Trial
NCT02986698
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
Phase ITERMINATEDOPERATIONALConfidence: HIGH
Sponsor
University of California, San Francisco
Collaborators
California Institute for Regenerative Medicine (CIRM)
Condition
Alpha Thalassemia Major
Intervention
in utero hematopoietic stem cell transplantation
Why stopped
Enrollment ended early due to interim data suggesting a lack of efficacy. Interim data did demonstrate that IUHSCT was safe and well tolerated, but the PI and DSMB agreed that enrollment should not continue under the current treatment protocol.
Provenance
Dataset: 2026-07-02 • Source: ClinicalTrials.gov API v2 • Last update: 2025-01-27