Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning — StellaPhase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.
Trial locations(4 sites)
United States
Mattel Children's Hospital - UCLA, Los Angeles, California Emory University/Childrens Healthcare of Atlanta, Atlanta, Georgia Boston Childrens Hospital, Boston, Massachusetts Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio Last updated December 2025