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4 recruiting studies matching your filters
Phase 1
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…
RPS19 Deficient Diamond-Blackfan Anemia
Apriligen, Inc.NCT07476183
Phase 1
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…
Sickle Cell AnemiaBeta-thalassemia MajorDiamond-blackfan Anemia
Paul SzabolcsNCT03653338
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may recei…
Acute LeukemiaAdenomatous PolyposisAdrenocortical Carcinoma+43 more
St. Jude Children's Research HospitalNCT03050268
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…
Bone Marrow Failure SyndromesErythrocyte DisorderLeukocyte Disorder+10 more
St. Jude Children's Research HospitalNCT02720679