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A Feasibility Study of Alendronate as Treatment for Osteonecrosis in Adults With Sickle Cell Disease
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
A Phase 1b Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants With Sickle Cell Disease
This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerabilit…
Arginine Therapy for the Treatment of Vaso-Occlusive Events in Children With Severe Sickle Cell Disease
The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful event…
Use of Point-of-Care Ultrasound (POCUS) for the Diagnosis of Acute Chest Syndrome in Patients With Sickle Cell Disease
Feasibility and reliability of ultrasound in the inpatient hematology setting.
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.
The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) an…
The Cognitive-Remediation of Executive and Adaptive Deficits in Youth (C-READY) Intervention: A Randomized Controlled Trial for Adolescents With Sickle Cell Disease to Prepare for Transition of Care
Randomized Controlled Trial (RTC) testing the efficacy of a telehealth adaptation of the Cognitive-Remediation of Executive and Adaptive Deficits in Youth (C-R…
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will eval…
Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide
This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the…
Feasibility and Efficacy of Attentional-Control Training in Sickle Cell Disease
Children with sickle cell disease (SCD) exhibit significantly reduced cognitive functioning (often difficulties with attention) compared to peers and siblings …
Predictors of Pain Severity and Pain-Related Outcomes in Individuals With Sickle Cell Disease
Sickle cell disease is a painful inherited disorder that affects approximately 100,000 people in the United States, and more than half of these individuals dev…
St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this ge…
Phase I Study to Determine the Safety and Tolerability of Escalating Doses of Panobinostat (LBH589) in Patients With Sickle Cell Disease
The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell diseas…