Clinical trials: Sickle Cell Disorder
Sickle Cell Anemia
Voxelotor Sickle Cell Exercise Study
This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older…
- Trial name
- NCT04581356
- Testing
- Voxelotor
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Elizabeth Yang, MD, PhD
Sickle Cell Disease
Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease
Propanolol is a beta blocker which has been found to inhibit the ability of epinephrine to upregulate sickle red cell adhesion to laminin and endothelial cells in vitro.
- Trial name
- NCT02012777
- Testing
- propranolol
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Miami
Sickle Cell Disease, Genetic Disease
Implementing an Individualized Pain Plan (IPP) for ED Treatment of VOE's in Sickle Cell Disease
The overall purpose of this proposed study is to improve management of vaso-occlusive episodes (VOEs) in adult EDs. We aim to implement NHLBI recommendations for VOE treatment by embedding Individualized Pain Plans (IPPs) in the…
- Conditions
- Sickle Cell Disease, Genetic Disease, Hematologic Diseases, Anemia, Sickle Cell
- Trial name
- ALIGN (NCT04584528)
- Testing
- Electronic Health Record (EHR) Embedded Individualized Pain Plan (IPP)
- Where
- 8 sites in United States
- Volunteers
- 279 people, healthy volunteers welcome
- Sponsor
- Duke University
Analyze the Results of Cardiorespiratory Stress Tests, Resting and Stress Echocardiography
Exploration of the Parameters Influencing the Effort Limitation of Patients Suffering From Homozygous Sickle Cell Anemia
The cardiovascular complications of sickle cell disease are associated with major morbidity and mortality, as well as impaired quality of life.
- Conditions
- Analyze the Results of Cardiorespiratory Stress Tests, Resting and Stress Echocardiography, Capillary Lactatemia Measurements at the Ear
- Trial name
- PHYSIO-EXDRE (NCT06743139)
- Testing
- Echography
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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.
Sickle Cell Disease
Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD)
An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell patients who are either homozygous for Hb S or have HbSB0 thalassemia.
- Trial name
- NCT01077921
- Testing
- Propranolol
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Laura M. De Castro, MD
Sickle Cell Disease
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
- Trial name
- SIKAMIC (NCT03806452)
- Testing
- Hydroxycarbamide
- Where
- 21 sites in Côte d’Ivoire, France and 4 more
- Volunteers
- 86 people
- Sponsor
- Theravia
Sickle Cell Disease, Sickle Cell Anemia
Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease
Sickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape.
- Trial name
- NCT01000155
- Testing
- vorinostat
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Dana-Farber Cancer Institute
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell
Diastolic Dysfunction in Sickle Cell Disease During Vaso-occlusive Crisis
This study is designed to investigate the presence and absence of abnormal heart muscle contractions during sickle cell crises. You will be asked to do echocardiography during and after your admission to the hospital.
- Trial name
- NCT01683968
- Where
- Saudi Arabia
- Volunteers
- 216 people
- Sponsor
- King Abdullah International Medical Research Center
Sickle Cell Disease
Dense Red Blood Cells in Sickle Cell Children
Quantitative and prognostic evaluation of dense red blood cells in sickle cell children: preliminary single center study from the Creteil pediatric cohort.
- Trial name
- DREPADENSE (NCT02887118)
- Where
- France
- Volunteers
- 82 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Transfusion-dependent Hemachromatosis, Thalassemia Major
Pilot Study for Patients With Poor Response to Deferasirox
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron…
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Trial name
- NCT00749515
- Testing
- Deferoxamine, Deferasirox, HIDA
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease
A Phase Ib Study of NVX-508 in Sickle Cell Disease
This Phase 1b study in adults with sickle cell disease (SCD) in steady-state (non-acutely ill) aims to evaluate safety and toxicity of NVX-508 in a multi-dosing paradigm as well as to determine the maximum tolerated dose (MTD) in…
- Trial name
- NCT03013426
- Testing
- NVX-508
- Sponsor
- Amma Owusu-Ansah, MD
Avascular Necrosis of Bone, Sickle Cell Disease
Autologous Bone Marrow Stem Cell Transplantation for Osteonecrosis in Sickle Cell Disease
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cells in sickle cell disease patients with osteonecrosis
- Trial name
- NCT02448121
- Testing
- Autologous bone marrow stem cell graft
- Volunteers
- 100 people
- Sponsor
- Federal University of Bahia
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
LCI-HEM-SCD-ST3P-UP-001: The Sickle Cell Trevor Thompson Transition Project (ST3P-UP Study)
This multi-center study will compare the effectiveness of adding virtual peer mentoring (PM) to a structured education-based (STE) transition program for emerging adults with sickle cell disease to determine its effect on…
- Trial name
- ST3P-UP (NCT03593395)
- Testing
- Peer Mentoring [PM], Structured Education Based Transition Program STE
- Where
- 14 sites in United States
- Volunteers
- 291 people
- Sponsor
- Wake Forest University Health Sciences
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, Blood Disease
Prevention of Cerebral Infarction in Sickle Cell Anemia - Comprehensive Sickle Cell Center
To conduct a prospective study aimed at the early detection and treatment of cerebral vascular disease prior to irreversible brain injury in young children with sickle cell anemia (SCA).
- Conditions
- Anemia, Sickle Cell, Blood Disease, Cerebrovascular Disorders, Cerebrovascular Accident
- Trial name
- NCT00005327
- Sponsor
- University of Southern California
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