An Observational Long-term Safety and Efficacy Follow-up Study After Ex-vivo Gene Therapy With BIVV003 in Participants With Severe Sickle Cell Disease (SCD) or With ST-400 in Participants With Transfusion-dependent Beta-thalassemia (TDT) With Autologous Hematopoietic Stem Cell Transplant

Study Summary

Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: * Long-term efficacy of the biological treatment effect of BIVV003 in SCD * Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events * Long-term efficacy of the biological treatment effect of ST-400 in TDT * Long-term efficacy of the clinical treatment effect of ST-400 in TDT

Interventions

Study Locations

Facility	City	State	Country
UCSF Benioff Children's Hospital	Oakland	California	United States
University of California Davis Health System	Sacramento	California	United States
Children's Healthcare of Atlanta	Atlanta	Georgia	United States
Boston Children's Hospital	Boston	Massachusetts	United States
Karmanos Cancer Institute	Detroit	Michigan	United States
Henry Ford Health System	Detroit	Michigan	United States
University of Minnesota	Minneapolis	Minnesota	United States

Official Trial Information

View on ClinicalTrials.gov