CIRM Funded Clinical Trials

In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major


Tippi MacKenzie
Disease Area: 
Fetal Alpha Thalassemia Major
Investigator:
CIRM Grant:
Award Value:
$12,131,817
Trial Stage: 
Phase 1
Trial Status: 
Not yet recruiting
Targeted Enrollment:
10
ClinicalTrials.gov ID:
Details: 

Untreated alpha thalassemia major is almost universally fatal in utero.  Current treatment requires in utero blood transfusions and monthly blood transfusions for life or a bone marrow transplant if a suitable donor is identified. The proposed treatment is a maternal bone marrow transplant in utero that takes advantage of maternal-fetal immune tolerance, and may provide a definitive cure. 

Design: 

Single arm, non-randomised study.

Goal: 

Safety and feasibility, efficacy.

Status: 

Patient recruitment initiating.