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12 recruiting studies matching your filters
High Sensitivity Screening of Compound Libraries to Discover a Drug for the Treatment of 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…
A Multi-Center, Randomized, Controlled, Cross-Over Study to Evaluate the Effectiveness of Hypoxic Red Blood Cells Processed With the Hemanext ONE® System Versus Conventional Red Blood Cells in Patients With Transfusion Dependent Sickle Cell Anemia
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE syste…
Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease
The goal of this interventional study is to learn about the impact of an intervention for health care providers that teaches individuation and perspective-taki…
Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin
Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA. The Food and Drug Administr…
Verifying Antibodies After Live Immunization Delivery (VALID): a Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease
The goal of this study is to learn if infants with sickle cell disease (SCD) develop adequate protection after measles vaccines. (not looking at any prolonged …
T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may…
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 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…
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 …
Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation
The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation…
An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic materia…
A Phase I Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life…