Refine your search

Search Results within category "Diabetes & Hormones"

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.

Search all categories
49 Study Matches

Phase 1 Study of MM-398 Plus Cyclophosphamide in Pediatric Solid Tumors

This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The goal is to find the highest dose of MM-398 that can be given safely when it is used together with the chemotherapy drug Cyclophosphamide.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Patrick Leavey
35610
All
12 Months to 20 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT02013336
STU 092013-007
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Histologically or cytologically-confirmed Ewing sarcoma, rhabdomyosarcoma, neuroblastoma, or osteosarcoma
• Disease progression after prior therapy in locally advanced or metastatic setting
• Measurable or evaluable disease based on the Response Evaluation Criteria in Solid Tumors (RECIST v1.1) criteria
• Age 12 months to <21 years
• Adequate bone marrow reserves, hepatic function, and renal function
• Recovered from effects of any prior surgery or cancer therapy
• Patients 18 years or older will provide written consent. A parent or legal guardian of a patient <18 years of age will provide informed consent and patients 11 to 18 years of age will provide written assent or as per participating institutional policy.
Exclusion Criteria:

• Clinically significant gastrointestinal disorders
• NYHA Class III or IV congestive heart failure, ventricular arrhythmias or uncontrolled blood pressure
• Active infection or unexplained fever
• Known hypersensitivity to any of the components of MM-398 or other liposomal products
• Recent Investigational therapy
• Pregnant or breast feeding; females of child-bearing potential must test negative for pregnancy at the time of enrollment
Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide
Neuroblastoma, Sarcoma, Ewing Sarcoma, Mycosis Fungoides, Rhabdomyosarcoma, Osteosarcoma, Recurrent or 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, Hodgkins Lymphoma, Kaposis sarcoma, Other Hematopoietic, Small Intestine, Soft Tissue
pediatric, MM-398, cyclophosphamide, irinotecan
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

Study of Etelcalceide in Pediatric Subjects With Secondary Hyperparathyroidism and CKD on Hemodialysis

Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis
Call 214-648-5005
studyfinder@utsouthwestern.edu
Mouin Seikaly
16504
All
up to 18 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03633708
STU-2019-0502
Show full eligibility criteria
Hide eligibility criteria
Inclusion criteria
• Dry weight ≥ 7 kg during screening.
• Diagnosed with CKD and SHPT undergoing hemodialysis at the time of screening.
• Diagnosis of SHPT with the mean of the 2 consecutive central laboratory iPTH values ≥ 400 pg/mL (42 pmol/L) during screening, on separate days and within 2 weeks of enrolment.
• Serum cCa value ≥ 9.0 mg/dL (2.25 mmol/L) for subjects ≥ 2 years of age and older and serum cCa value ≥ 9.6 mg/dL (2.4 mmol/L) for subjects 28 days to < 2 years of age obtained from the central laboratory during screening.
• Dialysate Ca level ≥ 2.5 mEq/L during screening.
• SHPT not due to vitamin D deficiency, per investigator assessment. Exclusion
• Anticipated or scheduled parathyroidectomy or kidney transplant during the study period.
• Subject has received a parathyroidectomy within 6 months prior to randomization.
• Receipt of cinacalcet therapy within 30 days prior to screening assessments and through randomization.
• Receipt of etelcalcetide within 6 months prior to screening assessments and through randomization.
Drug: Etelcalcetide
Chronic Kidney Disease, Secondary Hyperparathyroidism
sHPT, CKD, pediatric
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

Testicular Tissue Cryopreservation for Fertility Preservation

Call 833-722-6237
canceranswerline@utsouthwestern.edu
Ksenya Shliakhtsitsava
181933
Male
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02972801
STU-2020-1412
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Be male at any age.
• Be scheduled to undergo surgery, chemotherapy, drug treatment and/or radiation for the treatment or prevention of a medical condition or malignancy with risk of causing permanent and complete loss of subsequent testicular function.
• Or, have a medical condition or malignancy that requires removal of all or part of one or both testicles.
• Have newly diagnosed or recurrent disease. Those who were not enrolled at the time of initial diagnosis (i.e., patients with recurrent disease) are eligible if they have not previously received therapy that is viewed as likely to result in complete and permanent loss of testicular function.
• Have two testicles if undergoing elective removal of all or part of a testicle for fertility preservation only. Note: removal of both testicles will limit fertility preservation options.
• Sign an approved informed consent and authorization permitting the release of personal health information. The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent for specimen collection has been obtained, in accordance with institutional policies approved by the U.S. Department of Health and
• Consent for serum screening tests for infectious diseases [HIV-1, HIV-2, Hepatitis B, Hepatitis C], to be performed at the time of testicular tissue harvesting.
• Undergo a full history and physical examination and obtain standard pre-operative clearance (based on the most recent ACC/AHA Guideline for Perioperative Cardiovascular Evaluation for Noncardiac Surgery) as determined by their primary surgeon.
• Participating in long term follow-up is a requirement of the protocol.
Exclusion Criteria:

• Diagnosed with psychological, psychiatric, or other conditions which prevent giving fully informed consent.
• Diagnosed with an underlying medical condition that significantly increases their risk of complications from anesthesia and surgery.
Procedure: Testicular biopsy
Lymphoma, Sarcoma, Cancer, Mycosis Fungoides, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Male, Carcinoid Tumor, Cardiovascular, Colon, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Nose, Other Digestive Organ, Other Endocrine System, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Leukemia, Other, Hodgkins Lymphoma, Heart, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Other Hematopoietic, Small Intestine, Soft Tissue, Unknown Sites, Ill - Defined Sites, Autoimmune Disorders
Spermatogonial stem cells, Testis, Fertility, Infertility, Oncofertility
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

Safety and Efficacy of AT-001 in Patients With Diabetic Cardiomyopathy

Call 214-648-5005
studyfinder@utsouthwestern.edu
Ambarish Pandey
125045
All
40 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04083339
STU-2020-0312
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Type 2 Diabetes Mellitus
• Echocardiographic demonstration of diabetic cardiomyopathy
• Peak VO2 < 75% of predicted normal value based on age and gender
Exclusion Criteria:

• Prior diagnosis or signs/symptoms of overt/symptomatic heart failure / stage C heart failure
• Prior echocardiogrphic measurement of ejection fraction (EF) < 40%
• Prior acute coronary syndrome (ACS), coronary artery bypass graft (CABG), percutaneous coronary intervention (PCI), coronary artery disease (CAD) or stroke
• Severe or moderate cardiac valve disease requiring intervention
• Clinically significant arrhythmia
• Prior diagnosis of congenital, infective, toxic, infiltrative, post-partum, or hypertrophic cardiomyopathy
• Blood pressure > 140 mmHg (systolic) or > 90 mmHg (diastolic) at screening
• HbA1c >8.5% at screening
• Severe disease that would impact the performance of a cardio-pulmonary exercise test
Drug: AT-001, Drug: Placebo
Diabetic Cardiomyopathies
Type 2 Diabetes, Aldose Reductase Inhibitor, Stage B Heart Failure, Stage C Heart Failure, Cardiopulmonary Exercise Test
Parkland Health & Hospital System
  Email this study information to me
  Contact the study team
  See more information

The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study

This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling may result in identifying changes in genes of the tumor that indicate that a particular therapy may have activity. This is called an individualized cancer therapy (iCat) recommendation. The results of the tumor profiling and, if applicable, the iCat recommendation will be returned.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Laura Klesse
13954
All
up to 30 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02520713
STU 072015-038
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
• Diagnosis -- Histologic diagnosis of solid malignancy (excluding brain tumors and lymphoma) that meets at least one of the following criteria:
• Refractory, defined as tumor progression after initiation of standard first line therapy without having achieved a prior partial or complete remission OR Biopsy proven residual disease at the completion of planned standard initial front-line therapy.
• Recurrent, defined as tumor progression after achieving a prior partial or complete remission
• Newly diagnosed high risk disease, defined as having an expected event free survival of < 50% at 2 years.
• Lacks definitive diagnosis or classical genomic findings after histologic review and standard molecular testing (rare tumor group).
• Examples include (eligibility not limited to these examples):
• Histology typically associated with a fusion in which fusion is not detected.
• Ewing-like sarcoma
• Undifferentiated sarcoma
• Inflammatory myofibroblastic tumor without ALK fusion
• Infantile fibrosarcoma without NTRK fusion
• Specimen Samples
• Sufficient tumor specimen available to meet the minimum requirements for profiling from diagnosis or progression / recurrence --- OR
• Surgery / biopsy planned as part of clinical care that is anticipated to yield sufficient material to meet the minimum requirements for profiling; OR
• Patient has already had molecular profiling and patient has not yet started matched targeted therapy based on the report .
Exclusion Criteria:

• No Therapy Planned -- Patients who have declined further anticancer therapy will be excluded.
• Performance Status -- Patients with Lansky (age < 16 years) or Karnofsky (age ≥16 years) score < 50 will be excluded.
• Life Expectancy -- Patients with anticipated life expectancy < 3 months will be excluded.
Genetic: Genetic testing and GAIN report
Sarcoma, Pediatric Solid Tumor, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, 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, Heart, Kaposis sarcoma, Small Intestine, Soft Tissue, Ill - Defined Sites
Pediatric Solid Tumor
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

Preventing Metabolic Side Effects of Thiazide Diuretics With KMgCitrate

Chlorthalidone (CTD) may produce various metabolic disturbances, including hypokalemia, activation of Renin-Angiotensin- Aldosterone (RAA) system, oxidative stress, dyslipidemia, Fibroblast growth factor 23 (FGF23) synthesis, and magnesium depletion. These factors may interact with each other to contribute to the development of insulin resistances and metabolic syndrome. Smaller studies have suggested that Potassium magnesium Citrate (KMgCit) can ameliorate CTD- induced metabolic side effects independent of correction of hypokalemia. This study will tests if KMgCit ameliorates CTD induced metabolic effects independent of correction of hypokalemia.
Call 214-648-5940
danielle.pittman@utsouthwestern.edu
or
Call 214-648-5005
studyfinder@utsouthwestern.edu
Wanpen Vongpatanasin
17620
All
21 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02665117
STU 092015-058
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Treated or untreated stage I hypertension
Exclusion Criteria:

• Diabetes mellitus,
• Renal impairment (serum creatinine > 1.4 mg/dL),
• Any heart diseases such as congestive heart failure, sustained arrhythmia, or coronary heart disease,
• Chronic regular NSAID use,
• Allergy to thiazide diuretics,
• Gastro-esophageal reflux disease (GERD) requiring treatment with acid reducing agents or antacid more than once a week,
• Esophageal-gastric ulcer or history of gastrointestinal bleeding,
• Chronic diarrhea, vomiting,
• Excessive sweating,
• Unprovoked hypokalemia (serum K < 3.5 mmol/L) or hyperkalemia (serum K > 5.3 mmol/L),
• Abnormal liver function test (Aspartate transaminase (AST) or Alanine transaminase (ALT) above upper limit of normal range),
• Subjects on any potassium supplement on a regular basis for any reason, such as patients with primary aldosteronism,
• Pregnancy,
• History of major depression, bipolar disorder, or schizophrenia,
• History of substance abuse,
• Gout,
• Metabolic alkalosis, with serum bicarbonate > 32 meq/L,
• Severe dietary salt restriction, less than1/2 spoonful or 50 meq sodium/day.
• Patient with Claustrophobia will not have MRI but can still participate in the study without MRI
• Metal implants will not have MRI but can still participate in the study without MRI
Drug: Potassium Magnesium Citrate (KMgCit), Drug: Potassium Chloride (KCl), Drug: Chlorthalidone
Hypertension, Cardiovascular, Other Endocrine System
Hypokalemia, Isoprostanes, Insulin resistance, Aldosterone, Hepatic fat, Muscle Magnesium, Liver fat
  Email this study information to me
  Contact the study team
  See more information

A Research Study to See How Semaglutide Works Compared to Placebo in People With Type 2 Diabetes and Chronic Kidney Disease (FLOW)

The researchers are doing this study to see if semaglutide can slow down the growth and worsening of chronic kidney disease in people with type 2 diabetes. Participants will get semaglutide (active medicine) or placebo ('dummy medicine'). This is known as participants' study medicine - which treatment participants get is decided by chance. Semaglutide is a medicine, doctors can prescribe in some countries for the treatment of type 2 diabetes. Participants will get the study medicine in a pen. Participants will use the pen to inject the medicine in a skin fold once a week. The study will close when there is enough information collected to show clear result of the study. The total time participants will be in this study is about 3 to 5 years, but it could be longer.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ildiko Lingvay
55880
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03819153
STU-2019-0651
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female, age above or equal to 18 years at the time of signing informed consent. Japan: Male or female, age above or equal to 20 years at the time of signing informed consent
• Diagnosed with type 2 diabetes mellitus
• HbA1c less than or equal to 10% (less than or equal to 86 mmol/mol)
• Renal impairment defined either by: 1. serum creatinine-based eGFR greater than or equal to 50 and less than or equal to 75 mL/min/1.73 m^2 (CKD-EPI) and UACR greater than 300 and less than 5000 mg/g or 2. serum creatinine-based eGFR greater than or equal to 25 and less than 50 mL/min/1.73 m^2 (CKD-EPI) and UACR greater than 100 and less than 5000 mg/g
• Treatment with maximum labelled or tolerated dose of a renin-angiotensin-aldosterone system (RAAS) blocking agent including an angiotensin converting enzyme (ACE) inhibitor or an angiotensin II receptor blocker (ARB), unless such treatment is contraindicated or not tolerated. Treatment dose must be stable for at least 4 weeks prior to the date of the laboratory assessments used for determination of the inclusion criteria for renal impairment and kept stable until screening
Exclusion Criteria:

• Congenital or hereditary kidney diseases including polycystic kidney disease, autoimmune kidney diseases including glomerulonephritis or congenital urinary tract malformations
• Use of any glucagon-like peptide-1 (GLP-1) receptor agonist within 30 days prior to screening
• Myocardial infarction, stroke, hospitalisation for unstable angina pectoris or transient ischaemic attack within 60 days prior to the day of screening
• Presently classified as being in New York Heart Association (NYHA) Class IV heart failure
• Planned coronary, carotid or peripheral artery revascularisation
• Current (or within 90 days) chronic or intermittent haemodialysis or peritoneal dialysis
• Uncontrolled and potentially unstable diabetic retinopathy or maculopathy. Verified by a fundus examination performed within the past 90 days prior to screening or in the period between screening and randomisation. Pharmacological pupil-dilation is a requirement unless using a digital fundus photography camera specified for non-dilated examination
Drug: Semaglutide, Drug: Placebo (semaglutide)
Diabetes Mellitus, Type 2, Kidney
Parkland Health & Hospital System
  Email this study information to me
  Contact the study team
  See more information

A Heart Disease Study of Semaglutide in Patients With Type 2 Diabetes (SOUL)

The researchers are doing this study to look whether the type 2 diabetes medicine, semaglutide, has a positive effect on heart disease. Participants will either get semaglutide tablets or placebo tablets ("dummy" medicine) - which treatment is decided by chance. Participants must take one tablet with water every morning on an empty stomach and not eat or drink anything for at least 30 minutes. The study will last for about 3.5-5 years. Participants will have up to 25 clinic visits and 1 phone call with the study doctor. Women cannot be in the study if pregnant, breast-feeding or if they plan to become pregnant during the study period.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ildiko Lingvay
55880
All
50 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03914326
STU-2019-0647
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female, age equal to or above 50 years at the time of signing informed consent
• Diagnosed with type 2 diabetes mellitus
• HbA1c 6.5%
•10.0% (47
•86 mmol/mol) (both inclusive) (latest available and no more than 30 days old local laboratory assessment based on medical records or point of care measurement)
• At least one of the below conditions (a-d): a) Coronary heart disease defined as at least one of the following: i. Prior myocardial infarction ii. Prior coronary revascularisation procedure iii. 50% or above stenosis in coronary artery documented by cardiac catheterisation, computerized tomography coronary angiography iv. Coronary heart disease with ischaemia documented by stress test with any imaging modality b) Cerebrovascular disease defined as at least one of the following: i. Prior stroke ii. Prior carotid artery revascularisation procedure iii.50% or above stenosis in carotid artery documented by X-ray angiography, magnetic resonance angiography, computerized tomography angiography or Doppler ultrasound c) Symptomatic peripheral artery disease (PAD) defined as at least one of the following: i. Intermittent claudication with an Ankle-brachial index (ABI) below 0.85 at rest ii. Intermittent claudication with a 50% or above stenosis in peripheral artery (excluding carotid) documented by X-ray angiography, magnetic resonance angiography, computerized tomography angiography or Doppler ultrasound iii. Prior peripheral artery (excluding carotid) revascularization procedure iv. Lower extremity amputation at or above ankle due to atherosclerotic disease (excluding e.g. trauma or osteomyelitis) d) Chronic kidney disease defined as: i. eGFR below 60 mL/min/1.73 m^2 (based on medical records using latest available and no more than 6 months old assessment)
Exclusion Criteria:

• Any of the following: myocardial infarction, stroke, hospitalisation for unstable angina pectoris or transient ischaemic attack within the past 60 days prior to the day of screening
• Planned coronary, carotid or peripheral artery revascularisation known on the day of screening
• Heart failure presently classified as being in New York Heart Association Class IV
• Treatment with any glucagon-like peptide-1 receptor agonist within 30 days before screening
Drug: Semaglutide, Drug: Placebo
Diabetes Mellitus, Type 2
Parkland Health & Hospital System
  Email this study information to me
  Contact the study team
  See more information

Research Study to Compare Three Doses of Semaglutide Tablets Taken Once Daily in People With Type 2 Diabetes (PIONEER PLUS)

This study compares three doses of once daily semaglutide tablets in people with type 2 diabetes who were previously treated with other oral anti-diabetic medicines. Participants will be initiated on the lowest starting dose of 3 mg and gradually increased until they reach the final trial dose of 14 mg, 25 mg or 50 mg once daily semaglutide tablets. The final three doses will be randomized (i.e., decided by chance). Participants will be administered one tablet per day for 68 weeks. Women cannot take part if they are pregnant, breast-feeding or planning to become pregnant during the study period. Women who can get pregnant will be checked for pregnancy via urine tests. Once daily semaglutide tablets (3 mg, 7 mg and 14 mg) are approved for the treatment of type 2 diabetes in the US, in the EU and in some other countries, under the brand name Rybelsus®.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ildiko Lingvay
55880
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04707469
STU-2020-1301
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female, age above or equal to 18 years at the time of signing informed consent.
• Diagnosed with type 2 diabetes mellitus at least 180 days prior to the day of screening.
• HbA1c of 8.0-10.5% (64-91 mmol/mol) (both inclusive).
• BMI equal to or above 25 kg/m^2
• Stable daily dose(s) for 90 days prior to the day of screening of any of the following treatment regimens:
• No more than 3 of the following oral anti-diabetic drugs and at least 1 marked with a *:
• Metformin (equal to or above1500 mg or maximum tolerated or effective dose).
• Sulfonylureas (SU) (equal to or above half of the maximum approved dose according to local label or maximum tolerated or effective dose).
• Sodium/glucose cotransporter 2 (SGLT2) inhibitors (maximum tolerated dose).
• Dipeptidyl peptidase-4 (DPP-4) inhibitors (maximally indicated dose as per local label).
• Subjects, on treatment with stable dose of DPP-4 inhibitors at inclusion, must be willing to discontinue DPP-4 inhibitor treatment at randomisation (with no wash-out).
Exclusion Criteria:

• Treatment with any medication indicated for the treatment of diabetes or obesity other than stated in the inclusion criteria within the past 90 days prior to the day of screening. However, short term insulin treatment for a maximum of 14 days prior to the day of screening is allowed.
• Renal impairment measured as estimated glomerular filtration rate (eGFR) value of below 30 mL/min/1.73 m^2 according to Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation as defined by kidney disease improving global outcomes (KDIGO 2012) classification.
• Uncontrolled and potentially unstable diabetic retinopathy or maculopathy. Verified by a fundus examination performed within the past 90 days prior to screening or in the period between screening and randomisation. Pharmacological pupil-dilation is a requirement unless using a digital fundus photography camera specified for non-dilated examination.
Drug: Oral semaglutide
Diabetes Mellitus, Type 2
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

Children with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency tend to have elevated circulating levels of androgens, which can accelerate skeletal maturation and adversely impact adult height. Additionally, these children require supraphysiologic doses of hydrocortisone to suppress secretion of adrenal androgen precursors, and this treatment can retard linear growth. This study seeks to use oral abiraterone acetate (Zytiga)as an adjunct to approved CAH therapy (oral hydrocortisone and fludrocortisone) for pre-pubescent children with classic 21-hydroxylase deficiency in order to reduce daily requirement of hydrocortisone. In this Phase 1 study, the investigators will determine the minimum effective dose of abiraterone acetate that normalizes androstenedione levels during the 7-day Treatment Period.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Perrin White
17917
All
2 Years to 9 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT02574910
STU 112014-087
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
1. Pre-pubescent girls (age 2 years [12 kg minimum] to 8 years inclusive; skeletal age <10 years) or boys (age 2 years [12 kg] to 9 years inclusive; skeletal age <11 years). 2. Confirmed classic 21-hydroxylase deficiency evident by genotype groups A, A1 or B or clinical course (e.g., adrenal crisis with documented hyperkalemia and hyponatremia, at diagnosis or during a later evaluation; ambiguous genitalia in females). Documentation of one or both parents' genotypes may be required to confirm the subject's genotype. 3. Requirement for standard of care fludrocortisone (any dose) and ≥10 mg/m2/day of hydrocortisone for at least 1 month prior to the study consent. 4. Morning serum androstenedione concentrations >1.5 x Upper limit normal (ULN) after 7 days of dosing with doses of hydrocortisone required for physiologic replacement. 5. At least one parent (or other legally acceptable representative) must sign the informed consent form before the performance of any study procedures, but both parents must sign if both have parental rights. Children who are capable of providing assent (typically 10 years of age and older) must sign an assent form before the performance of any study procedures
Exclusion Criteria:
1. Evidence of central puberty: Tanner Stage >2 for breast development in girls or testicular volume >4 mL in boys, or random luteinizing hormone (LH) >0.3 milli-international units (mIU)/mL. Subjects with pubic and/or axillary hair as the only sign of puberty onset will be allowed. 2. Current or history of hepatitis from any etiology, including history of active viral hepatitis A, B, or C. 3. Patients with baseline hepatic impairment are excluded from this trial. To be eligible for this protocol, patients must meet all of the following criteria: AST, ALT and Total bilirubin < ULN Albumin > lower limits of normal (LLN) No evidence of ascites No evidence of encephalopathy 4. Abnormalities of liver function developing during the study 5. Abnormal renal function tests, defined as blood urea nitrogen (BUN) or creatinine >1.5 ULN for age. 6. Significant anemia (hemoglobin < 12 g/dl). If documented to be due to iron deficiency, subjects may be rescreened 3 months after this has been treated. 7. Clinically significant abnormality in the 12-lead electrocardiogram (ECG) 8. A history of a malabsorption syndrome. 9. Evidence of active malignancy. 10. Serious or uncontrolled co-existent disease, including active or uncontrolled infection. Subjects may be rescreened after resolution of any such condition. 11. Concurrent medical condition or disease other than 21-hydroxylase deficiency that may interfere with linear growth or that requires concomitant therapy that is likely to interfere with study procedures or results. 12. Asthma or other condition requiring treatment with systemic corticosteroids within the past 3 months. Asthma treatment with inhaled corticosteroids is permitted. 13. Treatment with potentially hepatotoxic medications (statins); strong inhibitors of CYP3A4 (ketoconazole, itraconazole, clarithromycin, atazanavir, nefazodone, saquinavir, telithromycin, ritonavir, indinavir, nelfinavir, voriconazole), or CYP3A4 inducers (e.g., phenytoin, carbamazepine, rifampin, rifabutin, rifapentine, phenobarbital). CYP2C8 substrates (rosiglitazone, pioglitazone, rapaglinide) and CYP2D6 substrates (dextromethorphan, thioridazine) should be avoided 14. Treatment with medications to affect puberty or synthesis of sex steroids, including gonadotropin releasing hormone agonists, aromatase inhibitors, or androgen receptor blockers (e.g., flutamide, spironolactone). However, a gonadotropin releasing hormone agonist may be started during the study for treatment-emergent central puberty without disqualifying the subject 15. Treatment with growth hormone at enrollment or during the course of the study. 16. Known allergies, hypersensitivity, or intolerance to abiraterone acetate or its excipients (refer to United States Prescribing Information). 17. Has received an investigational drug within 4 weeks of the planned first dose of study drug or is currently enrolled in an investigational interventional study. 18. Any condition that, in the opinion of the investigator, would make participation not be in the best interest (eg, compromise the well-being) of the subject or that could prevent, limit, or confound the protocol-specified assessments. 19. Presence or history of cataracts.
Drug: Abiraterone acetate
Congenital Adrenal Hyperplasia, Other Endocrine System
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

A Research Study to Look at How Semaglutide Compared to Placebo Affects Diabetic Eye Disease in People With Type 2 Diabetes (FOCUS)

This study will look at the long-term effects of semaglutide (active medicine) on diabetic eye disease when compared to placebo (dummy medicine). The study will be performed in people with type 2 diabetes. Participants will either get semaglutide or placebo in addition to their diabetes medicines - which treatment the participant gets is decided by chance. Participants will inject the study medicine using a pen-injector. The medicine must be injected in a skin fold in the stomach, thigh or upper arm once a week. The study will last for 5 years.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ildiko Lingvay
55880
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03811561
STU-2019-0479
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female, age greater than or equal to 18 years at the time of signing informed consent
• Diagnosed with type 2 diabetes mellitus greater than or equal to 10 years prior to the day of screening
• HbA1c of 7.0-10.0% (53-86 mmol/mol) (both inclusive)
• Eye inclusion criteria (both eyes must meet all criteria):
• Early Treatment Diabetic Retinopathy Study (ETDRS) level of 10-75 (both inclusive) evaluated by fundus photography and confirmed by central reading centre
• No ocular or intraocular treatment for diabetic retinopathy or diabetic macular oedema twelve months prior to the day of screening
• No anticipated need for ocular or intraocular treatment for diabetic retinopathy or diabetic macular oedema within six months after randomisation
• Best-corrected visual acuity greater than or equal to 30 letters using the ETDRS visual acuity protocol
• No previous treatment with pan-retinal laser photocoagulation
• No substantial non-diabetic ocular condition that, in the opinion of the ophthalmologist, would impact diabetic retinopathy or diabetic macular oedema progression during the trial
• No substantial media opacities that would preclude successful imaging
Exclusion Criteria:

• Any of the following: myocardial infarction, stroke, hospitalization for unstable angina pectoris or transient ischaemic attack within the past 60 days prior to the day of screening
• Planned coronary, carotid or peripheral artery revascularisation known on the day of screening
• Subjects presently classified as being in New York Heart Association (NYHA) Class IV
• Renal impairment measured as estimated Glomerular Filtration Rate (eGFR) value of eGFR less than 30 ml/min/1.73 m^2
• Personal or first degree relative(s) history of multiple endocrine neoplasia type 2 or medullary thyroid carcinoma
• Presence or history of malignant neoplasms within the past 5 years prior to the day of screening. Basal and squamous cell skin cancer and any carcinoma in-situ are allowed
• Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using highly effective contraceptive methods
• Current or previous (within 30 days before screening) treatment with any glucagon like peptide-1 (GLP-1) receptor agonist or dipeptidyl peptidase-4 (DPP-4) inhibitor
• Receipt of any investigational medicinal product within 30 days before screening
• Previous participation in this trial. Participation is defined as randomisation
• Known or suspected hypersensitivity to trial products or related products
• Any disorder, which in the investigator's opinion might jeopardise subject's safety or compliance with the protocol
Drug: Semaglutide, Drug: Placebo (semaglutide)
Diabetes Mellitus, Type 2
Parkland Health & Hospital System
  Email this study information to me
  Contact the study team
  See more information

A Study of the Efficacy and Safety of Relacorilant in Patients With Endogenous Cushing Syndrome (GRACE)

This is a Phase 3, double-blind, placebo-controlled, randomized-withdrawal study to assess the efficacy, safety and pharmacokinetics (PK) of relacorilant in patients with endogenous Cushing syndrome and concurrent type 2 diabetes mellitus/impaired glucose tolerance and/or uncontrolled hypertension
Call 214-648-5005
studyfinder@utsouthwestern.edu
Oksana Hamidi
179331
All
18 Years to 80 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03697109
STU-2019-0789
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Has a confirmed diagnosis of endogenous Cushing syndrome
• Meets at least one of the following criteria:
• Has Type 2 diabetes mellitus
• Has impaired glucose tolerance
• Has hypertension
Exclusion Criteria:

• Has non-endogenous source of hypercortisolemia
• Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism
• Has poorly controlled hypertension
• Has poorly controlled diabetes mellitus
• Has severe renal insufficiency
Drug: Relacorilant, Other: Placebo
Cushing Syndrome, Other Endocrine System
Cushing syndrome, Cushing disease, Hypercortisolemia, Cushingoid, Type 2 Diabetes, Impaired Glucose Intolerance, Hypertension, Adrenocorticotropic hormone, Primary Pigmented Nodular Adrenal Disease, Moon Facies, Dorsocervical Fat Pad, Adrenal Adenoma, Adrenal Autonomy, Cortisol, Cushing
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

A Research Study to Compare a New Weekly Insulin, Insulin Icodec Used With DoseGuide App, and Daily Insulins in People With Type 2 Diabetes Who Have Not Used Insulin Before (ONWARDS 5)

This study compares insulin icodec to different daily insulins in people with type 2 diabetes. The study will look at how well insulin icodec taken once weekly controls blood sugar compared to the insulins taken once daily. Participants will either get insulin icodec, that participants will have to inject once a week on the same day of the week, or a marketed insulin, that participants will have to inject once a day. Which treatment participants get is decided at random. The insulin is injected with a needle in a skin fold in the thigh, upper arm or stomach. Participants will measure their blood sugar every day. Participants will get a study phone to record safety data in the electronic diary (eDiary). If participants get a daily insulin they will record their insulin doses in the eDiary. If Participants get weekly insulin icodec, participants study phone will also have the DoseGuide App. The DoseGuide App gives dose recommendations based on their blood sugar and previous doses. Participants will record their insulin doses in the DoseGuide App. The study will last for about 1 year and 2 months. Participants will have 8 planned clinic visits with the study doctor. More visits will be planned to meet individual needs. At 6 clinic visits participants will have blood samples taken. Women cannot take part if pregnant, breast-feeding or plan to become pregnant during the study period.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ildiko Lingvay
55880
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04760626
STU-2020-1406
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
• Male or female.
• Age above or equal to 18 years at the time of signing informed consent.
• Diagnosed with T2D greater than or equal to 180 days prior to the day of screening.
• HbA1c above 7.0% (53 mmol/mol) as measured by central lab.
• Insulin naïve. However, short term insulin treatment for a maximum of 14 days prior to the day of screening is allowed, as is prior insulin treatment for gestational diabetes.
• Stable daily dose(s) greater than or equal to 90 days prior to the day of screening of any of the following antidiabetic drug(s) or combination regimen(s): a .Any metformin formulations greater than or equal to 1500 mg or maximum tolerated or effective dose. b .Any metformin combination formulations greater than or equal to 1500 mg or maximum tolerated or effective dose. c. Any of the following non-insulin antidiabetic drug classes including combinations (greater than or equal to half of the maximum approved dose according to local label or maximum tolerated or effective dose):i). Sulfonylureas ii). Meglitinides (glinides) iii). DPP-4 inhibitors iv. SGLT2 inhibitors v). Thiazolidinediones vi). Alpha-glucosidase inhibitors vii). Oral combination products (for the allowed individual Oral Antidiabetic Drugs (OADs)) viii). Oral or injectable GLP-1-receptor agonists
• Intensification with insulin is indicated to achieve glycaemic target (4.4-7.2 mmol/L, 80-130 mg/dL) at the discretion of the treating investigator.
Exclusion Criteria:

• Known or suspected hypersensitivity to trial product(s) or related products.
• Previous participation in this trial. Participation is defined as signed informed consent.
• Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using an adequate contraceptive method (adequate contraceptive measures as required by local regulation or practice).
• Participation in any clinical trial of an approved or non-approved investigational medicinal product within 30 days before screening. (Simultaneous participation in a trial with the primary objective of evaluating an approved or non-approved investigational medicinal product for prevention or treatment of COVID-19 disease or postinfectious conditions is allowed if the last dose of the investigational medicinal product has been received more than 30 days before screening)
• Any disorder which in the investigator's opinion might jeopardise subject's safety
Drug: Insulin icodec, Drug: Insulin Glargine 100U/mL, Drug: Insulin Degludec, Drug: Insulin Glargine 300U/mL
Diabetes Mellitus, Type 2
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

Efficacy and Safety of Relacorilant in Patients With Cortisol-Secreting Adrenal Adenomas (GRADIENT)

This is a Phase 3, randomized, double-blind, placebo-controlled study to assess the efficacy, and safety of relacorilant to treat hypercortisolism in patients with cortisol-secreting adrenal adenoma or hyperplasia associated with diabetes mellitus/ impaired glucose tolerance and/or uncontrolled systolic hypertension.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Oksana Hamidi
179331
All
18 Years to 80 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04308590
STU-2020-0377
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Shows lack of cortisol suppression
• Suppressed or low early-morning ACTH levels
• A radiologically confirmed adrenal lesion
• Has IGT or DM
• Has uncontrolled hypertension
Exclusion Criteria:

• Has severe, uncontrolled hypertension
• Has poorly controlled DM
• Has significantly abnormal liver test results or severe renal insufficiency
• Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism
Drug: relacorilant, Other: Placebo
Hypercortisolism
Cushing syndrome, Cushing, Hypercortisolemia, Type 2 Diabetes, Impaired Glucose Intolerance, Hypertension, Adrenocorticotropic hormone, Primary Pigmented Nodular Adrenal Disease, Macronodular adrenal hyperplasia, Adrenal Adenoma, Adrenal Autonomy, Cortisol, Autonomous cortisol secretion
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

A Multiple Ascending Dose Trial Investigating Safety, Tolerability and Pharmacokinetics of NNC0361-0041 (TOPPLE T1D)

The trial is a placebo-controlled, double-blinded within cohorts, randomized, multiple ascending dose trial with a sequential trial design. The primary outcome is to investigate the safety and tolerability of ascending subcutaneous weekly doses of NNC0361-0041 plasmid in patients with T1D.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Perrin White
17917
All
18 Years to 45 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04279613
STU-2021-0175
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
1. Willing to provide Informed Consent 2. Participants must live in a location with rapid access to emergency medical services 3. Age 18-45 years (both inclusive) at the time of signing informed consent 4. Must have a diagnosis of T1D for less than 48 months at randomization 5. Must have at least one diabetes-related autoantibody present (GAD65A; mIAA, if obtained within 10 days of the onset of insulin therapy; IA-2A; ICA; or ZnT8A) 6. Must have stimulated C-peptide levels greater than or equal to 0.2 pmol/ml measured during an MMTT conducted at least 21 days from diagnosis of diabetes and within one month (37 days) of randomization 7. Be willing to comply with intensive diabetes management 8. HbA1c ≤8.5% at screening 9. Subjects who are CMV and/or EBV seronegative at screening must be CMV and/or EBV PCR negative within 30 days of randomization and may not have had signs or symptoms of a CMV and/or EBV compatible illness lasting longer than 7 days within 30 days of randomization 10. Be up to date on recommended immunizations 11. Be at least 6 weeks from last live immunization 12. Be at least 4 weeks from killed vaccine other than flu vaccine 13. Participants are required to receive killed influenza vaccination at least 2 weeks prior to randomization when vaccine for the current or upcoming flu season is available 14. Be willing and medically acceptable to postpone live vaccines during the treatment period and for 3 months following last dose of study drug 15. If participant is female with reproductive potential, she must have a negative pregnancy test at screening and be willing to avoid pregnancy using a highly effective contraceptive method for the 12 months of the study 16. Males of reproductive age must use adequate contraceptive method during the treatment phase and for 3 months following last dose of study drug 17. Be at least 2 weeks from receiving a single dose COVID-19 vaccine or at least 2 weeks from completing a multi-dose COVID-19 vaccine series at the time of receiving study drug.
Exclusion Criteria:
Potential participants must not meet any of the following exclusion criteria: 1. One or more screening laboratory values as stated 1. Leukocytes < 3,000/μL 2. Neutrophils <1,500 /μL 3. Lymphocytes <800 /μL 4. Platelets <100,000 /μL 5. Haemoglobin <6.2 mmol/L (10.0 g/dL) 6. Potassium >5.5 mmol/L or <3.0 mmol/L 7. Sodium >150mmol/L or < 130mmol/L 8. AST or ALT ≥2.5 times the upper limits of normal 9. Bilirubin ≥ 1.5 times upper limit of normal 10. Glomerular Filtration Rate (eGFR) value of eGFR < 60 ml/min/1.73 m2 as defined by KDIGO 2012 (43) 11. Any other laboratory abnormality that might, in the judgment of the investigator, place the subject at unacceptable risk for participation in this trial 2. Current or ongoing use of non-insulin pharmaceuticals that affect glycemic control within prior 7 days of screening 3. Use of other immunosuppressive agents including chronic use of systemic steroids. Topical products are acceptable (nasal, conjunctival, skin) 4. Have active signs or symptoms of acute infection at the time of randomization 5. Have current, confirmed COVID-19 infection 6. Chronic active infection other than localized skin infections 7. Have evidence of prior or current tuberculosis infection as assessed by PPD, interferon gamma release assay or by history 8. Have evidence of current or past HIV, Hepatitis B infection 9. Have evidence of active Hepatitis C infection 10. Vaccination with a live virus within the last 6 weeks and killed vaccine within 4 weeks (except 2 weeks for flu vaccine) 11. Be currently pregnant or lactating, or anticipate getting pregnant within the one-year study period. 12. Have severe obesity: adults BMI ≥ 40 13. Have a history of malignancies 14. Untreated hypothyroidism or active Graves' disease 15. History of severe reaction to prior vaccination 16. Participation in any clinical trial of an approved or non-approved investigational medicinal product within 30 days after last blood draw (or 5 half-lives of investigational drug, whichever is greater) before screening, or currently enrolled in any other clinical trial 17. Subject is the investigator or any sub-investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the trial 18. Supine blood pressure at screening outside the range of 90-139 mmHg for systolic or 50-89 mmHg for diastolic. To exclude white-coat nervousness a single repeat measurement is allowed 19. Have any complicating medical issues or abnormal clinical laboratory results that may interfere with study conduct, or cause increased risk 20. Any condition that in the investigator's opinion may adversely affect study participation or may compromise the study results
Drug: NNC0361-0041, Other: Placebo
Type I Diabetes
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

Stereotactic Radiosurgery (SRS) for Brain Metastasis (SRS)

SRS dose escalation for brain metastases in radiation-naïve patients will establish true tolerable doses, which may exceed the current standard doses. This may lead to an improvement in local control, patient survival, and/or quality-of life.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Robert Timmerman
69821
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02645487
STU 022015-106
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria 1. Biopsy-proven non-hematopoietic malignancy, except for small cell lung cancer, germ cell cancer, or unknown primary tumor. 2. Radiographic evidence by MRI (or by CT scan with CT contrast if ineligible or intolerant of MRI) of brain metastasis. (If patient is unable to tolerate MRI contrast, an MRI without contrast is acceptable if lesions are visible) 3. All brain metastases must be outside the brain stem (midbrain, pons and medulla). 4. Patient must have 10 or less brain metastases. 5. The maximum diameter of any lesion must be less than or equal to 3.0 cm. 6. Previous treatment with surgery, radiation, chemotherapy, immunotherapy or any targeted agents are allowed provided that:
• Radiation was not to the brain.
• Surgery to the brain was > 7 days prior to SRS and there remains at least one additional brain metastasis that can be targeted with SRS 7. Age ≥ 18 years. 8. ECOG Performance Score of 2 or better/Karnofsky Performance Status score of 50-60 or better. 9. All men, as well as women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. 10. A female of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
• Has not undergone a hysterectomy or bilateral oophorectomy; or
• Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months). Medically acceptable birth control (contraceptives) includes:
• Approved hormonal contraceptives (such as birth control pills, patch, or ring: Depo-Provera, Implanon), or
• Barrier methods (such as a condom or diaphragm) used with a spermicide (a substance that kills sperm) 11. Ability to understand and the willingness to sign a written informed consent. Exclusion Criteria 1. Patients had craniotomy and surgery to the brain within 7 days from the date of SRS. 2. Patients with leptomeningeal metastasis. NOTE: For the purposes of exclusion, LMD is a clinical diagnosis, defined as positive CSF cytology and/or equivocal radiologic or clinical evidence of leptomeningeal involvement. Patients with leptomeningeal symptoms in the setting of leptomeningeal enhancement by imaging (MRI) would be considered to have LMD even in the absence of positive CSF cytology, unless a parenchymal lesion can adequately explain the neurologic symptoms and/or signs. In contrast, an asymptomatic or minimally symptomatic patient with mild or nonspecific leptomeningeal enhancement (MRI) would not be considered to have LMD. In that patient, CSF sampling is not required to formally exclude LMD, but can be performed at the investigator's discretion based on level of clinical suspicion. 3. Patients with a contraindication to both MRI (with or without contrast) and CT scan (with contrast) 4. Patients with life expectancy < 3 months. 5. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that, in the opinion of the investigator, would limit compliance with study requirements. 6. Subjects must not be pregnant or nursing at the time of SRS treatment due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants.
Radiation: Stereotactic Radiosurgery
Brain Neoplasms, Adult, Malignant, Lymphoma, Sarcoma, Multiple Myeloma, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Cardiovascular, 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, Hodgkins Lymphoma, Lymphoid Leukemia, Small Intestine, Soft Tissue
Parkland Health & Hospital System
  Email this study information to me
  Contact the study team
  See more information

Improving Chronic Disease Management With Pieces (ICD-Pieces)

ICD-Pieces (Parkland Intelligent e-Coordination and Evaluation System) trial is a National Institutes of Health (NIH) Healthcare Systems(HCS) Collaboratory demonstration project to improve management of patients with a triad of Chronic Kidney Disease, hypertension and diabetes with Pieces in four HCS including Parkland, Texas Health Resources (THR), ProHealth Physicians Incorporation and North Texas VA. Pieces is a decision support technology platform created by Parkland Center for Clinical Innovation(PCCI). The primary objective is to test the hypothesis that a collaborative model of primary care and subspecialty care intervention enhanced by Pieces and practice facilitators compared to standard clinical practice will reduce all-cause hospitalizations in patients with coexisting chronic kidney disease, diabetes and hypertension. Secondary objectives are: a)Test if implementation of the collaborative model will reduce 30-day readmissions, emergency room visits, cardiovascular events or deaths and disease-specific hospitalizations; b) Develop and validate risk predictive models for disease-specific hospitalizations, all-cause hospitalizations, 30-day readmissions, emergency room visits, cardiovascular events and deaths for patients with chronic kidney disease, diabetes and hypertension. c) Collect demographic and clinical data to assist phenotyping patients with chronic kidney disease, diabetes and hypertension. d) Obtain safety data including Acute Kidney Injury, progression of chronic kidney disease, electrolyte disturbances and medication errors, and drug toxicity; e) Collect resource utilization information including hospitalizations, emergency room visits, outpatient visits, and diagnostic or therapeutic procedures completed. Candidate patients in selected clinics will be enrolled over a period of 2 years and followed for 12 months. Pieces will ascertain both primary and secondary outcomes from the Electronic Health Record supported with data from the Dallas Fort Worth Hospital Council (DFWHC), Accountable Care Organization (ACO) reports and VA database, and deaths from Social Security Index (SSI) data.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Miguel Vazquez
17567
All
18 Years to 85 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02587936
STU 062015-016
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria
• CKD Inclusion Criteria (present at least ≥ 3 months apart) 1. There will be two or more Estimated Glomerular Filtration Rate (eGFRs) calculations less than 60ml/minute (corrected for BSA) or 2. Two or more positive tests for albuminuria and/or proteinuria Albuminuria/proteinuria can be defined by quantitative criteria with albumin/creatinine ratio greater than 30mg/g, urine protein creatinine ratio greater than 200mg/g or positive dipstick with protein detection (adjusted for urinary concentration/specific gravity).
• Diabetes Inclusion Criteria Only patients with type 2 diabetes will be enrolled in this study. 1. Random blood glucose greater than 200mg/dL 2. Hemoglobin A1C greater than 6.5% 3. Use of hypoglycemic agents or 4. Type 2 diabetes included in problem list
• Hypertension Inclusion Criteria 1. Systolic blood pressure greater than 140 mmHg on two different occasions at least one week apart 2. Diastolic blood pressure greater than 90 on two occasions at least more than one week apart 3. Use of antihypertensive agents except thiazide diuretics or 4. Hypertension included in problem list
Exclusion Criteria:

• Exclusion criteria will be minimal in this pragmatic trial. The collaborative model of care will not be implemented in patients younger than 18 years or older than 85 years of age or patients who have CKD stage 5/End Stage Renal Disease(ESRD.
• Primary care practitioners have the option of not implementing the intervention on any of their patients if they believe benefit to be minimal or risk too high due to patient comorbidities
Other: Collaborative Model of Primary care and Subspecialty care
Type 2 Diabetes, Hypertension, Chronic Kidney Disease, Diabetes, High BP
Chronic, Risk prediction model, Quality Improvement (QI), Pragmatic trial, Cluster randomization, Collaborative care, clinical informatics, Chronic kidney disease, hypertension, high BP, diabetes
Parkland Health & Hospital System
  Email this study information to me
  Contact the study team
  See more information

Internet-Based Cognitive Behavioral Therapy for Depressive Symptoms in Adolescents With Type 1 Diabetes Mellitus

This study evaluates the use of an established internet-based cognitive behavioral therapy intervention in a group of adolescents with type 1 diabetes and mild to moderate depressive symptoms. Half of the participants will receive the internet-based intervention while the other half will receive usual care.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Olga Gupta
136963
All
13 Years to 17 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03655067
STU-2020-0230
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Age 13-17 receiving ongoing medical care in the Diabetes Clinic at Children's Medical Center Dallas.
• Patients must be fluent in English (the materials on the site have not been validated in any other language), be in at least the 8th grade, report/demonstrate comfort with use of a computer, and regular, convenient and discreet access to the internet.
• Patients must experience at least sub-threshold depression (CES-D score > 15).
• Patients must have had the diagnosis of type 1 diabetes for at least 12 months.
Exclusion Criteria:

• Patients who are medically unstable during their diabetes clinic visit will be excluded (i.e. diabetes ketoacidosis, symptomatic hypoglycemia).
• Patients who are too severely depressed for this form of intervention (i.e. meet criteria for MDD, endorse suicidal intent, PHQ-A score ≥ 20), those with a diagnosis or symptoms of severe mental illness (schizophrenia, bipolar disorder), prior psychiatric hospitalization, prior self-harm attempt.
• Patients receiving ongoing counseling or therapy services within the last year, by a licensed professional (counselor, psychologist or psychiatrist).
• Patients who are currently taking or begin taking psychotropic medications during study participation will be excluded/withdrawn.
Behavioral: CATCH-IT
Type 1 Diabetes Mellitus, Depressive Symptoms
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors

This study will evaluate palbociclib in combination with chemotherapy (temozolomide with irinotecan and topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors. The main purpose of this study is to evaluate the safety of palbociclib in combination with chemotherapy in order to estimate the maximum tolerated dose. Pharmacokinetics and efficacy of palbociclib in combination with chemotherapy will be evaluated.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Tanya Watt
128737
All
2 Years to 20 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT03709680
STU-2019-0554
Show full eligibility criteria
Hide eligibility criteria
Inclusion: 1. Histologically confirmed relapsed or refractory solid tumor as follows:
• For dose escalation and dose determination parts: Histologically confirmed relapsed or refractory solid tumor (including CNS tumors but not lymphomas). Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll.
• For dose expansion and tumor specific cohorts: Histologically confirmed relapsed or refractory solid tumor including but not limited to EWS, rhabdoid tumor, rhabdomyosarcoma, neuroblastoma, and medulloblastoma. Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll. EWS is not eligible for TOPO and CTX tumor-specific cohorts. 2. Age ≥2 and <21 years at the time of study entry. 3. Lansky performance status ≥50% for patients ≤16 years of age, or Eastern Cooperative Oncology Group (ECOG) 0, 1 or 2 for patients >16 years of age. 4. Adequate bone marrow function.
• Absolute neutrophil count ≥1000/mm3;
• Platelet count ≥100,000/mm3 (transfusion independent);
• Hemoglobin ≥8.5 g/dL (transfusion allowed). 5. Adequate renal function: Serum creatinine level based on age/gender must within protocol specified limits. 6. Adequate liver function, including:
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 × upper limit of normal (ULN) or ≤5 × ULN for age, if attributable to disease involvement of the liver;
• Total bilirubin ≤1.5 × ULN for age. 7. Measurable disease as defined by RECIST version 1.1 or modified RANO criteria for CNS disease or INRC for neuroblastoma. 8. Recovered to CTCAE Grade ≤1, or to baseline, from any non-hematological acute toxicities of prior surgery, chemotherapy, immunotherapy, radiotherapy, differentiation therapy or biologic therapy, with the exception of alopecia. 9. Serum/urine pregnancy test (for all girls ≥8 years of age) negative at screening and at the baseline visit. 10. Evidence of a personally signed and dated informed consent document indicating that the patient or a legally acceptable representative/parent(s)/legal guardian of minors, has been informed of all pertinent aspects of the study. Minor study patients also must provide age appropriate assent according to the local guidelines, where applicable. 11. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other procedures. Exclusion: 1. For palbociclib with IRN and TMZ combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with an IRN-containing regimen that includes TMZ. Patients who have received the combination of IRN and TMZ and did not progress while on these medications are eligible. For patients enrolling in the palbociclib with TOPO and CTX combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with a TOPO-containing regimen that includes CTX. Patients who have received the combination of TOPO and CTX and did not progress while on these medications are eligible. 2. Prior intolerability to IRN and/or TMZ, for palbociclib with IRN and TMZ combination and prior intolerability to TOPO and/or CTX for palbociclib with TOPO and CTX combination. 3. Use of strong cytochrome P450 (CYP) 3A inhibitors or inducers. Patients who are receiving strong uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) inhibitors within 12 days of Cycle 1 Day 1 (C1D1) are not eligible for the palbociclib with IRN and TMZ combination. Patients who are receiving strong UGT1A1 inhibitors within 12 days of C1D1 are eligible for the palbociclib with TOPO and CTX combination (See Section 5.7.1 for list of products.) 4. Prior growth factors (including filgrastim) within 7 days before study entry or PEG-filgrastim within 14 days before study entry. 5. Radiation therapy within 14 days before study entry. 6. Systemic anti cancer therapy within 2 weeks prior to study entry and 6 weeks for nitrosoureas. 7. Previous high dose chemotherapy requiring stem cell rescue within 90 days or persistent AE >Grade 1. 8. Prior irradiation to >50% of the bone marrow (see Appendix 9). 9. Participation in other studies involving investigational drug(s) within 2 weeks or 5 half lives, whichever is longer, prior to study entry. 10. Major surgery within 4 weeks prior to study entry. Surgical biopsies or central line placement are not considered major surgeries. 11. Known or suspected hypersensitivity to palbociclib, IRN and/or TMZ. 12. Patients with known symptomatic brain tumors or brain metastases and require steroids, unless they have been on a stable or on a decreasing steroid dose for >14 days. 13. Patients with previously diagnosed brain metastases are eligible if they have completed their prior treatment and have recovered from the acute effects of radiation therapy or surgery prior to study entry for these metastases for at least 14 days post radiation and 4 weeks post-surgery and are neurologically stable. 14. Hereditary bone marrow failure disorder. 15. QTc >470 msec. 16. History of clinically significant or uncontrolled cardiac disease, including:
• History of or active congestive heart failure; if patient had congestive heart failure resolve and >1 year from resolution, patient will be considered eligible;
• Clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation or Torsades de Pointes);
• Diagnosed or suspected congenital or acquired prolonged QT syndrome;
• Need for medications known to prolong the QT interval;
• Uncorrected hypomagnesemia or hypokalemia because of potential effects on the QT interval;
• Left ventricular ejection fraction <50% or shortening fraction <28%. 17. Recent or ongoing clinically significant gastrointestinal disorder that may interfere with absorption of orally administered drugs (eg, gastrectomy). 18. Evidence of serious active or uncontrolled bacterial, fungal or viral infection or known history of hepatitis B virus, hepatitis C virus, or human immunodeficiency virus infection or acquired immunodeficiency syndrome-related illness. 19. Other severe acute or chronic medical or laboratory test abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results, and in the judgment of the Investigator, would make the patient inappropriate for entry into this study. 20. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or patients who are Pfizer employees, including their family members, directly involved in the conduct of the study. 21. Fertile male patients and female patients of childbearing potential who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 90 after the last dose of investigational product.
Drug: Palbociclib, Drug: Temozolomide, Drug: Irinotecan, Drug: Topotecan, Drug: Cyclophosphamide
Neuroblastoma, Sarcoma, Solid Tumors, Ewing Sarcoma, Medulloblastoma, Diffuse Intrinsic Pontine Glioma, Rhabdomyosarcoma, Rhabdoid Tumor, 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, 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, Ill - Defined Sites
Solid tumor, EWS, RMS, NBL, Brain, tumor
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

A Study to Evaluate Safety, Efficacy and Pharmacokinetics of Paricalcitol For Treatment of Secondary Hyperparathyroidism (SHPT) in Pediatric Participants With Stage 5 Chronic Kidney Disease (CKD)

The main objective of this study is to evaluate the safety, efficacy and pharmacokinetics of paricalcitol oral solution in pediatric participants of ages 0 to 9 years with SHPT associated with stage 5 CKD receiving Peritoneal Dialysis (PD) or Hemodialysis (HD). The 24-week study is divided into two 12-week dosing periods (Dosing Period 1 followed by Dosing Period 2).
Call 214-648-5005
studyfinder@utsouthwestern.edu
Raymond Quigley
15874
All
up to 9 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04064827
STU-2019-1512
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Participant is currently diagnosed with and/or being treated for secondary hyperparathyroidism (SHPT).
• Participant must be diagnosed with chronic kidney disease (CKD) stage 5 receiving peritoneal dialysis (PD) or hemodialysis (HD) for at least 30 days prior to initial Screening.
• For entry into the Washout Period (for vitamin D receptor activator [VDRA] non-naive participants), the participant must meet the appropriate laboratory criteria based upon the participant's age as described in the protocol.
• For entry into the Dosing Period (for VDRA-naive participants or VDRA non-naive participants who have completed the Washout Period), the participant must meet the appropriate laboratory criteria based upon the participant's age as described in the protocol.
Exclusion Criteria:

• Participant is expected or scheduled to receive a kidney transplant within 6 months of Screening or is a kidney transplant recipient.
• Participant is expected to discontinue peritoneal dialysis (PD) or hemodialysis (HD) within 6 months of the initial Screening visit.
• Participant has had a parathyroidectomy within 12 weeks prior to Screening.
• Participant is taking maintenance calcitonin, bisphosphonates, glucocorticoids (in a dose equivalent to more than > 0.16 mg/kg/day or 5 mg prednisone/day, whichever is lower), 4 weeks prior to Dosing.
• Participant is receiving calcimimetics at the time of Screening or is expected to initiate calcimimetics at any time throughout the study.
• Participant is unable to take oral medications.
Drug: Paricalcitol
Chronic Kidney Disease (CKD), Secondary Hyperparathyroidism (SHPT), Thyroid
Chronic Kidney Disease, Paricalcitol, Hyperparathyroidism, Pediatric Subjects, Peritoneal Dialysis (PD), Hemodialysis (HD), Intact parathyroid hormone (iPTH)
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

The Insulin-Only Bionic Pancreas Pivotal Trial

This multi-center randomized control trial (RCT) will compare efficacy and safety endpoints using the insulin-only configuration of the iLet Bionic Pancreas (BP) System versus Usual Care (UC) during a 13-week study period. Participants may be enrolled initially into a screening protocol and then transfer into the RCT protocol, or they may enter directly into the RCT protocol. The RCT will be followed by an Extension Phase in which the RCT Usual Care (UC) Group will use the insulin-only configuration of the iLet Bionic Pancreas System for 3 months. At the completion of use of the BP system (end of RCT for BP Group and end of Extension Phase for UC Group), participants will enter a 2-4 day Transition Phase and be randomly assigned to either transition back to their usual mode of therapy (MDI or pump therapy) based on therapeutic guidance from the iLet BP System or transition back to their usual mode of therapy based on what their own insulin regimens were prior to enrolling in the RCT.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Philip Raskin
15956
All
6 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04200313
STU-2019-1241
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• 1. Clinical diagnosis of T1D for at least one year and using insulin for at least 1 year 2. Diabetes managed using the same regimen (either pump or MDI, with or without CGM) for ≥ 3 months 3. Age ≥ 6 years old
• Exception: the initial 5-participant test run will be limited to >18 years old 4. Current use of a CGM, or if not a CGM user, at least 3 blood glucose meter tests daily on average over the last 4 weeks (according to judgment of investigator if meter is not available). 5. Willingness not to start any new non-insulin glucose-lowering agent during the course of the trial 6. For participants <18 years old, living with one or more parent/legal guardian knowledgeable about emergency procedures for severe hypoglycemia. 7. Investigator believes that the participant can safely use the iLet and will follow the protocol
• The investigator will take into account the participant's HbA1c level, compliance with current diabetes management, and prior acute diabetic complications. For this reason, there is no upper limit on HbA1c specified for eligibility. 8. If a GLP-1 agonist or pramlintide is being used, participant must be willing to discontinue use while the iLet BP system is being used, including the randomized trial and extension study.
Exclusion Criteria:

• Eligibility may be assessed initially in a separate screening protocol or at a screening visit in the RCT protocol. To be eligible for all phases of the study, a participant must meet all of the following inclusion criteria and none of the exclusion criteria: Inclusion 1. Clinical diagnosis of T1D for at least one year and using insulin for at least 1 year 2. Diabetes managed using the same regimen (either pump or MDI, with or without CGM) for ≥ 3 months 3. Age ≥ 6 years old • Exception: the initial 5-participant test run will be limited to >18 years old 4. Current use of a CGM, or if not a CGM user, at least 3 blood glucose meter tests daily on average over the last 4 weeks (according to judgment of investigator if meter is not available). 5. Willingness not to start any new non-insulin glucose-lowering agent during the course of the trial 6. For participants <18 years old, living with one or more parent/legal guardian knowledgeable about emergency procedures for severe hypoglycemia. 7. Investigator believes that the participant can safely use the iLet and will follow the protocol • The investigator will take into account the participant's HbA1c level, compliance with current diabetes management, and prior acute diabetic complications. For this reason, there is no upper limit on HbA1c specified for eligibility. 8. If a GLP-1 agonist or pramlintide is being used, participant must be willing to discontinue use while the iLet BP system is being used, including the randomized trial and extension study. Exclusion 1. Unable to provide informed consent (e.g. impaired cognition or judgment) 2. Unable to safely comply with study procedures and reporting requirements (e.g. impairment of vision or dexterity that prevents safe operation of the bionic pancreas, impaired memory) 3. Unable to speak and read English • For pediatric participants, both caregivers and participants must be able to speak and read English 4. Plan to change usual diabetes regimen in the next 3 months
• This would include changing from MDI to pump. pump to MDI, change in insulin automation delivery system, starting a CGM if not previously used, changes in drug therapy specifically for glucose control except for changes in one insulin analog to another.
• Changes in insulin dose, carb ratio, sensitivity factor and basal rate profile are allowed. 5. Current use of non-FDA approved closed-loop or hybrid closed-loop insulin delivery system 6. Use of Apidra as the pre-study rapid-acting insulin analog and unwilling to switch to lispro or aspart for the duration of the study 7. Known hemoglobinopathy (sickle cell trait is not an exclusion) 8. Current participation in another diabetes-related clinical trial 9. History of cystic fibrosis, pancreatitis, or other pancreatic disease, including pancreatic tumor or insulinoma, or history of complete pancreatectomy 10. Electrically powered implants (e.g. cochlear implants, neurostimulators) that might be susceptible to RF interference 11. Established history of allergy or severe reaction to adhesive or tape that must be used in the study 12. Current use of SGLT2 inhibitors or a sulfonylurea drug (use more than 3 months prior to enrollment is acceptable) • If using GLP1 agonist, pramlintide, or metformin drugs must be on a stable dose for 3 months prior to enrollment (and as per inclusion criterion #8, must be willing to discontinue use of GLP-1 agonist or pramlintide while using the iLet BP system during the RCT and the extension phase). 13. Pregnant (positive urine hCG), breast feeding, plan to become pregnant in the next 3 months, or sexually active without use of contraception 14. Renal failure on dialysis or with an eGFR <30mL/min • If eGFR is not available within the last 12 months, it must be obtained as part of usual care in order to confirm eligibility. 15. Presence of a medical condition or use of a medication that, in the judgment of the investigator, clinical protocol chair, or medical monitor, could compromise the results of the study or the safety of the participant. Conditions to be considered by the investigator may include the following:
• Alcohol or drug abuse
• Use of prescription drugs that may dull the sensorium, reduce sensitivity to symptoms of hypoglycemia, or hinder decision making during the period of participation in the study
• Coronary artery disease that is not stable with medical management, including unstable angina, angina that prevents moderate exercise (e.g. climbing a flight of stairs) despite medical management, or within the last 12 months before screening a history of myocardial infarction, percutaneous coronary intervention, enzymatic lysis of a presumed coronary occlusion, or coronary artery bypass grafting
• Congestive heart failure with New York Heart Association (NYHA) Functional Classification III or IV
• History of TIA or stroke in the last 12 months
• Untreated or inadequately treated mental illness
• History of eating disorder within the last 2 years, such as anorexia, bulimia, or diabulemia or omission of insulin to manipulate weight
• History of intentional, inappropriate administration of insulin leading to severe hypoglycemia requiring treatment 16. Employed by, or having immediate family members employed by Beta Bionics, or being directly involved in conducting the clinical trial, or having a direct supervisor at place of employment who is also directly involved in conducting the clinical trial (as a study investigator, coordinator, etc.); or having a first-degree relative who is directly involved in conducting the clinical trial
Device: Bionic Pancreas
Diabetes Mellitus, Type 1 Diabetes, Diabetes Mellitus, Type 1, Other Endocrine System, Pancreas
Artificial Pancreas, Closed-loop Insulin Delivery
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Determine the Safety, Tolerability and Pharmacokinetics of single doses of ARCT-810 in Clinically Stable Patients (stable on standard of care treatment, e.g., diet ± ammonia scavengers) with Ornithine Transcarbamylase Deficiency.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Markey McNutt
59152
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT04442347
STU-2020-0554
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Males and females ≥18 years of age with documented diagnosis of late onset OTC deficiency confirmed with genetic testing.
• Subject's Ornithine Transcarbamylase deficiency (OTCD) is stable as evidenced by a) no clinical symptoms of hyperammonemia and b) an ammonia level <100 µmol/L (170 µg/dL) at the Screening evaluation. Subjects must remain free from symptoms of hyperammonemia throughout the screening period.
• If using nitrogen ammonia scavenger therapy, must be on a stable regimen for ≥ 28 days prior to signing informed consent and must remain on the same regimen throughout the screening period
• Must have maintained a stable protein restricted diet +/- amino acid supplementation for at least 28 days prior to signing informed consent and continue to maintain a stable diet for the duration of the study
• No clinically significant abnormal findings on medical history, clinical laboratory test results (other than ammonia) vital sign measurements, 12-lead ECG results, or physical examination
• Males must be surgically sterile or willing to use adequate contraception; females must be post-menopausal, surgically sterile or willing to use adequate contraception
Exclusion Criteria:

• History of clinically significant disease(s) (other than OTCD)
• Abnormal hepatic enzymes, significant renal impairment, clinically significant anemia or uncontrolled diabetes
• Blood Pressure greater than 160/100 mm Hg
• Malignancy within 5 years prior to study
• Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational drug, whichever is longer
• Gene therapy within 1 year prior to screening
• Prior organ transplant
• Positive viral serology test results for HIV type 1 or 2 antibodies, hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody
Biological: ARCT-810, Other: Placebo
Ornithine Transcarbamylase Deficiency
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

A Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders

The purpose of this study is to assess REN001 safety in subjects with fatty acid oxidation disorders.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Markey McNutt
59152
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT03833128
STU-2020-1111
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
Subjects must give written, signed and dated informed consent Confirmed diagnosis of FAOD A diagnostic acylcarnitine profile, in blood or cultured fibroblasts A stable treatment regimen for at least 30 days prior to enrollment
Exclusion Criteria:
Unstable or poorly controlled disease Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer Have been hospitalized within 3 months prior to screening for any major medical event Pregnant or nursing females
Drug: Low Dose REN001, Drug: High Dose REN001
Other Endocrine System, Fatty Acid Oxidation Disorders
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

Extension Study to Evaluate the Safety of Long-Term Use of Relacorilant in Patients With Cushing Syndrome

This is an open-label extension study to evaluate the long-term safety of relacorilant in patients with endogenous Cushing syndrome who successfully completed participation in a Corcept-sponsored study of relacorilant and may benefit from continuing treatment.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Oksana Hamidi
179331
All
Not specified
Phase 2
This study is NOT accepting healthy volunteers
NCT03604198
STU-2020-0455
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Major
Inclusion Criteria:

• Have completed a Corcept-sponsored study of relacorilant in endogenous Cushing syndrome with at least 80% compliance with the dosing schedule.
• According to the Investigator's opinion will benefit from continuing treatment with relacorilant
Exclusion Criteria:

• Major
Exclusion Criteria:

• Premature discontinuation from a relacorilant parent study.
• Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism
• Has poorly controlled hypertension
• Has Stage ≥ 4 renal failure
Drug: relacorilant
Cushing Syndrome, Other Endocrine System
Cushing Syndrome, Cushing Disease, Cushing, Hypercortisolemia, Cushingoid, Type 2 Diabetes, Impaired Glucose Intolerance, Hypertension, Adrenal Corticotrophic Hormone (ACTH), Adrenocortical Carcinoma, Primary Pigmented Nodular Adrenal Disease (PPNAD), Moon Facies, Dorsocervical Fat Pad, Adrenal Adenoma, Adrenal Carcinoma, Adrenal Autonomy, Cortisol
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

A Research Study to Compare Two Types of Insulin, a New Insulin, Insulin Icodec and an Available Insulin, Insulin Glargine, in People With Type 2 Diabetes Who Have Not Used Insulin Before (ONWARDS 1)

This study compares insulin icodec (a new insulin taken once a week) to insulin glargine (an insulin taken once daily which is already available on the market) in people with type 2 diabetes. The study will look at how well insulin icodec taken weekly controls blood sugar compared to insulin glargine taken daily. Participants will either get insulin icodec that participants will have to inject once a week on the same day of the week or insulin glargine that participants will have to inject once a day at the same time every day. Which treatment participants get is decided by chance. The insulin is injected with a needle in a skin fold in the thigh, upper arm or stomach. The study will last for about 1 ½ years. Participants will have 37 clinic visits and 26 phone calls with the study doctor. At 11 clinic visits participant will have blood samples taken. At 8 clinic visits participants cannot eat or drink (except for water) for 8 hours before the visit. Participants will be asked to wear a sensor that measures the blood sugar all the time in 5 periods of about one month during the study (about 5 months in total). Women cannot take part if pregnant, breast-feeding or plan to become pregnant during the study period.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Ildiko Lingvay
55880
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04460885
STU-2020-1108
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female aged above or equal to 18 years at the time of signing informed consent.
• Diagnosed with type 2 diabetes mellitus (T2D) 180 days or more prior to the day of screening.
• HbA1c from 7.0-11.0% (53.0-96.7 mmol/mol) both inclusive at screening confirmed by central laboratory analysis.
• Insulin naïve. However, short term insulin treatment for a maximum of 14 days prior to the day of screening is allowed, as is prior insulin treatment for gestational diabetes.
• Stable daily dose(s) 90 days or more prior to the day of screening of any of the following anti-diabetic drug(s) or combination regimen(s): a. Any metformin formulations at least or greater than 1500 mg or maximum tolerated or effective dose. b. Any metformin combination formulations equal to or above 1500 mg or maximum tolerated or effective dose. c. Any of the following oral anti-diabetic drug classes including combinations ((equal to or above half of the maximum approved dose according to local label or maximum tolerated or effective dose): Sulfonylureas, Meglitinides (glinides), dipeptidyl peptidase-4 (DPP-4) inhibitors, Sodium-glucose co-transporter-2 (SGLT2) inhibitors, Thiazolidinediones, Alpha-glucosidase inhibitors, Oral combination products (for the allowed individual oral anti-diabetic drugs), Oral or injectable glucagon-like peptide 1 (GLP-1) receptor agonists
• Body mass index (BMI) equal to or below 40.0 kg/m^2.
Exclusion Criteria:

• Any episodes (as declared by the subject or in the medical records) of diabetic ketoacidosis within 90 days prior to the day of screening.
• Myocardial infarction, stroke, hospitalisation for unstable angina pectoris or transient ischaemic attack within 180 days prior to the day of screening.
• Chronic heart failure classified as being in New York Heart Association Class IV at screening.
• Anticipated initiation or change in concomitant medications (for more than 14 consecutive days) known to affect weight or glucose metabolism (e.g. treatment with orlistat, thyroid hormones, or corticosteroids).
• Uncontrolled and potentially unstable diabetic retinopathy or maculopathy. Verified by a fundus examination performed within the past 90 days prior to screening or in the period between screening and randomisation. Pharmacological pupil-dilation is a requirement unless using a digital fundus photography camera specified for non-dilated examination
Drug: Insulin icodec, Drug: Insulin glargine
Diabetes Mellitus, Type 2, Other Endocrine System, Pancreas
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

Celliant Socks to Increase Tissue Oxygenation and Complete Wound Closure in Diabetic Foot Wounds

This study is a prospective, multicenter, double-blind, 1:1 randomized clinical trial. The purpose of this study is to demonstrate that the use of Celliant Socks increases tissue oxygenation (via oxygen saturation, StO2) and incidence of wound closure in subjects with diabetic foot ulcers. This study will use hyperspectral imaging and wound assessment to measure these outcomes. The study will enroll 254 evaluable subjects total, 127 per arm to meet the Primary Endpoint. Enrollment may continue up to twenty-five hundred (2500) evaluable subjects total to meet the Key Secondary Endpoint of complete wound closure.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Lawrence Lavery
116716
All
22 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04709419
STU-2020-1386
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Diagnosis of diabetes mellitus
• Subject is willing and able to wear a sports-style tube sock at least 22 hours a day.
• Ankle Brachial Index (ABI) ≥0.5 (bedside ABI is acceptable for screening purposes as the formal imaging ABI may not be resulted prior to surgery) or toe pressure of ≥30mmHg
• One or more diabetic foot ulcers (only one will be treated) that are located in the ankle area or below that has persisted a minimum of 30 days prior to the Screening visit
• Diabetic Foot Ulcers ≥1cm2 and ≤16cm2
• Ulcer grade I or II, Stage A, I or II Stage B, according to University of Texas Wound Classification System
• 22 years of age or older
Exclusion Criteria:

• Has clinically significant renal disease to require hemo or peritoneal dialysis
• Subject has untreated osteomyelitis
• Ulcers within 5cm of target ulcer or connected by fistulas
• Ulcer has decreased by 30% or more at the end of the run-in period
• Subject has untreated cellulitis
• Subject has untreated charcot
• Major immunodeficiency including HIV
• Is pregnant or plans to become pregnant
• Is nursing or actively lactating
• Developmental disability/significant psychological disorder that in the opinion of the investigator could impair the subject's ability to provide informed consent, participate in the study protocol or record study measures, including untreated schizophrenia, bipolar disorder and psychiatric hospitalization within the last 2 years.
• Active alcohol or substance abuse in the opinion of the investigator that could impair the subject's ability to provide informed consent, participate in the study protocol or record study materials
Device: Celliant Diabetic Medical Socks, Device: Control (placebo) Medical Socks
Diabetic Foot Ulcer
Parkland Health & Hospital System
  Email this study information to me
  Contact the study team
  See more information

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of treatment with crinecerfont. Duration of participation is approximately 20 months.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Oksana Hamidi
179331
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04490915
STU-2020-0941
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
1. Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit. 2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH. 3. Be on a stable regimen of steroidal treatment for CAH. 4. Patients of childbearing potential must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) or other highly effective contraception during the study.
Exclusion Criteria:
1. Have a diagnosis of any of the other known forms of classic CAH. 2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy. 3. Have a clinically significant unstable medical condition or chronic disease other than CAH. 4. Have a history of cancer unless considered cured. 5. Are pregnant. 6. Have a known history of clinically significant arrhythmia or abnormalities on ECG. 7. Have a known hypersensitivity to any corticotropin releasing hormone antagonists. 8. Have received any other investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study. 9. Have current substance dependence, or current substance (drug) or alcohol abuse. 10. Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.
Drug: Crinecerfont, Drug: Placebo
Congenital Adrenal Hyperplasia
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency (foresiGHt)

A 38 week dosing trial of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus placebo-control. A daily somatropin product arm is also included to assist clinical judgement on the trial results. Approximately 240 adults (males and females) with growth hormone deficiency will be included. Randomization will occur in a 1:1:1 ratio (lonapegsomatropin : placebo : daily somatropin product). This is a global trial that will be conducted in, but not limited to, the United States, Europe, and Asia.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Oksana Hamidi
179331
All
23 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04615273
STU-2020-1037
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria 1. Age between 23 and 75 years, inclusive, at screening. 2. AGHD Diagnosis Criteria For adult-onset AGHD: documented history of structural hypothalamic-pituitary disease, hypothalamic-pituitary surgery, cranial irradiation, 1-4 non-GH pituitary hormone deficiencies, a proven genetic cause of GHD, or traumatic brain injury (TBI). A. For all countries except Japan: Subjects must satisfy at least one of the following criteria: 1. Insulin tolerance test: peak GH ≤5 ng/mL 2. Glucagon stimulation test according to body mass index (BMI)
• i. BMI ≤30 kg/m2: peak GH ≤3 ng/mL
• ii. BMI >30 kg/m2: peak GH ≤1 ng/mL 3. Three or four pituitary axis deficiencies (i.e., adrenal, thyroid, gonadal, and/or vasopressin; not including GH) with IGF-1 SDS ≤ -2.0 at screening 4. Macimorelin test: peak GH ≤2.8 ng/mL 5. Growth hormone releasing hormone (GHRH) + arginine test according to BMI:
• i. BMI <25 kg/m2, peak GH <11 ng/mL
• ii. BMI ≥25-≤30 kg/m2, peak GH <8 ng/mL
• iii. BMI >30 kg/m2, peak GH <4 ng/mL B. For Japan only: Subjects with adult onset AGHD and deficiency of one or more other pituitary hormones need to satisfy at least one of the following criteria, while subjects with isolated GHD and no evidence of intracranial structure disorder (structural hypothalamic-pituitary disease) or with adult onset AGHD without deficiency of other pituitary hormones need to satisfy at least 2 of the following criteria: 1. Insulin tolerance test: peak GH ≤1.8 ng/mL 2. Glucagon test: peak GH ≤1.8 ng/mL 3. Growth Hormone Releasing Peptide-2 (GHRP-2) tolerance test: peak GH ≤9 ng/mL 3. IGF-1 SDS ≤ -1.0 at screening as measured by central laboratory. 4. hGH treatment naïve or no exposure to hGH therapy or GH secretagogue for at least 12 months prior to screening. 5. For subjects on hormone replacement therapies for any hormone deficiencies other than GH (e.g., adrenal, thyroid, estrogen, testosterone) must be on adequate and stable doses for ≥6 weeks prior to and throughout screening. 6. For subjects not on glucocorticoid replacement therapy, documentation of adequate adrenal function at screening defined. 7. For males not on testosterone replacement therapy: morning (6:00
•10:00AM) total testosterone within normal limits for age. 8. On a stable diet and exercise regime at screening with no intention to modify diet or exercise pattern during the trial, i.e., no weight reduction program intended during the trial or within the last 90 days prior to or through screening. 9. No plans to undergo bariatric surgery during the trial. 10. Normal fundoscopy at screening (without signs/symptoms of intracranial hypertension or diabetic retinopathy above stage 2 / moderate). For subjects with a diagnosis of diabetes mellitus at screening, this must be documented with a fundus photograph. 11. Able and willing to provide a written informed consent and authorization for protected health information (PHI) disclosure in accordance with Good Clinical Practice (GCP). Exclusion Criteria 1. Known Prader-Willi Syndrome and/or other genetic diseases that may have an impact on an endpoint. 2. Diabetes mellitus at screening if any of the following criteria are met: 1. Poorly controlled diabetes, defined as HbA1c >7.5% at screening. 2. Diabetes mellitus (defined as HbA1c ≥6.5% and/or fasting plasma glucose ≥126 mg/dL and/or plasma glucose ≥200 mg/dL two hours after oral glucose tolerance test) diagnosed <26 weeks prior to screening 3. Change in diabetes regimen (includes dose adjustment) within <90 days prior and throughout screening 4. Use of any diabetes drugs other than metformin and/or DPP-4 inhibitors for a cumulative duration of greater than 4 weeks within 12 months prior to screening 5. Diabetes-related complications at screening (i.e., nephropathy as judged by the investigator, neuropathy requiring pharmacological treatment, retinopathy stage 2 / moderate and above within 90 days prior to screening or during screening) 3. Active malignant disease or history of malignancy. Exceptions to this exclusion criterion: 1. Resection of in situ carcinoma of the cervix uteri 2. Complete eradication of squamous cell or basal cell carcinoma of the skin 3. Subjects with GHD attributed to treatment of intracranial malignant tumors or leukemia, provided that a recurrence-free survival period of at least 5 years prior to screening is documented in the subject's file based on a Magnetic Resonance Imaging (MRI) result 4. Evidence of growth of pituitary adenoma or other benign intracranial tumor within the last 12 months before screening. 5. Subjects with acromegaly without remission / with documented remission less than 24 months prior to screening. 6. Subjects with Cushing's disease without remission / with documented remission less than 24 months prior to screening. 7. Subjects with prior cranial irradiation or hypothalamic-pituitary surgery: the procedure took place less than 12 months prior to screening. 8. eGFR <60 mL/min/1.73m2 determined based on Modification of Diet in Renal Disease (MDRD) equation. 9. Hepatic transaminases (i.e., AST or ALT) >3 times the upper limit of normal. 10. Heart failure NYHA class 3 or greater (NYHA 1994). 11. QTcF ≥ 451 milliseconds on 12-lead ECG at screening. 12. Poorly controlled hypertension. 13. Cerebrovascular accident within 5 years prior to screening. 14. Anabolic steroids (other than gonadal steroid replacement therapy) or oral/intravenous/intramuscular corticosteroids within 90 days prior to or throughout screening. 15. Currently using or have used within 26 weeks prior to screening any weight-loss or appetite-suppressive medications. 16. Known history of hypersensitivity and/or idiosyncrasy to any of the test compounds (somatropin) or excipients employed in this trial. 17. Known history of neutralizing anti-hGH antibodies. 18. Inability to undergo scanning by DXA or a non-interpretable DXA scan at screening. 19. Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using adequate contraceptive methods 20. Male subjects must use a condom, or his female partner of childbearing potential must use an effective form of contraception as described above, from the beginning of screening to the last trial visit. 21. Known substance abuse or known (or previous) eating disorders, including anorexia nervosa, bulimia and severe gastrointestinal disease affecting normal eating (as judged by the investigator). 22. Any disease or condition that, in the judgement of the investigator, may make the subject unlikely to comply with the requirements of the trial or any condition that presents undue risk from the investigational product or procedures. 23. Participation in another interventional clinical trial involving an investigational compound within 26 weeks prior to screening or in parallel to this trial.
Drug: Lonapegsomatropin, Other: Placebo, Drug: Somatropin
Endocrine System Diseases, Hormone Deficiency, Growth Hormone Deficiency
Human Growth Hormone, hGH, rhGH, GHD, Adult Growth Hormone Deficiency, Long Acting Growth Hormone, Lonapegsomatropin, Prodrug, Growth Failure, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon hGH
UT Southwestern
  Email this study information to me
  Contact the study team
  See more information

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
Show full eligibility criteria
Hide eligibility criteria
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
Children’s Health
  Email this study information to me
  Contact the study team
  See more information

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.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Tanya Watt
128737
All
2 Years to 21 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT03245151
STU 072017-006
Show full eligibility criteria
Hide eligibility criteria
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
Children’s Health
  Email this study information to me
  Contact the study team
  See more information