Clinical trials: Hemoglobinopathy
Sickle Cell Disease
Evaluating Thromboelastography (TEG) and ETP in Sickle Adults
The primary aim of this study is to investigate the reported enhanced coagulation status (prothrombotic status) in patients with sickle cell disease using 2 laboratory tests; thromboelastography (TEG) and Endogenous Thrombin…
- Trial name
- NCT02770534
- Testing
- blood test
- Where
- United Kingdom
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or…
- Trial name
- NCT03207009
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
Lung Ultrasound in Pediatric Acute Chest Syndrome
Sickle-cell disease is a common disease with serious complications, in particular acute chest syndrome (ACS), which can be life threatening.
- Trial name
- Lupacs (NCT06497153)
- Testing
- lung ultrasound
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hydrops Fetalis, Fetal Growth Retardation
Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGR
The objective of this pilot study is to prospectively evaluate amniotic fluid of pregnancies complicated by non-immune hydrops and severe symmetrical intrauterine growth restriction by tandem mass spectrometry for inborn errors…
- Trial name
- TandemMS (NCT00143039)
- Testing
- Tandem MS test for inborn errors of metabolism
- Where
- 11 sites in United States
- Volunteers
- 48 people
- Sponsor
- Obstetrix Medical Group
Sickle Cell Disease, Sickle Cell Anemia
Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body.
- Trial name
- NCT04476277
- Testing
- Biotin label
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Vaso-occlusive Crisis
Outcomes of Vaso-occlusive Crisis in Pediatric Sickle Cell Disease
This study aims to assess the incidence and clinical outcomes of vaso-occlusive crises in pediatric patients with sickle cell disease. Data will be collected and analyzed to identify risk factors and related complications.
- Trial name
- NCT07200869
- Testing
- No intervention
- Where
- Egypt
- Volunteers
- 25 people
- Sponsor
- Assiut University
Anemia, Sickle Cell
Use of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease
The purpose of this study is to estimate the MTD of Mg pidolate in combination with HU in patients with sickle cell disease who have been on a therapeutic dose (15-30 mg/kg/day) of HU for at least 6 months.
- Trial name
- NCT00143572
- Testing
- Magnesium Pidolate, Hydroxyurea
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- St. Jude Children's Research Hospital
Anemia, Sickle Cell
Transfusion Treatment in Patients With SCD
The "National Transfusion Treatment Survey in patients with sickle cell disease (SCD)" is a prospective longitudinal systemic study that was created in order to evaluate the therapeutic approach, mainly transfusional, in patients…
- Trial name
- NCT03397017
- Testing
- Transfusion
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Società Italiana Talassemie ed Emoglobinopatie
Thalassemia
Endocrine and Nutritional Assessment in B Thalassemia Major
B Thalassemia patients developed short stature and low weight in spite no evident endocrine abnormalities. One hypothesis is that they developed some degree of malnutrition,then the purpose of this study is to assess the…
- Trial name
- NCT00456690
- Testing
- Nutritional recommendations
- Where
- Israel
- Volunteers
- 133 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease, Thalassemia
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who…
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy
- Trial name
- NCT03367546
- Testing
- Blood and Marrow Transplant
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Blood Disease, Anemia, Sickle Cell
Pediatrics:Chlamydia, Sickle Cell Anemia and Stroke Risk - Ancillary to STOP II
To establish a link among Chlamydia infection, sickle cell anemia, and stroke risk.
- Conditions
- Blood Disease, Anemia, Sickle Cell, Chlamydia Infections, Cerebrovascular Accident
- Trial name
- NCT00037388
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Pain Crisis
Natural History of Sickle Cell Disease
This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.
- Trial name
- NCT00081523
- Where
- 3 sites in United States
- Volunteers
- 3,500 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Thalassemia Major
Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major.
- Trial name
- AnloThal (NCT01125254)
- Testing
- Amlodipine
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- University of Campinas, Brazil
Sickle Cell Disease
Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor
Intro: Sickle cell disease is a genetic disorder caused by a mutation of the β hemoglobin called HbS, which causes red blood cell (RBC) abnormalities responsible for hemolysis, mainly intravascular, leading to chronic anemia.
- Trial name
- HEMOPROVE (NCT05199766)
- Testing
- Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Thalassemia
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Background: Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs.
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Trial name
- NCT02105766
- Testing
- Alemtuzumab, Sirolimus, Cyclophosphamide
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure, Hemoglobinopathies
Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases
This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation.
- Conditions
- Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia
- Trial name
- NCT07660783
- Testing
- ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
- Where
- 2 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease
Comparison of Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease
Although the survival of children with sickle cell disease (SCD) has dramatically improved over the last decades in the US and Europe, mortality remains high in adults.
- Trial name
- DREPA-RIC (NCT04046705)
- Testing
- Allogeneic matched related haematopoietic stem cell transplantation, Standard arm
- Volunteers
- 78 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
Clonidine With Morphine in Patient Controlled Analgesia Pump in Vaso-Occlusive Crisis in Sickle Cell Disease Patient
Vaso-occlusive crisis are highly painful in Sickle-cell patients. Morphine is the treatment of choice for this pain. Various adjuncts have been studied for the treatment of vaso-occlusive crisis.
- Trial name
- NCT05848531
- Testing
- Clonidine
- Where
- Belgium
- Volunteers
- 40 people
- Sponsor
- Centre Hospitalier Universitaire Saint Pierre
Beta Thalassemia
Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia
The current study is to investigate the potential roles of N-acetyl cysteine and Alpha-lipoic acid in patients with beta-thalassemia.
- Trial name
- NAC/ALA (NCT07157722)
- Testing
- Iron chelating intervention, N Acetyl cysteine 600mg, Alpha Lipoic Acid 600 MG Oral Tablets
- Where
- Egypt
- Volunteers
- 66 people
- Sponsor
- Tanta University
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University