Clinical trials: Hereditary Anemias
Graft Versus Host Disease, Leukemia
OTI-010 for Graft-Versus-Host Disease Prophylaxis in Treating Patients Who Are Undergoing Donor Peripheral Stem Cell Transplantation for Hematologic Malignancies
RATIONALE: OTI-010 may be effective for graft-versus-host disease prophylaxis (prevention) in patients who are undergoing donor peripheral stem cell transplantation for hematologic malignancies (cancer of the blood or bone…
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00081055
- Testing
- autologous expanded mesenchymal stem cells OTI-010, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Mesoblast International Sàrl
Sickle Cell Disease
Is the Preoperative Preparation of Sickle Cell Patients Optimal: Assessment of Practices and Post-operative Complications
Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis…
- Trial name
- NCT04264871
- Where
- Belgium
- Volunteers
- 250 people
- Sponsor
- Queen Fabiola Children's University Hospital
Hepatitis C, Fibrosis
The Prevalence and Severity of HCV Infection in Thalassemia Major and Thalassemia Intermedia in Siriraj Hospital
The primary objective is to assess the prevalence and genotypes of HCV infection in thalassemia major and thalassemia intermediate patients who have received blood transfusion in the Department of Medicine, Faculty of Medicine…
- Conditions
- Hepatitis C, Fibrosis, Thalassemia
- Trial name
- NCT02904252
- Testing
- Transient elastography
- Where
- Thailand
- Volunteers
- 158 people
- Sponsor
- Mahidol University
Sickle-Cell Disease
Sevuparin Infusion for the Management of Acute VOC in Subjects With SCD
A Multi-Centre, Phase II, Randomized, Double-Blind, Placebo-Controlled Study to investigate Efficacy and Safety of Sevuparin Infusion for the Management of Acute Vaso-Occlusive Crisis (VOC) in Subjects with Sickle-Cell Disease…
- Trial name
- NCT02515838
- Testing
- Sevuparin
- Where
- 20 sites in Bahrain, Jamaica and 5 more
- Volunteers
- 147 people
- Sponsor
- Modus Therapeutics AB
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Cystic Fibrosis, Anemia, Iron-Deficiency
Does a Daily Iron Tablet Improve Anemia in Cystic Fibrosis
This study questions whether low-dose ferrous sulfate taken by mouth daily for 6 weeks increases hemoglobin concentration in adult subjects with cystic fibrosis and hypoferremic anemia.
- Trial name
- NCT01755455
- Testing
- Ferrous sulfate 325mg
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Dartmouth-Hitchcock Medical Center
Sickle Cell Disease
Codeine in Sickle Cell Disease
The objective of this study is to determine if a subject's genetic make-up would affect the treatment response to codeine in subjects with sickle cell disease.
- Trial name
- NCT00174538
- Testing
- Codeine (30 mg)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- PriCara, Unit of Ortho-McNeil, Inc.
Stroke Ischemic, Sickle Cell Disease
Evaluating Barriers to Stroke Screening and Prevention in Children With Sickle Cell Disease
DISPLACE is a three part, multi-center U.S. based study to evaluate the barriers to stroke screening and prevention in children with sickle cell anemia (SCA).
- Trial name
- DISPLACE (NCT03621826)
- Testing
- Questionnaire/Interview
- Where
- United States
- Volunteers
- 5,247 people
- Sponsor
- Medical University of South Carolina
Twin Dichorionic Diamniotic Placenta, Twin Monochorionic Monoamniotic Placenta
Austrian Registry on the Outcome of Monochorionic Pregnancies
About one third of twin pregnancies are resulting from a single fertilized oocyte. Two third of these monozygotic twins share a common placenta and are therefore called monochorionic.
- Conditions
- Twin Dichorionic Diamniotic Placenta, Twin Monochorionic Monoamniotic Placenta, Twin-To-Twin Transfusion Syndrome, Twin Pregnancy With Antenatal Problem
- Trial name
- MonoReg (NCT03171909)
- Where
- 13 sites in Austria
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Anemia, Sickle Cell
MAST - Magnesium for Sickle Cell Acute Crisis in Children
The purpose of this study is to determine if intravenous magnesium sulfate treatment is effective in reducing the length of stay and pain in children with sickle cell disease suffering an acute vaso-occlusive episode.
- Trial name
- NCT00313963
- Testing
- Magnesium Sulfate, Normal Saline
- Where
- Canada
- Volunteers
- 120 people
- Sponsor
- The Hospital for Sick Children
Sickle Cell Disease
A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS)…
- Trial name
- NCT04983264
- Testing
- GBT021601
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Pfizer
Pain, Sickle Cell Disorder
Ketamine Infusion for Acute Sickle Cell Crisis in the Emergency Department
Pain associated with sickle cell disease is a common emergency department visit. It is also frequently associated with a high emergency department recidivism rate for pain control and admissions to the hospital.
- Trial name
- KISS (NCT02417298)
- Testing
- Normal saline, Ketamine
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Billy Sin
Anemia, Sickle Cell
Trial of Oral Glutamine in Patients With Sickle Cell Anemia
Children with sickle cell anemia (SCA) seem to have higher energy needs than children who do not have the disease. This may be the reason why children and teenagers with sickle cell anemia tend to be smaller, weigh less, and have…
- Trial name
- NCT00131508
- Testing
- Glutamine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- St. Jude Children's Research Hospital
Hereditary Red Blood Cell Disorder (Disorder)
Luspatercept in Patients Affected With Rare Inherited Anemias
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias
- Trial name
- LUSPARA (NCT07331818)
- Testing
- Reblozyl
- Where
- 6 sites in France, Italy
- Volunteers
- 45 people
- Sponsor
- EuroBloodNet Association
Beta-Thalassemia
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
A clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator.
- Trial name
- NCT00069862
- Testing
- Deferitrin (GT56-252), desferoxamine (DFO)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Sickle Cell Disease
Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers
The investigators are conducting a Phase II prospective and placebo controlled study of a topical cream containing sodium nitrite compared to the current standard of care.
- Trial name
- NCT02863068
- Testing
- Topical Sodium Nitrite
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- caterina P minniti
Sickle Cell Disease
Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound
Sickle cell disease is a life-long blood condition that can cause damage to the brain and other organs of the body. Children may develop severe, debilitating clinical states, with stroke or abnormal blood flow to the brain.
- Trial name
- NCT00152113
- Testing
- Hematopoietic Stem Cell Transplantation, CliniMACS, See intervention description
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with…
- Trial name
- NCT00445978
- Testing
- Sapropterin Dihydrochloride
- Where
- 12 sites in United States
- Volunteers
- 32 people
- Sponsor
- BioMarin Pharmaceutical
Hematologic Malignancies, Nonmalignant Diseases
Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies
- Trial name
- NCT03609840
- Testing
- Thiotepa
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease
Medication Adherence in Youth With Sickle Cell Disease (SCD)
Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD.
- Trial name
- NCT02143076
- Testing
- Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital