Clinical trials: Hemoglobinopathies
Non-transfusion Dependent Thalassemia
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload.
- Trial name
- THALASSA (NCT00873041)
- Testing
- deferasirox
- Where
- 21 sites in United States, Greece and 7 more
- Volunteers
- 166 people
- Sponsor
- Novartis Pharmaceuticals
B Thalassemia
Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia
to evaluate thyroid function ,HbA1C,\&lipid profile in thalassemic patient to correlate thyroid function , Hba1c , lipid profile e ferritin level in thalassemic patient
- Trial name
- NCT06377865
- Volunteers
- 72 people
- Sponsor
- Assiut University
Sickle Cell Disease
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can…
- Trial name
- SUN (NCT03587272)
- Testing
- Alemtuzumab, low dose total body irradiation, Sirolimus
- Where
- 7 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Robert Nickel
Thalassemia
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia
The aim of this study is to determine the safety and therapeutic effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on beta thalassemia patients.
- Trial name
- NCT07673302
- Testing
- Thalidomide, Hydroxy Urea
- Where
- Pakistan
- Volunteers
- 240 people
- Sponsor
- Riphah International University
Thalassemia Major, Hemosiderosis
Efficacy Study in Removing Excess Iron From the Heart
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
- Trial name
- NCT00105495
- Testing
- Ferriprox (deferiprone), Desferal (deferoxamine)
- Where
- 4 sites in Greece, Italy
- Volunteers
- 60 people
- Sponsor
- ApoPharma
Sickle Cell Trait
Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT)
The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying…
- Trial name
- NCT06071377
- Testing
- Biologic Specimen Collection
- Where
- 8 sites in United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Alliance for Sickle Cell Centers
Sickle Cell Disease, Thalassemia
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and…
- Conditions
- Sickle Cell Disease, Thalassemia, Pediatric Cancer
- Trial name
- RBC-IMPACT (NCT05255445)
- Testing
- Red Blood Cell (RBC) Transfusion
- Where
- 14 sites in United States, Brazil
- Volunteers
- 157 people
- Sponsor
- Westat
Vaso-occlusive Crisis, Sickle Cell Disease
Evaluation of Virtual Reality to Save Morphinic in the Treatment of Vaso-occlusive Seizures of Sickle Cell Patients Consulting in the Emergency Room
The management of pain in the emergency department is a major issue, especially for sickle cell patients who regularly consult for vaso-occlusive seizure (VOS).
- Trial name
- ReVCVO (NCT04756375)
- Testing
- Virtual Reality, Usual Care
- Where
- France
- Volunteers
- 97 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids that would otherwise be started during a sickle cell pain crisis…
- Conditions
- Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children
- Trial name
- NCT07799571
- Testing
- Buprenorphine Buccal Film [Belbuca]
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Natasha Archer
Hematologic Malignancies, Nonmalignant Diseases
Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609814
- Testing
- Clofarabine, Fludarabine Injection
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease, Graft Versus Host Disease
Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease
To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving…
- Trial name
- NCT02867800
- Testing
- Diphenhydramine, Acetaminophen, Methylprednisolone
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Monica Bhatia
Thalassemia Major
Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH)
To assess the effect of different risk factors on the growth parameters of thalassemic patients in Assiut University children Hospital (AUCH) In order to help in decreasing the morbidity and mortality resulting from iron overload…
- Trial name
- NCT05303506
- Testing
- serum ferritin and AST
- Volunteers
- 50 people
- Sponsor
- Assiut University
Pulmonary Hypertension, Sickle Cell Anemia
A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study
This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease.
- Trial name
- ASSET-3 (NCT00360087)
- Testing
- Bosentan
- Volunteers
- 236 people
- Sponsor
- Actelion
Nursing Caries
Nonpharmacological Approaches and Parental Education in Children With Sickle Cell Disease
Aim: Sickle cell disease (SCD) is a hereditary disease. Approximately 5% of the world's population carries trait genes for haemoglobin disorders, mainly, sickle-cell disease and thalassaemia.
- Trial name
- NCT04121247
- Testing
- nonpharmacological approaches to parents of children with sickle cell disease training program
- Where
- Turkey (Türkiye)
- Volunteers
- 163 people, healthy volunteers welcome
- Sponsor
- Akdeniz University
Sickle Cell Disease
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years…
- Trial name
- NCT04774536
- Testing
- CRISPR_SCD001
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Mark Walters, MD
Sickle Cell Anemia, Pulmonary Hypertension
Tricuspid Regurgitant Jet Velocity as an Independent Marker for Mortality in Sickle Cell Anemia
The purpose of this study is to evaluate patients with pulmonary hypertension and sickle cell disease who have had multiple echocardiograms.
- Trial name
- NCT01783093
- Testing
- Data Collection
- Where
- United States
- Volunteers
- 209 people
- Sponsor
- Namita Sood
Drepanocytosis, Chronic Neurological Deficiency
Transfusion Exchanges and Cognition in Sickle Cell Disease
DREPA-COG is an observational study evaluating the effect of exchange transfusions on cognitive function in adults with severe sickle cell disease (SS or Sβ0).
- Trial name
- DREPA-COG (NCT07168447)
- Where
- 6 sites in France
- Volunteers
- 85 people
- Sponsor
- Centre Hospitalier St Anne
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
Beta-Thalassemia
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity.
- Trial name
- NCT06568926
- Testing
- Serum Ferritin level
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Sohag University
Sickle Cell Disease
Mesenchymal Stromal Cells for Haplo Hematopoietic Cell Transplantation for Sickle Cell Disease
This trial is being conducted as a step toward testing the long-term hypothesis that freshly cultured, autologous mesenchymal stromal cells (MSCs) grown in platelet lysate-containing medium will modulate recipient T-cell immune…
- Trial name
- NCT03298399
- Testing
- Autologous MSCs
- Sponsor
- Emory University