Clinical trials: Sickle Cell Anemia
Thalassemia, Sickle Cell Anemia
Sickle Cell Anemia Screening and Prevention in Northern Israel
Since 1987, a screening for β Thalassemia in pregnant women is carried on in northern Israel, and from 1999 all the samples were tested also for Hgb S, Hgb C, Hgb D, Hgb O Arab and others.
- Trial name
- NCT00735488
- Testing
- Observational
- Where
- Israel
- Volunteers
- 69 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Graft Versus Host Disease
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant
This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents…
- Trial name
- ASCENT (NCT03924401)
- Testing
- Abatacept
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- Emory University
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle-cell Disease (SCD)
Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve…
- Trial name
- DREPAtient (NCT04413539)
- Where
- France
- Volunteers
- 1,088 people
- Sponsor
- Argo Sante
Sickle Cell Anemia
Serial Assessment of Fertility Experiences
The SAFE study is a long-term research project that watches people with sickle cell anemia (SCA) over time. The main goal is to see how a medicine called hydroxyurea affects their growth, puberty, and ability to have children.
- Trial name
- SAFE (NCT07116772)
- Where
- 3 sites in Dominican Republic, Jamaica and 1 more
- Volunteers
- 250 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
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
Sickle Cell Disease (SCD)
Self-administered Acupressure in Patients With Sickle Cell Disease
The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupressure analgesia in patients with sickle cell disease (SCD).
- Trial name
- NCT07875777
- Testing
- Verum Acupressure (using AcuWand)
- Where
- 2 sites in United States
- Volunteers
- 300 people
- Sponsor
- University of Cincinnati
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients With Pulmonary Arterial Hypertension (PAH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in sickle cell disease (SCD) patients diagnosed with pulmonary arterial hypertension.
- Trial name
- NCT00310830
- Testing
- Bosentan
- Volunteers
- 14 people
- Sponsor
- Actelion
Sickle Cell Trait, Sickle Cell Disease
Blood Sampling for Research Related to Sickle Cell Disease
This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease.
- Conditions
- Sickle Cell Trait, Sickle Cell Disease, Sickle Cell Anemia
- Trial name
- NCT00542230
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Disease
Sickle-cell Disease Registry of the GPOH
Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately.
- Trial name
- SichReg (NCT03327428)
- Where
- Germany
- Volunteers
- 1,000 people
- Sponsor
- University Hospital Heidelberg
Sickle Cell Disease Without Crisis
The Impact of Oxidative Stress on Erythrocyte Biology
This study will address if red blood cells transfused to a sickle cell patient from a donor with a glucose-6-phosphate-dehydrogenase (G6PD) enzyme deficiency have a different lifespan as measured by the percentage of red blood…
- Trial name
- RBC Survival (NCT04028700)
- Testing
- G6PD Deficient Red Blood Cell Transfusion, Non-G6PD deficient Red Blood Cell Transfusion
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
PK and PD Responses to Oral L-Citrulline in Patients With Sickle Cell Disease
To evaluate PK and PD responses to L-citrulline given orally for four weeks to patients with sickle cell disease who are otherwise healthy.
- Trial name
- NCT00343954
- Testing
- L-citrulline tablets, 1000 mg
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Angiogenix
Anemia, Sickle Cell
Folic Acid Supplementation in Children With Sickle Cell Disease
Folic acid supplementation (1mg/d) is the standard recommendation for Canadian children with Sickle cell disease (SCD), even though it can provide up to six times the recommended intake amount for healthy children.
- Trial name
- NCT04011345
- Testing
- Folic Acid Supplement
- Where
- Canada
- Volunteers
- 31 people
- Sponsor
- University of British Columbia
Hemoglobinopathy, Sickle Cell Disease
A Long-term Follow-up Study in Patients Who Received BEAM-101
This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing…
- Trial name
- NCT07373639
- Testing
- Long-Term Follow-up Study of patients who received BEAM-101
- Where
- 17 sites in United States
- Volunteers
- 50 people
- Sponsor
- Beam Therapeutics Inc.
Pulmonary Hypertension
Anticoagulation For Pulmonary Hypertension in Sickle Cell Disease
Sickle cell disease (SCD) is often referred to as a hypercoagulable state. However, the contribution of coagulation activation to the pathogenesis of SCD remains uncertain.
- Trial name
- NCT01036802
- Testing
- Warfarin
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of North Carolina, Chapel Hill
Priapism Due to Sickle Cell Disease
A Trial for Prevention of Recurrent Ischemic Priapism in Men With Sickle Cell Disease: A Pilot Study
To conduct a randomized controlled internal pilot feasibility trial for the prevention of recurrent ischemic priapism referred to as the Priapism in Nigeria (PIN) trial.
- Trial name
- PIN (NCT05142254)
- Testing
- Tadalafil, Hydroxyurea
- Where
- 2 sites in Nigeria
- Volunteers
- 64 people
- Sponsor
- Vanderbilt University Medical Center
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
Sickle Cell Disease, Thalassemia, Beta
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow.
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Trial name
- NCT05736419
- Testing
- Fludarabine, Cyclophosphamide, Tacrolimus
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- Memorial Sloan Kettering Cancer Center