled will only include
hearing-impaired infants with asymptomatic cCMV.
Primary Objective: To determine if treatment of cCMV-infected hearing impaired infants with
isolated hearing loss with the antiviral drug valganciclovir reduces the mean slope of total
hearing thresholds over the 20 months after randomization compared to untreated cCMV-infected
infants with isolated hearing loss.
Main Secondary Objectives:
1. To determine if valganciclovir treatment improves the following outcomes when compared
to the control group:
1. The slope of best ear hearing thresholds over the 20 months after randomization.
2. The MacArthur-Bates Communicative Development Inventory (CDI) percentile score for
words produced at 20 months of age.
2. To evaluate safety measures based on all grade 3 or greater new adverse events
designated by the NIAID Division of AIDS (DAIDS) toxicity tables.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Kenneth Lee
93887
All
1 Month to 12 Months old
Phase 2
This study is NOT accepting healthy volunteers
NCT03107871
STU-2019-1329
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Inclusion Criteria:
• Age greater than or equal to 1 month and less than or equal to 12 months at the time
of randomization; AND
• Positive congenital CMV by urine culture or polymerase chain reaction test(PCR), OR
saliva culture or PCR followed by confirmatory urine PCR by 21 days of age, OR urine
culture or PCR after 21 days of age followed by newborn dry blood spot PCR; AND
• Confirmed sensorineural hearing loss (SNHL) by auditory brainstem response (ABR)
testing. For ABR assessments, hearing loss is defined as levels greater than 25 dB
normal hearing levels (NHL) at 1, 2, or 4 kHz in one or both ears.
Exclusion Criteria:
• Imminent demise; OR
• Known hypersensitivity reaction to valganciclovir, ganciclovir, or any components of
the investigational product formulation; OR
• ALT (Alanine Aminotransferase) five times baseline U/L, hepatomegaly, or significant
gastrointestinal disorders (e.g., eosinophilic esophagitis, ulcerative colitis); OR
• Absolute neutrophil count (ANC) less than 500 cells/mm^3, Hemoglobin less than 8 g/dL,
or platelets less than 50,000/mm^3, splenomegaly, or significant hematologic disorders
(e.g., hemophilia, leukemia, sickle cell anemia); OR
• Creatinine clearance less than 60 mL/min/1.73m^2 or significant renal disorders (e.g.,
nephrotic syndrome); OR
• Receiving other antiviral medications or immune globulin therapy; OR
• Receiving other investigational drugs; OR
• Breast feeding from a mother receiving antiviral or immunosuppressive medication; OR
• Known HIV positive mother (risk of immunosuppression); OR
• Subject is currently using list of prohibited medication specified by the package
insert; OR
• Other known cause contributing to SNHL (e.g., meningitis, aminoglycoside ototoxicity);
OR
• Bilateral profound SNHL or auditory neuropathy spectrum disorder; OR
• Existing conductive hearing loss or mixed permanent hearing loss is present; OR
• Evidence of intracranial calcification; OR
• Evidence of hydrocephalus; OR
• Microcephaly; OR
• Presence of petechiae; OR
• Intrauterine growth retardation; OR
• Chorioretinitis, optic atrophy or pale optic nerves; OR
• Parent or guardian unable to speak English or Spanish; OR
• Subject exposed to a language other than English or Spanish a majority of the time; OR
• Subject unable to complete hearing assessments or parent/guardian unable to complete
communication questionnaires; OR
• < 32 weeks gestational age at birth; OR
• Weight at the time of birth < 1800 g.