Clinical trials: Sickle Cell Disorders
Sickle Cell Disease, Thalassemia
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.
- Trial name
- NCT02504619
- Testing
- CordIn
- Where
- 3 sites in United States, France
- Volunteers
- 1 people
- Sponsor
- Gamida Cell ltd
Anemia, Sickle Cell, Sickle Cell Disease
Hydroxyurea and EPO in Sickle Cell Disease
The proposed study is a Phase 1/2 multi-center study evaluating the safety and efficacy of erythropoietin (EPO) in combination with hydroxyurea in the treatment of chronic anemia in patients with sickle cell disease (SCD).
- Trial name
- ACHiEvE-SCD (NCT05451940)
- Testing
- Hydroxyurea, Epoetin Alfa-BioSimilar
- Where
- 2 sites in United States, Nigeria
- Volunteers
- 17 people
- Sponsor
- Julia Xu
Sickle Cell Disease
Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease
The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care.
- Trial name
- iTransition (NCT02200510)
- Testing
- Self-management intervention for Adolescents with SCD, Patient Portal Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet assessments to identify type, intensity, frequency, and phase of pain among SCD adolescents is lacking.
- Trial name
- NCT03291613
- Testing
- Pinpoint App
- Where
- 2 sites in United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
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
Sickle Cell Anemia, Severe Aplastic Anemia
Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood.
- Conditions
- Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
- Trial name
- NCT00004143
- Testing
- Campath, Chemo and/or TBI Allo SCT
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- David Rizzieri, MD
Sickle Cell Anemia
Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia
The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA).
- Trial name
- NCT02961218
- Testing
- ACZ885
- Where
- 15 sites in United States, Canada and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or…
- Trial name
- NCT03020615
- Testing
- Hydroxyurea
- Where
- 4 sites in United States
- Volunteers
- 58 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Anemia
Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE)
The primary goal of the Phase III SCATE trial is to compare 30 months of alternative therapy (hydroxyurea) to standard care (observation) in children with sickle cell anemia and conditional (170 - 199cm/sec) Transcranial Doppler…
- Trial name
- SCATE (NCT01531387)
- Testing
- Hydroxyurea
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 38 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Acute Chest Syndrome, Sickle Cell Disease
Feasibility Study of Unfractionated Heparin in Acute Chest Syndrome
The purpose of this study is to determine the feasibility of performing a larger multicenter phase III trial to assess the effects of unfractionated heparin (UFH) in acute chest syndrome (ACS).
- Trial name
- NCT02098993
- Testing
- Unfractionated heparin
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Craig Seaman
Sickle Cell Disease
Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)
This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care.
- Trial name
- STRIDE2 (NCT02766465)
- Testing
- Busulfan, Fludarabine, r-ATG
- Where
- 35 sites in United States
- Volunteers
- 138 people
- Sponsor
- Medical College of Wisconsin
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
Sickle Cell Disease
Development and Adaptation of I-STRONG for SCD
This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.
- Trial name
- NCT06110754
- Testing
- I-STRONG for SCD
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- Emory University
Sickle Cell Disease
Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease
This is a randomized, double-blind, placebo-controlled study in sickle cell disease participants with a history of Vaso-occlusive Crises (VOCs).
- Trial name
- NCT05098028
- Testing
- Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 44 people
- Sponsor
- Bausch Health Americas, Inc.
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
Stroke (CVA) or TIA, Diabetes
Risk Factors, Costs, and Impacts of ED Boarding
The goal of this observational study is to learn about the risk factors, costs, and operational impacts of emergency department boarding (patients admitted to the hospital but remaining in the emergency department awaiting…
- Conditions
- Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease)
- Trial name
- NCT07532564
- Where
- United States
- Volunteers
- 30,486 people, healthy volunteers welcome
- Sponsor
- University of Maryland, Baltimore
Sickle Cell Disease (SCD)
Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease
The goal of the study was to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle…
- Trial name
- STEADFAST (NCT04053764)
- Testing
- Crizanlizuamb, Standard of Care
- Where
- 24 sites in United States, Brazil and 9 more
- Volunteers
- 58 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia, Stroke
Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial
The goal of this study is to determine the effectiveness of blood transfusion therapy for prevention of silent cerebral infarct (stroke) in children with sickle cell anemia.
- Trial name
- SIT (NCT00072761)
- Testing
- transfusion therapy
- Where
- United States
- Volunteers
- 196 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Disease
Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial
The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.
- Trial name
- STArT (NCT04839354)
- Testing
- L-Arginine Hydrochloride, Normal saline
- Where
- 10 sites in United States
- Volunteers
- 271 people
- Sponsor
- Claudia R. Morris
Sickle Cell Disease
iCanCope With Sickle Cell Pain
The project will test a tailored web and smartphone-based application (iCanCope with SCD) to improve pain self-management and functioning in youth (aged 12-18) with sickle cell disease.
- Trial name
- NCT03201874
- Testing
- Pain self-management, Education
- Where
- 4 sites in United States, Canada
- Volunteers
- 137 people
- Sponsor
- Seattle Children's Hospital