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52 Study Matches

Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia

The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for pediatric acquired severe aplastic anemia.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Kathryn Dickerson
156007
All
up to 25 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02845596
STU 052018-057
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Inclusion Criteria:
1. Confirmed diagnosis of idiopathic SAA, defined as:
• Bone marrow cellularity <25%, or <30% hematopoietic cells.
• Two out of three of the following (in peripheral blood): neutrophils <0.5 x109/L, platelets <20 x109/L, reticulocyte count <60 x109/L with hemoglobin <8g/dL. 2. Age ≤25 years old. 3. No suitable fully matched related donor available (minimum 6/6 match for Human Leukocyte antigen (HLA) -A and B at intermediate or high resolution and DRB1 at high resolution using DNA based typing). 4. At least two unrelated donors noted on National Marrow Donor Program (NMDP) search who are well matched (9/10 or 10/10 for HLA-A, B, C, DRB1, and DQB1 using high resolution). 5. Signed informed consent for the randomized trial by patient and/or legal guardian. 6. Adequate organ function defined as in the judgment of the investigator, there is not irreversible organ damage that would preclude the patient from meeting the organ function inclusion criteria for HSCT listed in section 2.3.4 by the intended time of HSCT (6-8 weeks after randomization) or preclude patients from receiving horse ATG.
Exclusion Criteria:
1. Inherited bone marrow failure syndromes (IBMFS). The diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow. Telomere length testing should be sent on all patients to exclude Dyskeratosis congenita, but if results are delayed or unavailable and there are no clinical manifestations of DC, patients may enroll. If patients have clinical characteristics suspicious for Shwachman Diamond syndrome, this syndrome must be excluded by pancreatic isoamylase testing or gene mutation analysis. Note: pancreatic isoamylase testing is not accurate in children less than 3 years. 2. Clonal cytogenetic abnormalities or fluorescence In Situ Hybridization (FISH) pattern consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (see section 4.2.3.1 for details of the required MDS FISH panel). 3. Known severe allergy to horse ATG. 4. Prior allogeneic stem cell transplant. 5. Prior solid organ transplant. 6. Infection with human immunodeficiency virus (HIV). 7. Active Hepatitis B or C. This should be excluded in patients where there is clinical suspicion of hepatitis (e.g. elevated LFTs). 8. Female patients who are pregnant or breast-feeding. 9. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ.
Drug: cyclosporine, Procedure: Matched Unrelated Donor Hematopoietic Stem Cell Transplant, Drug: horse anti-thymocyte globulin (ATG), Drug: rabbit anti-thymocyte globulin (ATG), Drug: methotrexate, Drug: fludarabine, Drug: cyclophosphamide, Radiation: low-dose total body irradiation (TBI), Procedure: Immunosuppressive Therapy (IST)
Severe Aplastic Anemia
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Entinostat and Pembrolizumab in Treating Patients With Myelodysplastic Syndrome After DNMTi Therapy Failure

This phase Ib trial studies the side effects and best dose of entinostat when given together with pembrolizumab in treating patients with myelodysplastic syndrome after deoxyribonucleic acid (DNA) methyltransferase inhibitor (DNMTi) therapy failure. Entinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving entinostat together with pembrolizumab may work better in treating patients with myelodysplastic syndrome after DNMTi therapy failure.
Call 1-888-980-6050
canceranswerline@utsouthwestern.edu
Prapti Patel
103509
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT02936752
STU-2018-0140
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Inclusion Criteria:

• Pathologically confirmed myelodysplastic syndrome (MDS) diagnosis (regardless of initial International Prognostic Scoring System [IPSS] risk category) or oligoblastic acute myeloid leukemia (AML) with 21-30% bone marrow (BM) blasts in whom DNMTi have failed; patients who have developed AML after DNMTi therapy can be enrolled as long as they have initiated DNMTi therapy while they were in the MDS or oligoblastic AML (20-30% BM blasts) phase and the study chair agrees; failure of DNMTis is defined as: failure to achieve a complete response (CR), partial response (PR) or hematologic improvement (HI) after at least 4 cycles of DNMTi or progressed after such therapy
• Eastern Cooperative Oncology Group (ECOG) performance status =< 2
• Calculated creatinine clearance by Modification of Diet in Renal Disease (MDRD) (CrCl) >= 60 ml/min/1.73 squared meter
• Total bilirubin =< 2.0 mg/dL unless due to Gilbert's syndrome, hemolysis, or ineffective hematopoiesis
• Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x upper limit of normal (ULN)
• Females of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to start of first cycle of therapy
• Patients must have no clinical evidence of central nervous system (CNS) or pulmonary leukostasis, disseminated intravascular coagulation, or CNS leukemia
• Patients must have no serious or uncontrolled medical conditions
• The effects of entinostat and MK-3475 (pembrolizumab) on the developing human fetus are unknown; for this reason, women of child-bearing potential and men who are sexually active with women of childbearing 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 she or her partner is participating in this study, she should inform her treating physician immediately; men who are sexually active with women of childbearing potential, treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of entinostat and MK3475 (pembrolizumab) administration
• Ability to understand and the willingness to sign a written informed consent document
• Patients, who relapsed 6 months after bone marrow transplant and have no evidence of active graft versus host disease and are off systemic immunosuppressant medications for at least 2 months and have received hypomethylating agents (HMA) therapy before or after transplant and meet other eligibility criteria of progression after at least 4 months of DNMTi therapy, are eligible to be enrolled in this clinical trial
Exclusion Criteria:

• Any patients eligible for allogeneic stem cell transplantation (allo-SCT) and willing to undergo allo-SCT as determined at time of screening for trial; patients who are ineligible or not interested in undergoing allo-SCT will be eligible for the trial
• Any serious medical condition, uncontrolled intercurrent illness (e.g., active infection, symptomatic congestive heart failure [CHF], unstable angina, cardiac arrhythmias, laboratory abnormalities, or psychiatric illness and/or biopsychosocial conditions that may limit compliance
• Patients with known active cancers who are on therapy for those cancers at time of screening
• Patients who are human immunodeficiency virus (HIV) positive may participate IF they meet the following eligibility requirements:
• They must be stable on their anti-retroviral regimen, and they must be healthy from an HIV perspective
• They must have a CD4 count of greater than 250 cells/mcL
• They must not be receiving prophylactic therapy for an opportunistic infection
• Patients with a known positive test for hepatitis B virus surface antigen (HBV sAg) or hepatitis C virus ribonucleic acid (HCV antibody) indicating acute or chronic infection might be enrolled if the viral load by polymerase chain reaction (PCR) is undetectable with/without active treatment
• Pregnant or breast feeding females (lactating females must agree not to breast feed while taking the study drugs)
• Use of any other experimental drug or therapy within 21 days of baseline
•patients who have had chemotherapy or radiotherapy within 4 weeks of entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
• Known hypersensitivity to MK-3475 (pembrolizumab) or history of allergic reactions to compounds of similar chemical or biologic composition to anti-PD1 or PD-L1 antibodies or entinostat
• Prior treatment with any anti-PD-1 blocking therapies or histone deacetylase inhibitors (HDACi), or anti-CTLA-4 antibody, CD137 agonist or other immune activating therapy such as anti-CD 40 antibody within the last 3 months of enrollment in the study
• Any history of active or severe autoimmune disease: inflammatory bowel disease, including ulcerative colitis and Crohn's disease, rheumatoid arthritis, systemic progressive scleroderma, systemic lupus erythematosus, autoimmune vasculitis (e.g., Wegener's granulomatosis), CNS or motor neuropathy considered of autoimmune origin (e.g. Guillain-Barre syndrome, myasthenia gravis, multiple sclerosis); patients with hypothyroidism with stable hormone replacement therapy dosing are allowed on study
• Has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis
Drug: Entinostat, Biological: Pembrolizumab
Myelodysplastic Syndrome, Blasts 21-30 Percent of Bone Marrow Nucleated Cells
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Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Markey McNutt
59152
All
2 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03921541
STU-2019-0572
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Inclusion Criteria:
1. The subject and/or parent/guardian provides written informed consent/assent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol 2. A current diagnosis of ARG1-D. For entry into this study, subjects must also fulfill the following plasma arginine criterion: 1. The average of all measured values of plasma arginine during the screening period prior to the randomization visit (Visit 1, Study Day 0) is ≥ 250 µmol/L 2. If a subject is re-screened, the only values that are considered for eligibility assessment are those in the current screening period 3. Subjects must be ≥ 2 years of age on the date of informed consent/assent 4. The subject must be assessable for clinically meaningful within-subject change (clinical response) on at least one component of one assessment included in the key secondary endpoint. To be considered assessable, the subject must be able to complete the assessment, and must have a baseline deficit in the specific component as defined in the protocol 5. Have received documented confirmation from the investigator and/or dietician that the subject is capable of maintaining their diet in accordance with dietary information presented in the protocol, i.e., is capable of maintaining their current level of protein consumption including natural protein and essential amino acid supplementation 6. Subjects receiving ammonia scavenger therapy, anti-epileptic drugs, and/or medications for spasticity (e.g., baclofen) must be on a stable dose of the medication for at least 4 weeks prior to randomization and be willing to remain on a stable dose during the double-blind portion and blinded follow-up portions of the study 7. Female and male subjects may participate. Female subjects of child-bearing potential must have a negative serum pregnancy test during the screening period before receiving the first dose of study treatment, and a negative urine pregnancy test on the day of the first dose, prior to the first dose. If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, postmenopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation; progesterone-only hormonal contraception associated with inhibition of ovulation; intrauterine device (IUD); or intrauterine hormone-releasing system (IUS)
Exclusion Criteria:
1. Hyperammonemic episode (defined as an event in which a subject has an ammonia level > 100 umol/L with one or more symptoms related to hyperammonemia requiring hospitalization or emergency room management) within the 6 weeks before the first dose of study drug in administered 2. Active infection requiring anti-infective therapy within 3 weeks prior to first dose 3. Known active infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C 4. Extreme mobility deficit, defined as either the inability to be assessed on the GFAQ or a score of 1 on the GFAQ 5. Other medical conditions or comorbidities that, in the opinion of the investigator would interfere with study compliance or data interpretation (e.g., severe intellectual disability precluding required study assessments) 6. Has participated in a previous interventional study with pegzilarginase 7. Has a history of hypersensitivity to polyethylene glycol (PEG), that in the judgment of the investigator, puts the subject at unacceptable risk for adverse events 8. Subject is being treated with botulinum-toxin-containing regimens or plans to initiate such regimens during the double-blind or blinded follow-up portions of the study or received surgical or botulinum-toxin treatment within last 6 months for spasticity related complications 9. Is currently participating in another therapeutic clinical trial or has received any investigational agent within 30 days (or 5 half-lives whichever is longer) prior to the first dose of study treatment on this study 10. Previous liver or hematopoietic transplant procedure.
Drug: Pegzilarginase, Drug: Placebo, Drug: Pegzilarginase
Arginase I Deficiency, Hyperargininemia
ARG1-D
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A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia. All patients will be treated with eltrombopag for the 26-week Treatment Period, followed by a 52-week Follow-Up Period. Patients who have been previously untreated with immunosuppressive therapy will be treated according to the standard of care, hATG/cyclosporine, in addition to eltrombopag. Patients with relapsed/refractory SAA or recurrent AA will be enrolled into one of two treatment options: hATG/cyclosporine plus eltrombopag or cyclosporine plus eltrombopag, depending on prior treatment with immunosuppressive therapy. After initiating treatment with eltrombopag, patients will have their dose assessed and modified as tolerated, until the targeted platelet count or maximum dose is achieved. Pharmacokinetic assessments will be performed at time points intended to capture steady state PK of the starting dose and highest dose achieved. Upon completion of the Treatment and Follow-Up Periods, all patients will be offered the opportunity to enroll in an additional 3 year Long Term Follow-Up Period.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Kathryn Dickerson
156007
All
1 Year to 18 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03025698
STU 052017-089
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For Cohort A: 1. Prior history of diagnosis of SAA 2. Diagnosis of relapsed/refractory SAA or recurrent AA following IST for SAA at the time of enrollment. Patients with recurrent AA (e.g., losing their response) are exempt from meeting the diagnostic criteria for relapsed SAA at the time of enrollment, but must have been previously diagnosed with SAA. 3. Agree to concurrent eltrombopag treatment with appropriate, investigator-selected IST with either hATG + CsA or CsA. For Cohort B: 4. Diagnosis of SAA at the time of enrollment 5. Patients must not have been previously treated for SAA 6. Patients must agree to treatment with hATG + CsA concurrent with eltrombopag. For all patients, regardless of cohort: 7. Age 1 to <18 years 8. Where appropriate, assessments to rule out congenital/inherited bone marrow failure syndromes and other causes of immune-mediated pancytopenia, which may be treated with transplant, must be completed prior to enrollment. 9. Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient. (Candidacy for HSCT will be determined as per local practice.) 10. Bone marrow aspirate and biopsy at any time during the 4 weeks prior to first dose of eltrombopag 11. Normal karyotype with FISH for chromosomes 7 and 8 12. Performance status score: Karnofsky ≥50 or Lansky ≥50 (depending on age) 13. Serum creatinine ≤2.5 × ULN 14. Total bilirubin ≤1.5 × ULN 15. Written informed consent signed by a parent or legal guardian prior to initiation of any study specific procedure.
Exclusion Criteria:
1. Prior and/or active medical history of:
• Fanconi anemia (via chromosomal breakage test or growth arrest by flow cytometry)
• Other known underlying congenital/inherited marrow failure syndromes
• Symptomatic Paroxysmal Nocturnal Hemoglobinuria (PNH) and/or PNH clones >50% of PMN or RBC at time of enrollment
• Any cytogenetic abnormalities, including but not limited to chromosome 7 or myelodysplasia, in bone marrow within 4 weeks of study enrollment
• Myelodysplastic syndrome (MDS)
• Other known or suspected underlying primary immunodeficiency
• Any malignancy 2. Active infection not responding to appropriate therapy 3. Prior eltrombopag or other thrombopoietin receptor (TPO-R) agonist treatment for at least 2 months and a lack of response. 4. Any out of range lab values Creatinine >2.5 × upper limit of normal (ULN), Total bilirubin >1.5 × ULN Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5 × ULN
Drug: Eltrombopag, Drug: hATG, Drug: CsA
Aplastic Anemia
Eltrombopag, hATG, CsA, previously untreated or relapsed severe aplastic anemia, recurrent aplastic anemia, Severe aplastic anemia, Pharmacokinetics, Immunosuppressive therapy, ETB115
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A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency (AEB1102)

The purpose of this study is to investigate the long-term safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous AEB1102 in patients who complete Study CAEB1102-101A.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Markey McNutt
59152
All
2 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03378531
STU-2018-0103
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Inclusion Criteria:
1. Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing 2. Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient to continue dosing with AEB1102 3. If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment 4. If sexually active (male or female), must be surgically sterile, post-menopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration 5. Patient or legal guardian is able and willing to provide written informed consent and where required assent, and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification), prior to any screening procedures
Exclusion Criteria:
1. Clinically significant concurrent disease, serious intercurrent illness, or other extenuating circumstances
Drug: AEB1102
Arginase I Deficiency, Hyperargininemia
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A Study of ARQ 531 in Patients With Selected Hematologic Malignancies

This is an open-label, multi-center Phase 1/2 study of ARQ 531 in patients with selected hematologic malignancies.
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canceranswerline@utsouthwestern.edu
Farrukh Awan
180091
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT03162536
STU-2019-0811
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Inclusion Criteria Each prospective subject must meet ALL of the following inclusion criteria in order to be eligible for this study: 1. Signed written informed consent granted prior to initiation of any study-specific procedures. 2. 18 years of age and older. 3. For the dose escalation cohorts, relapsed or refractory subjects with a diagnosis of B-cell NHL, CLL/SLL and WM who have received at least two prior systemic therapies . Subjects must have failed or are intolerant to standard therapies and cannot be a candidate for standard salvage regimens. Subjects with low grade lymphoma must be progressing and requiring treatment.. 4. For the expansion cohorts, the following criteria must be met:
• Cohort A: Relapsed/Refractory (R/R) CLL/SLL subjects with at least 2 prior systemic therapies and previously treated with a covalent BTKi who must have a documented BTK mutation on C481 residue
• Cohort B: R/R CLL/SLL subjects who have failed or were intolerant to a BTKi with documentation of the absence of BTK mutation on C481 residue. In this study, intolerance to standard therapy is defined as having experienced a grade 3 or higher adverse event that was caused by the standard therapy and resulted in treatment discontinuation.
• Cohort C: Richter's transformation subjects who have failed at least one prior therapy
• Cohort D: Follicular Lymphoma (FL) subjects who have failed at least 2 prior systemic therapies and are histology grade 1, 2, or 3A
• Cohort E: Mantle Cell Lymphoma (MCL) subjects who have failed at least 2 prior systemic therapies
• Cohort F: Marginal Zone Lymphoma (MZL) subjects who have failed at least 2 prior systemic therapies
• Cohort G: High-grade B-cell lymphoma subjects who have failed at least 2 prior systemic therapies and have known MYC and BCL2 and/or BCL6 translocations
• Cohort H: Waldenström macroglobulinemia (WM) subjects who have failed at least 2 prior systemic therapies 5. Disease status requirement: 1. For CLL subjects, symptomatic disease that mandates treatment (Hallek et al. 2018). 2. For B-cell NHL subjects, measurable disease by imaging scan. 3. For WM, serum immunoglobulin M (IgM) with a minimum IgM level of ≥ 2 times the upper limit of normal (ULN). 6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2. 7. Good organ function 1. Creatinine clearance of ≥ 60 mL/min as estimated by the Cockcroft-Gault equation or by 24-hour urine collection. 2. Total bilirubin ≤ 1.5 x institutional ULN (total bilirubin of ≤ 3 x institutional ULN in subjects with documented Gilbert's syndrome). 3. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × institutional ULN. 4. Platelet count ≥ 50,000/µL 5. Absolute neutrophil count (ANC) ≥ 1000/µL. 6. Hemoglobin (Hgb) ≥ 8.0 g/dL, stable for ≥ 1 week. 8. For men and women of child-bearing potential, willing to use adequate contraception (e.g., latex condom, cervical cap, diaphragm, abstinence, etc.) for the entire duration of the study. 9. Female subjects of child-bearing potential must have a negative serum pregnancy test within 14 days of the first day of drug dosing. 10. Ability to swallow oral medications without difficulty. Exclusion Criteria Potential subjects who meet ANY of the following exclusion criteria are not eligible for enrollment into this study: 11. Had immunotherapy, radiotherapy, radioimmunotherapy, biological therapy, chemotherapy, or treatment with an investigational product within 5 half-lives or four weeks (whichever is shorter) prior to treatment initiation, or oral therapy within 5 half-lives or one week (whichever is shorter) prior to treatment initiation. 12. Transformation of FL to a more aggressive subtype of lymphoma or grade 3b FL 13. Subjects currently being treated with the following drugs: 1. CYP 2C9 substrates with a narrow therapeutic index (such as warfarin, phenytoin) 2. CYP 2C8 substrates with a narrow therapeutic index (such as paclitaxel) 3. CYP 2C19 substrates with a narrow therapeutic index (such as S-mephenytoin) 4. CYP 2D6 substrates with a narrow therapeutic index (such as thioridazine, pimozide) 5. P-gp substrates with a narrow therapeutic index (such as digoxin) Note: A washout period of at least 5 times the half-life after the last dose of any of the above treatments is required for a subject to be eligible for study enrollment. 14. Prior allogeneic bone marrow transplant. 15. Active central nervous system (CNS) involvement. 16. Pregnant or breast-feeding women. 17. Has significant, ongoing co-morbid conditions which would preclude safe delivery of the study drug. 18. Uncontrolled illness including but not limited to ongoing or active infection, symptomatic congestive heart failure (New York Heart Association [NYHA] Class III or IV heart failure), unstable angina pectoris, cardiac arrhythmia, cardiac infarction in the past six months, and psychiatric illness that would limit compliance with study requirements. 19. QTc prolongation (defined as a QTc > 450 msecs) or other significant electrocardiogram (ECG) abnormalities including 2nd degree atrioventricular (AV) block type II, 3rd degree AV block, or bradycardia (ventricular rate less than 50 beats/min). If the screening ECG has a QTc > 450 msecs, the ECG can be submitted for a centralized, cardiologic evaluation. 20. Active human immunodeficiency virus (HIV) infection, Hepatitis B, or Hepatitis C infection. 21. Other medical or psychiatric illness or organ dysfunction which, in the opinion of the Investigator, would either compromise the subject's safety or interfere with the evaluation of the safety of the study agent. 22. History of prior cancer within < 1 year, except for basal cell or squamous cell carcinoma of the skin, cervical cancer in situ or other in situ carcinomas.
Drug: ARQ 531, Drug: ARQ 531
Lymphoma, B-cell, Waldenström Macroglobulinemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Follicular Lymphoma, Marginal Zone Lymphoma, Mantle Cell Lymphoma, Diffuse Large B Cell Lymphoma, Richter's Transformation
ARQ 531, Hematologic Malignancies, SLL, CLL, B-cell NHL, WM, BTK (Bruton's tyrosine kinase), cancer, refractory, relapsed, Acalabrutinib, ArQule, BGB-3111, Blood Protein Disorders, Follicular Lymphoma, BTK Intolerant, C481, C481S, C481S Mutation, Cardiovascular Diseases, Chronic Lymphocytic Leukemia, DLBCL (Diffuse Large B-cell lymphoma), GS-4059, Hemorrhagic Disorders, Hemostatic Disorders, Ibrutinib, Immune System Diseases, Immunoproliferative Disorders, Leukemia, Leukemia, B-Cell, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Lymphoid, Lymphatic Diseases, Lymphoma, Lymphoma, B-Cell, Lymphoma, B-Cell, Marginal Zone, Lymphoma, Mantle-Cell, Lymphoma, Non-Hodgkin, Lymphoproliferative Disorders, Mantle Cell Lymphoma, Marginal zone lymphoma, Neoplasms, Neoplasms by Histologic Type, Neoplasms, Plasma Cell, NHL (Non-Hodgkin's Lymphoma), ONO-4059, Paraproteinemias, Small Lymphocytic Lymphoma, Tirabrutinib, Vascular Diseases, Waldenstrom Macroglobulinemia, Zanubrutinib
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A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 Alterations

Phase 1 will evaluate the safety and tolerability at different dose levels of repotrectinib in pediatric and young adult subjects with advanced or metastatic malignancies harboring anaplastic lymphoma kinase (ALK), receptor tyrosine kinase encoded by the gene ROS1 (ROS1), or neurotrophic receptor kinase genes encoding TRK kinase family (NTRK1-3) alterations to estimate the Maximum Tolerated Dose (MTD) or Maximum Administered Dose (MAD) and select the Pediatric Recommended Phase 2 Dose (RP2D).
Call 1-888-980-6050
canceranswerline@utsouthwestern.edu
Theodore Laetsch
148176
All
4 Years to 12 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04094610
STU-2019-1268
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Key
Inclusion Criteria:
1. Documented genetic ALK, ROS1, or NTRK1-3 alteration (point mutation, fusion, amplification) as identified by local testing in a Clinical Laboratory Improvement Amendments (CLIA) laboratory in the US or equivalently accredited diagnostic lab outside the United States (US) is required. 2. Age 4 to <12 years. 3. Capability to swallow capsules intact (without chewing, crushing, or opening). 4. Prior cytotoxic chemotherapy is allowed. 5. Prior immunotherapy is allowed. 6. Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.03 Grade less than or equal to 1. 7. All subjects must have measurable disease by RECIST v1.1 or Response Assessment in Neuro-Oncology Criteria (RANO) criteria at time of enrollment. 8. Subjects with a primary CNS tumor or CNS metastases must be neurologically stable on a stable or decreasing dose of steroids for at least 14 days prior to enrollment. 9. Subjects must have a Lansky (< 16 years) score of at least 50. 10. Life expectancy greater than or equal to 12 weeks. 11. Adequate hematologic, renal and hepatic function. Key
Exclusion Criteria:
1. Subjects with neuroblastoma with only bone marrow disease evaluable by bone marrow aspiration only. 2. Major surgery within 14 days (2 weeks) of start of repotrectinib treatment. Central venous access (Broviac, Mediport, etc.) placement does not meet criteria for major surgery. 3. Known active infections (bacterial, fungal, viral including HIV positivity). 4. Gastrointestinal disease (e.g., Crohn's disease, ulcerative colitis, or short gut syndrome) or other malabsorption syndromes that would impact drug absorption. 5. Any of the following cardiac criteria:
• Mean resting corrected QT interval (ECG interval measured from the onset of the QRS complex to the end of the T wave) for heart rate (QTc) > 470 msec obtained from three ECGs, using the screening clinic ECG machine-derived QTc value
• Any clinically important abnormalities in rhythm, conduction, or morphology of resting ECG (e.g., complete left bundle branch block, third degree heart block, second degree heart block, PR interval > 250 msec)
• Any factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, congenital long QT syndrome, family history of long QT syndrome, or any concomitant medication known to prolong the QT interval 6. Peripheral neuropathy of CTCAE ≥grade 2. 7. Subjects being treated with or anticipating the need for treatment with strong CYP3A4 inhibitors or inducers.
Drug: Oral repotrectinib (TPX-0005)
Lymphoma, Locally Advanced Solid Tumors, Metastatic Solid Tumors, Primary CNS Tumors
ALK, ROS1, NTRK1-3, Primary CNS tumor, anaplastic large cell lymphoma, metastatic solid tumor, advanced solid tumor, sarcoma, infantile fibrosarcoma, glioblastoma, soft tissue schwannoma, solitary fibrous tumor, glioma, inflammatory myofibroblastic tumor, pediatric
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Effects of Dexrazoxane Hydrochloride on Biomarkers Associated With Cardiomyopathy and Heart Failure After Cancer Treatment (HEART)

This clinical trial studies the effects of dexrazoxane hydrochloride on biomarkers associated with cardiomyopathy and heart failure after cancer treatment. Studying samples of blood in the laboratory from patients receiving dexrazoxane hydrochloride may help doctors learn more about the effects of dexrazoxane hydrochloride on cells. It may also help doctors understand how well patients respond to treatment.
Call 1-888-980-6050
canceranswerline@utsouthwestern.edu
Daniel Bowers
10760
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01790152
STU 092013-038
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Inclusion Criteria:

• STRATUM I AND STRATUM II: LEUKEMIA AND LYMPHOMA SURVIVORS
• Previously enrolled leukemia and lymphoma survivors, randomized to + or
•DRZ on P9404, P9425, P9426, or DFCI 95-01 (high-risk patients only)
• STRATUM I: Alive and in continuous first complete remission from their original cancer (leukemia/lymphoblastic lymphoma [P9404, high-risk DFCI 95-01] or Hodgkin lymphoma [P9425/P9426])
• STRATUM I: Did not have progressive disease or induction failure requiring off-protocol therapy including hematopoietic cell transplantation
• STRATUM I: Must not have been diagnosed with any subsequent malignancy that required additional cardiotoxic therapies (i.e., radiotherapy to the chest [also includes fields directed towards the neck, upper abdomen, or spine], or additional anthracyclines or anthraquinones); patients with history of subsequent malignancy that did not require such therapies remain eligible
• STRATUM I: All patients and/or their parents or legal guardians must sign a written informed consent
• STRATUM II: Among leukemia and lymphoma patients randomized to + or
•DRZ on P9404, P9425, P9426, and DFCI 95-01 (high risk patients only) who have relapsed or have experienced a subsequent malignancy that precludes eligibility since their original diagnosis, the study committee will review the available data (both from Children's Oncology Group's [COG?s] Statistics and Data Center [SDC] and the participating institution) to determine if individual patients are to be selected for Stratum 2; in recognition that local institutions sometimes have more updated relapse/subsequent cancer data than SDC, in cases where local data is more updated, local data will be used preferentially; the study will petition the Institutional Review Board (IRB) specifically for a waiver of consent to include any relapse and subsequent cancer data obtained from existing records for analysis of the secondary aims; patients selected for Stratum 2 will be those for whom late relapse or subsequent cancer is reported but who lack clear confirmation in existing records (either at SDC or at the local institution)
• STRATUM II: Alive, but have experienced relapse of their original cancer and/or have developed a subsequent cancer (other than non-melanomatous skin cancer) since their original diagnosis
• STRATUM II: All patients and/or their parents or legal guardians must sign a written informed consent
• STRATUM III: OSTEOSARCOMA SURVIVORS
• Previously enrolled osteosarcoma survivors treated on P9754 who are alive and able (themselves and/or parents/legal guardian) to provide written informed consent; note that relapse and subsequent malignancy are not exclusion criteria for P9754 survivors
• Comparison subjects for P9754 survivors will be eligible to be enrolled from any ALTE11C2 participating COG site (even if that institution did not participate on P9754), according to the following criteria:
• Newly diagnosed, previously untreated biopsy-proven moderate or high grade osteosarcoma without metastasis; patients with low grade osteosarcoma, parosteal or periosteal sarcoma are ineligible
• < 31 years of age at time of initial osteosarcoma diagnosis
• Diagnosis occurred between January 1, 1999 through December 31, 2002; duration of therapy can extend beyond 2002
• No evidence of poor or low cardiac function at time of initial osteosarcoma diagnosis; if reports from the time are available: shortening fraction >= 28% by echocardiogram and within the institutional normative range for age, or radionuclide angiogram ejection fraction >= 50%; if imaging reports from the time are no longer available, there must be no documentation within available medical records that suggest poor or low cardiac function at time of diagnosis
• Comparison subject must have institutional records (e.g., clinic note, treatment summary, chemotherapy roadmap) documenting lifetime receipt of 450 to 600 mg/m^2 of doxorubicin (doses within 10% are acceptable); this includes initial therapy as well as any subsequent therapy for relapse or second cancer, if relevant; as such, comparison subjects who have had osteosarcoma relapse or subsequent malignancies remain eligible so long as they meet all other eligibility criteria
• No anthracycline or anthraquinone aside from doxorubicin was ever given as part of initial or subsequent therapies
• No exposure to DRZ at any point in time
• All patients and/or their parents or legal guardians must sign a written informed consent
• STRATUM IV: CARDIOMYOPATHY CASES, NOT OTHERWISE ELIGIBLE FOR STRATUMS 1, 2, AND 3
• Individuals diagnosed with cancer prior to age 21 years, who required treatment with chemotherapy and/or radiotherapy, achieved initial remission, and remained alive after completing anti-cancer-therapy for at least 1 year
• Must have screening echocardiograms for heart function as part of cancer therapy and off-therapy evaluations available (Digital Imaging and Communications in Medicine [DICOM] format). Images from Video Home System (VHS) tapes and reports only (without images) are not suitable
• Cannot have a known history of congenital heart disease (patent foramen ovale remain eligible) or underlying genetic syndrome associated with abnormal cardiovascular development or health (e.g., down syndrome)
• Based on echocardiography, must have either left ventricular fractional shortening =< 28.0% or ejection fraction =< 50.0% on at least two occasions, with at least one of these measurements occurring after cancer therapy completion and be in the absence of sepsis or any uncontrolled infection
• If the fractional shortening or ejection fraction criteria is only met on one occasion, this must be after cancer therapy completion, be in the absence of sepsis or any uncontrolled infection, and the patient must have subsequently started on chronic medical therapy for cardiomyopathy (e.g., beta-blocker, angiotensin-converting enzyme [ACE]-inhibitor, angiotensin receptor blocker) lasting at least 6 months
• For all participants (stratums 1, 2, 3, and 4), all institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Other: Assessment of Therapy Complications, Other: Laboratory Biomarker Analysis, Other: Quality-of-Life Assessment, Other: Questionnaire Administration
Lymphoblastic Lymphoma, Osteosarcoma, Recurrent Lymphoma, Leukemia in Remission, Hodgkin Lymphoma in Remission, Recurrent Leukemia, Recurrent Malignant Neoplasm
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Safety Study of Cord Blood Units for Stem Cell Transplants

Background: - Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants have been performed in children and adults for blood cancers and other diseases in the world. These transplants have helped save lives and improve treatments. However, not all available units of cord blood have been collected, stored, and licensed according to specific government requirements. These unlicensed units can still be used in transplant, but they can only be given as part of specific research studies. This study will evaluate the safety of giving these unlicensed units by recording any problems that may occur during and after giving the cord blood. Objectives: - To test the safety and effectiveness of unlicensed cord blood units in people who need stem cell transplants. Eligibility: - Individuals who are scheduled to have a stem cell transplant. Design: - Participants will be screened with a medical history and physical exam. - Participants will receive the cord blood unit as part of their stem cell transplant procedure. The transplant will be performed according to the current standard of care for the procedure. - After the transplant, participants will be monitored for up to 1 year. Any problems or side effects from the transplant will be treated as necessary. All outcomes will be reported to the National Cord Blood Program and to the Center for International Blood and Marrow Transplant.
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Victor Aquino
10208
All
Not specified
Phase 2
This study is NOT accepting healthy volunteers
NCT01861093
STU 082013-056
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• INCLUSION CRITERIA:
• Patients of any age or either gender with indications for receipt of investigational HPC-CORD BLOOD who are participating in an NIH-IRB approved clinical trial for unrelated hematopoietic stem cell transplantation.
• Signed informed consent (and assent when applicable). EXCLUSION CRITERIA:
• Patients who are receiving licensed CB products (only)
• Patients who are receiving unlicensed CB products from other CB banks (i.e. NMDP)
Procedure: Cord Blood Units
Leukemia, Lymphoma, Aplastic Anemia, Myelodysplastic Syndrome (MDS)
Unrelated Hematopoietic Stem Cell Transplantation, Cryopreserved Cord Blood Units, National Cord Blood Program
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Aneurysmal Subarachnoid Hemorrhage Trial RandOmizing Heparin (ASTROH)

A Blind-adjudication Multi-center Phase II Randomized Clinical Trial of Continuous Low-dose Intravenous Heparin Therapy in Coiled Low-grade Aneurysmal Subarachnoid Hemorrhage Patients with Significant Hemorrhage Burden.
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Babu Welch
67812
All
18 Years to 70 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02501434
STU 042016-042
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Inclusion Criteria:
1. Age ≥ 18 and ≤ 70 years 2. Historical modified Rankin Scale Score 0-1 3. WFNS SAH Scale grade ≤ 2, due to a spontaneous subarachnoid hemorrhage attributable to a ruptured cerebral aneurysm. Cranial nerve deficit (e.g. CN III paresis) is permissible even for WFNS grade 2. • Initial WFNS grade may be determined at admission or enrollment, preferably after the patient's mental status has been optimized by resuscitation and interval treatment of hydrocephalus (i.e., placement of intraventricular catheter) or reversal/wearing-off of sedating medications used commonly during patient transfers and transport or procedure related anesthesia. 4. Admission head CT showing modified Fisher grade 3 aSAH primarily in the supratentorial space. Snapshot images of up to four relevant axial cuts from the admission head CT will need to be uploaded via the imaging database to confirm the modified Fisher grade 3 eligibility of the potential subject prior to enrollment. Minimal intraventricular hemorrhage is acceptable. The Modified Fisher CT rating scale: Grade 1 (minimal or diffuse thing SAH without IVH); Grade 2 (minimal or thin SAH with IVH), Grade 3 (thick cisternal clot without IVH), Grade 4 (thick cisternal clot with IVH) [From: Claassen J et al. Effect of cisternal and ventricular blood on risk of delayed cerebral ischemia after subarachnoid hemorrhage: the Fisher scale revisited. Stroke 2001;32:2012-2020.] 5. Location and pattern of the SAH must have the majority of the SAH in the supratentorial space caused by either an intradural anterior circulation aneurysm OR a basilar apex posterior circulation aneurysm with primarily supratentorial hemorrhage extension. Angiographic location of the aneurysm will be confirmed by catheter digital subtraction angiography (DSA) usually obtained during the coil embolization procedure. Patients with PICA or other posterior circulation aneurysms as the cause of the SAH will be excluded. 6. Onset of symptoms of aSAH (ictus) occurred < 24 hours prior to presentation at the treating facility. 7. Initiation of aneurysm securement procedure occurred < 48 hours from the ictus AND less than 36 hours from admission to the treating facility. • In patients where the exact time of the ictus is uncertain, an estimated time of ictus may be assigned and that time will be used for the inclusion criteria above assuming the estimation is deemed to be reasonably reliable [i.e., actual time is highly likely to be within 6 hours of estimated time]. 8. All aneurysm(s) suspected to be responsible for the hemorrhage or potentially responsible for the hemorrhage must be secured in the following manner prior to enrollment: • Endovascular Coil Embolization with a post-embolization Raymond-Roy Score of 1 (Complete) or 2 (Residual Neck) 9. Ability to screen the patient and obtain a head CT 2-12 hours after the completion of the coiling procedure and then ability to initiate the study drug 12 ± 4 hours after the completion of aneurysm coiling procedure.. 10. After recovering from anesthesia following the aneurysm coiling procedure, the patient must remain a WFNS SAH grade ≤ 2 without evidence of a significant new focal neurological deficit including monoparesis / monoplegia, hemiparesis / hemiplegia, or receptive, expressive or global aphasia. New minor cranial nerve defect without any other new findings is permissible. If an NIHSS score was obtained prior to the aneurysm coiling procedure, a post-coiling (pre-enrollment) NIHSS score must not have increased by ≥ 4 points and GCS score must not be decreased by ≤ 2 points. The clinician at the local site should use their best clinical judgment as to whether a significant neurological decline has occurred due to the procedure. 11. Patient is willing and able to return for study follow-up visits. 12. Patient or their Legally Authorized Representative (LAR) has provided written informed consent.
Exclusion Criteria:
1. Angio-negative SAH. 2. A likely hemorrhage event within several days prior to admission related hemorrhage ictus due to the increased risk of early vasospasm. Prior sentinel headache with negative CT or prior sentinel headache where the patient did not seek medical attention does not exclude the patient. 3. Surgical clipping of the ruptured aneurysm or any non-ruptured aneurysm on the same admission prior to enrollment. 4. SAH not caused by aneurysm rupture or aneurysm is identified to be traumatic, mycotic, blister or fusiform type by catheter DSA. 5. Any intracranial stent placement or non-coil intra-aneurysmal device (i.e., stent- assisted coiling with Neuroform, Enterprise, LVIS, LVIS Jr, Barrel Stent, Pulse Rider, WEB, LUNA, Medina or a similar device) where the stent device is implanted to treat the ruptured aneurysm and / or antiplatelet therapy is needed. 6. Patient has remaining aneurysm(s) that are untreated and could reasonably be considered a possible alternate cause of the aSAH based on the observed bleeding pattern. Adequate treatment of these aneurysms by coiling embolization would result in the aneurysms no longer causing an exclusion. MRI may be used in some situations to determine that the associated aneurysms did not rupture based on lack of blood seen adjacent to the additional aneurysms. 7. Femoral arteriotomy stick above the inferior epigastric artery OR angiographic, CT, or clinical evidence of an arteriotomy related retroperitoneal hematoma or large flank hematoma. A stable groin hematoma is not an exclusion. 8. Patient received heparin in any form within the last 100 days prior to admission (not including coiling procedure). 9. Thrombocytopenia (platelet count less than 100,000
•assuming clumping has been ruled out as a cause), confirmed active disseminated intravascular coagulation (DIC) at the time of enrollment OR a documented history of coagulopathy or bleeding diathesis. 10. Diagnosis of sepsis (SIRS criteria plus the presence of known or suspected infection) or current documented active bacterial or viral infection prior to enrollment (Example: significant URI, community-acquired pneumonia). A minor non- complicated community-aquired urinary tract infection would not be an exclusion but should be treated promptly. 11. New parenchymal hemorrhage or new infarction larger the 15cc in volume, or significant increased mass effect as seen on the post coiling pre-enrollment head CT when compared to baseline admission head CT. New hyyperdensity on CT scan related to contrast staining is not an exclusion. 12. Patient has a documented history of heparin induced thrombocytopenia (HIT) 13. Patient developed SAH-induced cardiac stunning prior to enrollment, with an ejection fraction< 40%, or requiring IV medicaitons for blood pressure maintenance. 14. Pre-admission daily antiplatelet therapy or any oral or subcutaneous anticoagulation therapy prior to, or during the coil embolization procedure. A single 325 mg Aspirin (or lower dose) given duiring the coil embolization peri-procedural period is acceptable if this is the local standard of care, but should be documented. 15. Thrombolytic therapy within 24 hours prior to enrollment (rtPA, urokinase, etc.) 16. Plan for antiplatelet or oral anticoagulation therapy from the time of the coil embolization procedure until 14 full days after enrollment. Antiplatelet therapy may be resumed after the 14-day window. 17. Concurrent significant intracranial pathology identified prior to enrollment, including but not limited to, Moyamoya disease, high suspicion or documented CNS vasculitis, severe fibromuscular dysplasia, arteriovenous malformation, arteriovenous fistula, significant cervical or intracranial atherosclerotic stenotic disease (≥70%), or malignant brain tumor. 18. Uncontrollable hypertension (>180 systolic and/or >110 diastolic) that is not correctable prior to enrollment. 19. Known seizure or epilepsy disorder (diagnosed prior to this aSAH diagnosis) where anti-epileptic medication was previously taken by the patient or have been recommended to be taken by the patient. Childhood seizures that have resolved and no longer require treatment are not part of this exclusion criteria 20. Serious co-morbidities that could confound study results including but not limited to: Multiple Sclerosis, dementia, severe major depression, cancer likely to cause death in 2 years, multi-system organ failure, or any other conditions that could cause any degree of cognitive impairment. 21. Immunosuppression therapy including chronic corticosteroid usage. 22. Remote history of previous ruptured cerebral aneurysm. 23. History of gastrointestinal hemorrhage or major systemic hemorrhage within 30 days, hemoglobin less than 8 g/dL, INR ≥1.5, severe liver impairment (AST< ALT< AP 2 x normal or cirrhosis), creatinine > 2.0 mg/dL, or estimated GFR < 60 ml/min. 24. Major surgery (including open femoral, aortic, or carotid surgery) within previous 30 days. 25. Currently pregnant. 26. Enrollment in another research study that would conflict with this study.
Drug: Continuous Low-Dose Intravenous Unfractionated Heparin Infusion (14 days)
Aneurysmal Subarachnoid Hemorrhage, Neurobehavioral Manifestations, Vasospasm, Intracranial, Intracranial Aneurysm, Heparin-induced Thrombocytopenia Type II
aneurysmal subarachnoid hemorrhage, unfractionated heparin, neurobehavioral manifestations, delayed ischemic neurological deficits, cerebral aneurysm, coil embolization, Montreal Cognitive Assessment
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Study of INTERCEPT Blood System for Red Blood Cells in Regions at Potential Risk for Zika Virus Transfusion-Transmitted Infections (RedeS) (RedeS)

Stage A: To evaluate the safety and efficacy of the INTERCEPT Blood System for Red Blood Cells Pathogen Reduction Treatment (PRT) in comparison to conventional RBCs in adult patients who require RBC transfusion support. Stage B: To provide early access to the INTERCEPT pathogen reduction system for RBC in regions where a substantial proportion of the population has been infected or is at risk of infection by the Zika virus (ZIKV), and the risk of asymptomatic infection among qualified blood donors is recognized. Besides the reduction of risk of transfusion transmitted ZIKV, the intent of the study is also to reduce the risk of transfusion-transmitted infections (TTI) in general, including transfusion related sepsis and other emerging or concurrent endemic pathogens (e.g. Dengue and Chikungunya), and to reduce the risk of TA-GVHD. As part of this treatment use study, additional data will be provided on the safety of INTERCEPT-treated RBCs (IBS RBCs) supplied for routine clinical transfusion practice.
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Ravindra Sarode
48082
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03037164
STU 032018-059
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Stage A:
Inclusion Criteria:
1. Age ≥ 18 years 2. Patients who are required or expected to require a transfusion of RBC component(s). 3. Signed and dated informed consent 4. Female patients of child-bearing potential must: 1. Have negative serum or urine pregnancy tests prior to study treatment to rule out pregnancy, and 2. Agree to use at least one medically accepted method of birth control that results in a low failure rate (i.e. less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptives, some intrauterine devices, sexual abstinence or vasectomized partner for the duration of study participation and for an additional 28 days. Stage A:
Exclusion Criteria:
1. Confirmed positive baseline serum/plasma antibody specific to IBS RBC as determined by INTERCEPT antibody screening panel prior to receiving their first study transfusion 2. Pregnant or breast feeding. 3. Patients who require neonatal transfusions and intrauterine transfusions. 4. Patients with documented IgA deficiency or a history of severe allergic reactions to blood products. 5. Presence of a RBC warm autoantibody or positive DAT with a panreactive eluate. 6. Have had an RBC transfusion during current hospitalization prior to enrollment (within 28 days) 7. Have received investigational products, including investigational blood products, pharmacologic agents or imaging materials, within the prior 28 days. Prior receipt of conventional blood products tested with an investigational ZIKV NAT test are not considered grounds for exclusion despite the investigational nature of the NAT test. 8. Patients presenting with or expected to have massive hemorrhage (≥10 RBC units within 24 hours) or expected to require massive transfusion protocols Stage B: Inclusion Criteria 1. Age ≥ 18 years 2. Patients who are require or expected to require a transfusion of RBC component(s). 3. Signed and dated informed consent Stage B: Exclusion Criteria 1. Confirmed positive baseline serum/plasma antibody specific to IBS RBC as determined by INTERCEPT antibody screening panel prior to receiving their first study transfusion 2. Pregnant or breast feeding. 3. Patients who require neonatal transfusions and intrauterine transfusions. 4. Patients with documented IgA deficiency or a history of severe allergic reactions to blood products.
Device: INTERCEPT Blood System for Red Blood Cells, Device: Conventional (Control)
Anemia
INTERCEPT, Red Blood Cells, RBC, Pathogen Inactivation, Zika, Cerus, Pathogen Reduction
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Comparing Individualized vs. Weight Based Protocols to Treat VOE in SCD Occlusive Episodes in Sickle Cell Disease (COMPARE-VOE)

The purpose of this research study is to compare two different ways to give opioid pain medicine to treat sickle cell disease pain that is bad enough to go to the emergency department for treatment. One way uses your weight to decide how much pain medicine to give you while in the emergency department. This is called weight based treatment. The other way uses how much pain medicine you take at home and how much medicine you needed during past emergency department visits to decide how much medicine to give you. This is called patient specific treatment.
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Ava Pierce
75453
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03933397
STU-2019-0671
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Inclusion Criteria:

• All adult (18 years or older);
• SCD patients with the following genotypes: Hgb SS, SC, and SB+ and SB- thalassemia
Exclusion Criteria:

• determined to not benefit from opioids and therefore won't receive opioids in any future ED visit.
Other: Patient-Specific Protocol, Other: Weight-based Protocol, Drug: Morphine, Drug: Hydromorphone
Sickle Cell Disease
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The Cxbladder Hematuria Clinical Utility Study

To evaluate the clinical utility associated with the integration of Cxbladder into the evaluation of subjects presenting with hematuria for evaluation of urothelial carcinoma (UC) without compromising detection of UC.
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Yair Lotan
59883
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03988309
STU-2019-1020
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Inclusion Criteria : 1. Patient is undergoing investigation of recent confirmed hematuria (by either flexible or rigid cystoscopy/TURBT), including hematuria subjects referred due to suspicious/positive imaging, in order to determine the presence of urothelial carcinoma. 2. Able to provide a voided urine sample of the required minimum volume 3. Able to give written consent 4. Able and willing to comply with study requirements 5. Aged 18 years or older Exclusion Criteria 1. Prior history of bladder malignancy, prostate or renal cell carcinoma 2. Prior genitourinary manipulation (flexible or rigid cystoscopy / catheterisation, urethral dilation) in the 14 days before urine collection, 3. History of glomerulonephritis, nephrosis or other renal inflammatory disorders, recent history of pyelonephritis 4. Previous alkylating based chemotherapy 5. Pregnancy
Diagnostic Test: Cxbladder
Urothelial Carcinoma, Hematuria
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International Registry for Severe Chronic Neutropenia

OBJECTIVES: I. Document the clinical course of severe chronic neutropenia (SCN). II. Monitor and assess long term safety of primary treatment in SCN patients in the United States, Canada, Europe, and Australia. III. Study the incidence and outcome of adverse events such as osteoporosis, splenomegaly, cytogenetic abnormalities, myelodysplastic syndrome, and leukemia. IV. Evaluate growth and development and hematologic parameters. V. Monitor for clinically significant changes in primary treatment response over time. VI. Establish a physician network to increase the understanding of SCN. VII. Establish a demographic database to allow for future research.
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Kathryn Dickerson
156007
All
3 Months and over
N/A
This study is NOT accepting healthy volunteers
NCT00004342
STU 062010-080
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PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Severe chronic neutropenia (SCN), i.e.: Absolute neutrophil count less than 500/mm3 on 3 occasions within the last 3 months (less than 200/mm3 for cyclic neutropenia) Bone marrow aspirate consistent with SCN History of infection No drug induced neutropenia No myelodysplastic syndrome No aplastic anemia No thrombocytopenia or anemia unless due to Shwachman-Diamond syndrome or glycogen storage disease type IB Prior enrollment on Amgen SCN trials eligible Bone marrow aspiration within 1 year required Cytogenetic evaluation strongly suggested --Prior/Concurrent Therapy-- At least 5 years since prior chemotherapy --Patient Characteristics-- No rheumatoid arthritis No systemic lupus erythematosus No HIV seropositivity
Neutropenia
chronic neutropenia, disease-related problem/condition, hematologic disorders, neutropenia, rare disease, severe chronic neutropenia
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Low VW Activity in Adolescent HMB (Low VWF)

This is a research study for patients diagnosed with heavy menstrual bleeding (HMB) and low Von Willebrand Factor (VWF). Menstruation, also known as a period, is the regular discharge of blood and tissues from the uterus. HMB is having a heavier amount of discharge during menstrual period. Low Von Willebrand Factor means that the participant has lower level of a blood protein that is important for clotting of blood and so, the participant is at a higher risk for bleeding. The purpose of this project is to study the genetic differences of adolescent females with HMB and low VWF activity and compare the genetic differences with their bleeding manifestations, response to medications and outcome.
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Ayesha Zia
149180
Female
up to 21 Years old
This study is NOT accepting healthy volunteers
NCT02933411
STU 012017-074
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Inclusion Criteria:

• Post-menarchal females less than 21 years of age
• HMB defined as PBAC score greater than 100
• VWF:Activity more than or equal to 30 and less than or equal to 50 IU/dL x 2
• VWF: Activity /VWF:Ag ratio greater than or equal to 0.6
• Normal VW multimers, if performed
Exclusion Criteria:

• Post menarchal females age greater than or equal to 21 years
• VWF: Activity less than 30 or greater than 50 IU/dL consistently, type 2 or type 3 VWD
• Presence of other bleeding disorders (thrombocytopenia, platelet function defect, coagulation factor deficiency, fibrinogen defect or deficiency)
Other: Genetic Analysis, Other: Medical Record Data Abstraction, Other: Pictorial Blood Assessment Chart (PBAC) score, Other: Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Von Willebrand Factor Deficiency
Bleeding Disorders, Heavy Menstrual Bleeding
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Avazzia-University of Texas Southwestern Medical Center-Tennant Biomodulator® PRO Perfusion Study

This study is an open label trial designed to test the effectiveness of the Tennant Biomodulator® PRO electrical stimulation device (Avazzia), which uses BEST™ (Bio-Electric Stimulation Technology), on hospital in-patients to improve perfusion in the treated and contralateral limb.
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Lawrence Lavery
116716
All
18 Years to 89 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03843307
STU 042018-005
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Inclusion Criteria:

• Able to provide Informed Consent
• Ankle Brachial Index (ABI) ≥ 0.5 (bedside ABI is acceptable for screening purposes as the formal imaging ABI may not be resulted prior to therapy)
• Participant is a hospital in-patient for the duration of study procedures
• One or more chronic lower extremity wounds that are located in the ankle area or below that has persisted a minimum of 30 days prior to the Screening visit
• 18 years of age or older
Exclusion Criteria:

• Unable to provide informed consent
• <18 years of age
• Participant has a demand-type cardiac pacemaker, implanted defibrillator or other implanted metallic or electronic device.
• Participant has untreated osteomyelitis
• Participant has active cellulitis
• Participant has active charcot
• 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 participant's ability to provide informed consent, participate in the study protocol including untreated schizophrenia, bipolar disorder and psychiatric hospitalization within the last 2 years.
Device: Avazzia Tennant Biomodulator® PRO
Perfusion, Complications, Wound, Foot
Electrical stimulation, Perfusion, Foot wound, Foot pain
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ATHN 7: Hemophilia Natural History Study (ATHN 7)

This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment. The hemophilia treatment center (HTC) physician and participant will decide on the FDA-approved treatment to be used which may include non-factor products, bypassing agents, or clotting factor replacement products. The goal of this research is to study the use of hemophilia treatment products and their outcomes.
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Ayesha Zia
149180
All
Not specified
This study is NOT accepting healthy volunteers
NCT03619863
STU-2019-0673
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Inclusion Criteria:
1. Congenital hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility; 2. Able to give informed consent (by patient or parent/authorized guardian); and 3. Co-enrollment in the ATHNdataset.
Exclusion Criteria:
1. Presence of any known bleeding disorder other than congenital hemophilia A or B; 2. Presence of concurrent hemophilia and a second hemostatic defect (low Von Willebrand Factor (VWF) without Von Willebrand disease (VWD) diagnosis is not excluded); and 3. Unable or unwilling to comply with the study protocol.
Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor
Hemophilia, Non-Factor Products, Bypassing Agents, Clotting Factor Replacement Products
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A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.
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canceranswerline@utsouthwestern.edu
Victor Aquino
10208
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01351545
STU 052011-121
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Inclusion Criteria:

• Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment
• Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment
• Pediatric and adult patients of any age
Exclusion Criteria:

• Patients who are receiving only licensed CBUs
• Cord blood transplant recipients at international transplant centers
• Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
• Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Automimmune Diseases, Severe Aplastic Anemia
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Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)

Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA. The purpose of this follow-up study is to evaluate the long-term effects of hydroxyurea in children who have participated in the BABY HUG study.
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studyfinder@utsouthwestern.edu
An Pham
170449
All
2 Years to 7 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT00890396
STU 032012-018
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Inclusion Criteria:

• All children who completed at least 18 months of follow-up visits in the initial BABY HUG study
• Children from the initial BABY HUG study who are on a chronic transfusion program or who are recipients of a bone marrow transplant
Exclusion Criteria:

• Any child who was not enrolled in the initial BABY HUG study for at least 18 months
Anemia, Sickle Cell
Sickle Cell Anemia, Hydroxyurea
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MMRF Molecular Profiling Protocol

Here we propose an "integrative sequencing approach" utilizing a 1500 gene exome comparative analysis between multiple myeloma or related plasma cell malignancies and normal cells coupled to capture transcriptome sequencing to provide a nearly comprehensive landscape of the genetic alterations for the purpose of identifying informative and/or actionable mutations in patients with multiple myeloma and plasma cell malignancies. The approach will enable the detection of point mutations, insertions/deletions, gene fusions and rearrangements, amplifications/deletions, and outlier expressed genes among other classes of alterations.
Call 1-888-980-6050
canceranswerline@utsouthwestern.edu
Ankit Kansagra
177999
All
18 Years and over
This study is NOT accepting healthy volunteers
NCT02884102
STU-2018-0178
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Inclusion Criteria:
1. Patients must have a diagnosis of multiple myeloma or related malignancy 2. Patients are undergoing standard of care bone marrow aspirates 3. Patients (male or female) from any race or ethnicity must be at least 18 years of age at the time of registration. 4. Procedure-specific signed informed consent form prior to initiation of any study-related procedures.
Exclusion Criteria:
1. It is the enrolling study physician's discretion to decide if a patient is not fit enough to undergo a bone marrow aspirate. 2. Patients who are incarcerated are not eligible to participate. 3. Women who are pregnant 4. Patients who have had another malignancy within the last five (5) years (except for basal or squamous cell carcinoma, or in situ cancer of the cervix) where there is a possibility to contaminate the bone marrow aspirate.
Genetic: genetic sequencing
Multiple Myeloma, Plasma Malignancy
genetic sequencing, relapsed
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Shock, Whole Blood, and Assessment of TBI S.W.A.T. (LITES TO 2) (SWAT)

The LITES Network is an operational trauma center consortium which has the expertise, track record and confirmed capabilities to conduct prospective, multicenter, injury care and outcomes research of relevance to the Department of Defense (DoD). Hemorrhage and Traumatic Brain Injury (TBI) are responsible for the largest proportion of all trauma-related deaths. It is the poly-trauma patient who suffers both hemorrhagic shock and traumatic brain injury where a paucity of evidence exists to direct treatment, limiting the development of beneficial trauma practice guidelines. The use of Whole Blood (WB) for early trauma resuscitation has been touted as the 'essential next step' in the evolution of trauma resuscitation. Despite its historical and more recent use, little is known regarding WB's benefit relative to the 'current practice' ratio-based blood component therapy in the acutely bleeding patient, and even less is known regarding its effects in patients with TBI. AIM#1: Evaluate patient centered outcomes associated with early whole blood resuscitation practice as compared to component resuscitation in poly-trauma patients with hemorrhagic shock and further characterize outcome benefits in those with traumatic brain injury. AIM#2: Characterize blood pressure and resuscitation endpoints during the acute resuscitation phase of care and the associated/attributable outcomes for traumatic brain injury in patients with hemorrhagic shock. General Hypothesis #1: Whole blood resuscitation will be associated with improved mortality and resuscitation outcomes in poly-trauma patients and long term neurological outcome in those patients with traumatic brain injury as compared to those resuscitated with component therapy. General Hypothesis #2: Differences in prehospital and acute phase resuscitation systolic blood pressure will be associated with differential outcomes in patients with traumatic brain injury at discharge and at 6 months. Study Design: The LITES network will perform a multicenter, prospective, observational cohort study over a 4 year period to determine the impact of whole blood resuscitation in trauma patients with hemorrhagic shock at risk of large volume resuscitation with and without TBI. Early whole blood resuscitation will be compared to standard component resuscitation. The study will also further characterize blood pressure and resuscitation endpoints in poly-trauma patients with traumatic brain injury. Six Trauma sites with appropriate characteristics will be selected from 12 LITES Network sites across the country. Study Setting: The study will be performed utilizing busy level I trauma centers within the LITES Network located across the country, at sites where either whole blood has currently been incorporated into standard of care or where component blood transfusion is being utilized for patients in hemorrhagic shock at risk for large volume resuscitation. Study Population: The study will focus on patients who suffer blunt or penetrating injury, transported to a SWAT participating LITES trauma center with evidence of hemorrhagic shock at risk of large volume blood resuscitation.
Call 214-648-5005
studyfinder@utsouthwestern.edu
Joseph Minei
14988
All
15 Years to 90 Years old
This study is NOT accepting healthy volunteers
NCT03402035
STU 032018-032
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Inclusion Criteria:
Patients with blunt or penetrating injury who meet the following criteria: 1, 2, and 3 1. Has 2 or more of any of the following: 1. Hypotension (systolic blood pressure ≤ 90 mmHg) in the prehospital or emergency department setting, 2. Penetrating mechanism, 3. Positive FAST abdominal ultrasound, 4. Heart Rate ≥ 120 in the prehospital or emergency department setting. AND 2. Taken to the Operating Room (laparotomy, thoracotomy or vascular exploration) or Interventional Radiology within 60 minutes of arrival. AND 3. Need of blood/blood component transfusion in prehospital setting, ED or OR within 60 minutes of arrival.
Exclusion Criteria:
1. Age ≤ 14 2. CPR > 5 consecutive minutes without ROSC 3. Penetrating brain injury with brain matter exposed 4. ED death
Traumatic Brain Injury, Hemorrhagic Shock
Whole Blood Resuscitation
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ROTEM® Sigma Performance Evaluation - Method Comparison With Predicate Device and Reference Intervals (ROSI-EVA)

ROTEM® sigma is a new thromboelastometric coagulation analysis system. The new system is fully automated allowing for a simple and safe operation compared to its predecessor model ROTEM® delta. Method comparison of ROTEM® sigma with predecessor model ROTEM® delta) - confirmation of equality. Confirmation of reference intervals of the ROTEM® systems.
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studyfinder@utsouthwestern.edu
Michael Cripps
132904
All
18 Years and over
This study is also accepting healthy volunteers
NCT02379104
STU-2019-0498
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Inclusion Criteria:
Patients:
• Age ≥ 18 years
• Informed written consent
• Patients with an acute bleeding during surgery
• Or: trauma patients with acute bleeding
• Or: hypercoagulable (above normal fibrinogen) patients at the ICU Healthy Volunteers:
• Adult healthy individuals (≥ 18 years)
• Written informed consent
Exclusion Criteria:
Patients: • none Healthy Volunteers:
• Any indications of alcohol or illegal drug abuse
• Any chronic disease with possible effect on the coagulation (liver disease, coronary heart disease)
• Any acute illness within the last 14 days
• Any hemostasis associated disease (myocardial infarction, thrombosis , stroke etc.) within the last year
• Intake of any coagulation affecting medication (aspirin, pain-killers, anti-rheumatic drugs, marcumar, platelet inhibitors) within the last 14 days
• Females only: known pregnancy
• Females only: breast feeding
• Any indication for any hemostasis disorder as interrogated by a specific history of bleeding tendencies
Device: ROTEM sigma
Blood Coagulation Disorders
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