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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…
RH Genotype Matched Red Cell Transfusions for Patients With Sickle Cell Disease
To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.
A Phase 1/2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants With Sickle Cell Disease to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, pH and Food Effect, and Preliminary Efficacy of BMS-986470
The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BM…
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…
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has …
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…
Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening
Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-l…
A Phase 2 Randomized Double-blind, Placebo-controlled Clinical Trial of Intravenous Citrulline for Vaso-occlusive Pain Episode in Hospitalized Patients With Sickle Cell Disease (CONQUER SCD Pain Trial)
The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also…