Browse Studies — Arctuva

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

Estimated

~$10 – $7,500

Paul SzabolcsNCT03653338

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

Estimated

~$10 – $8,750

Apriligen, Inc.NCT07476183

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

Estimated

~$10 – $750

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

Estimated

~$10 – $750

St. Jude Children's Research HospitalNCT02720679