Single-Center, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Thalassemia Major

NCT#/Phase: NCT02986698
Phase 1
IRB#: 2017-083
Description: This study evaluates the safety for the maternal subject and the fetal subject of IUHCT for alpha-thalassemia major when performed concurrently with clinically indicated IUT.
Inclusion: Fetal Subject Inclusion Criteria: Male or female fetuses 18-25 weeks gestational age, diagnosed with alpha thalassemia major by either chorionic villus sampling (CVS), amniocentesis, or cordocentesis, or by identification of parents as genetic carriers and identification of fetal anemia, for whom parents elect to pursue IUT, and undergo subsequent IUT for the remainder of gestation.
Enrollment Status: Open for enrollment
Sponsor: UC San Francisco
Principal Investigator(s): Elliott Vichinsky, MD
Contact(s): Elliott Vichinsky, MD (510-428-3885 x3651)
Ashutosh Lal, MD
Study Link: