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Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Background: Fanconi anemia (FA) is an inherited disorder. People with FA are more likely to get certain cancers, especially squamous cell carcinoma (SCC). The…
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…
Trial of Darbepoetin Plus Slow-release Intravenous Iron to Decrease Transfusions and Improve Iron Status and Neurodevelopment in Preterm Infants
In this phase II trial, the investigators overarching goal is to demonstrate the feasibility and potential benefit of darbepoetin (Darbe) plus slow-release int…
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…
A Phase 2, Multicenter, Open-Label Study of Zolacabtagene Autoleucel (BMS-986353), CD19-Targeted NEX-T CAR T Cells, in Participants With Chronic Immune Thrombocytopenia (cITP) and Autoimmune Hemolytic Anemia (AIHA)
The purpose of this study is to evaluate the safety and efficacy of Zola-cel (BMS-986353), in participants with chronic immune thrombocytopenia (cITP) and auto…
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…
Let's Get REAL: A Pilot Trial of a Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasi…
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment o…
Better Birth Outcomes and Experiences Through Technology, Education, and Reporting (BETTER)
This is a pragmatic randomized control trial to evaluate the BETTER intervention compared to standard obstetrical care (control) to determine whether it helps …
A Socio-ecological Approach for Improving Self-management in Adolescents With SCD
The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behavi…
Iron Replacement to Reduce Anemia During Neoadjuvant Chemotherapy
This is an open-label, single institution study evaluating the efficacy of intravenous iron sucrose monotherapy in patients with thoracic, breast, GU and GI ma…
A Phase 1, Open-label, Single-arm Study of APR 2020 in Transfusion Dependent, Steroid Resistant Pediatric and Adolescent Subjects With RPS19 Deficient Diamond Blackfan Anemia by Transplantation of Autologous CD34+ Stem Cells Transduced With CLIN LV EFS coRPS19 PRE* (APR-2020)
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. Th…