Clinical trials: Congenital Hemolytic Anemia
Sickle Cell Disease
Testing the Sickle Cell Caregiver Collaboration for Child Development (SCCCD) Intervention
Sickle cell disease affects 100,000 people and 2,000 newborns each year; 50% of these children have a developmental deficit (>2 SD) before the age of 3.
- Trial name
- SCCCD (NCT06562439)
- Testing
- Sickle Cell Collaboration for Child Development
- Where
- United States
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle…
- Trial name
- NCT04443907
- Testing
- OTQ923
- Where
- 3 sites in United States
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Beta Thalassemia Major
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
- Trial name
- NCT03947632
- Testing
- serum ferritin and hemoglobin test
- Volunteers
- 367 people
- Sponsor
- Aga Khan University
Sickle Cell Disease
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine).
- Trial name
- ASCENT1 (NCT05405114)
- Testing
- NDec - oral decitabine-tetrahydrouridine, HU - Hydroxyurea
- Where
- 118 sites in United States, Canada and 12 more
- Volunteers
- 96 people
- Sponsor
- Novo Nordisk A/S
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)
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
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years…
- Trial name
- ZnSCD (NCT06260891)
- Testing
- 25 mg/day zinc, 40 mg/day zinc
- Where
- 6 sites in United States
- Volunteers
- 34 people
- Sponsor
- University of California, San Francisco
Thalassemia
The Role of OCTA in Patients Affected by Beta Thalassemia
To investigate, using optical coherence tomography angiography (OCTA), the retinal and choriocapillaris vascular features in patients affected by Beta Thalassemia.
- Trial name
- NCT04582110
- Testing
- Optical coherence tomography angiography
- Where
- Italy
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- Federico II University
Transfusional Iron Overload, Iron Overload
Treatment of Iron Overload Requiring Chelation Therapy
The purpose of this open-label study is to assess liver iron concentration using MRI imaging in subjects with beta-thalassemia when administered with either SPD602 or deferasirox for the treatment of chronic transfusional iron…
- Conditions
- Transfusional Iron Overload, Iron Overload, Iron Chelation, Beta-thalassemia
- Trial name
- NCT01927913
- Testing
- SPD602, Deferasirox
- Sponsor
- Shire
Anemia, Sickle Cell
Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the…
- Trial name
- NCT01054768
- Testing
- alpha-lipoic acid and acetyl-L-carnitine, Control
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Β-thalassemia
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia.
- Trial name
- NCT06655662
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- 3 sites in China
- Volunteers
- 8 people
- Sponsor
- Shenzhen Hemogen
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
Data Collection, Sickle Cell Disease
Data Collection For Patients With Sickle Cell Disease
To create a database of clinical information, participant /disease characteristics, and treatment history collected from patients with Sickle Cell Disease.
- Trial name
- NCT07911618
- Testing
- data collection
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- M.D. Anderson Cancer Center
Sickle Cell Disease
An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia
This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102). The open-label extension study will evaluate long-term safety and tolerability.
- Trial name
- NCT04053803
- Testing
- IMR-687
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 30 people
- Sponsor
- Imara, Inc.
Beta Thalassemia
Genetic Factors Affecting the Severity of Beta Thalassemia
Patients with genetic diseases can have widely differing severities. We are looking for genetic factors which influence the severity of beta thalassemia.
- Trial name
- NCT00159042
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- Deborah Rund
Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease
This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD.
- Trial name
- NCT02140554
- Testing
- lovo-cel
- Where
- 11 sites in United States
- Volunteers
- 54 people
- Sponsor
- Genetix Biotherapeutics Inc.
Beta-Thalassemia
Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major
Beta thalassemia major is a serious genetic disease of the blood. Treatments are limited, and although a bone marrow transplant from a compatible donor can be curative, only a limited percentage of individuals with this disease…
- Trial name
- NCT00336362
- Testing
- Hydroxyurea
- Where
- Greece
- Volunteers
- 26 people
- Sponsor
- University of Washington
Sickle Cell Disease, Pain
Validation of the Sickle Cell Disease Pain Burden Interview
The purpose of this study is to validate a brief survey tool, the SCD Pain Burden Interview (SCPBI), which can be used in the clinical and/or research settings to assess the impact of pain on children with sickle cell disease.
- Trial name
- NCT00804362
- Where
- United States
- Volunteers
- 131 people
- Sponsor
- Connecticut Children's Medical Center
Sickle Cell Disease, Stem Cell Transplant Complications
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a…
- Conditions
- Sickle Cell Disease, Stem Cell Transplant Complications, Red Blood Cell Disorder, Pure Red Cell Aplasia
- Trial name
- Sickle-AID (NCT03214354)
- Testing
- Alemtuzumab, Total Body Irradiation, Sirolimus
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- University of Calgary
Sickle Cell Trait, Sickle Cell Disease (SCD)
Sickle Cell Kidney Biorepository
Kidney disease is a major cause of illness and death in people with sickle cell disease and sickle cell trait. Despite these concerning facts, we do not (1) have an in-depth understanding of how kidney disease starts in sickle…
- Conditions
- Sickle Cell Trait, Sickle Cell Disease (SCD), Chronic Kidney Disease
- Trial name
- SCeK (NCT07064174)
- Where
- 2 sites in United States
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
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