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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
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
The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.
Age
18 - 45 years
Sex
ALL
Healthy Volunteers
No
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
Start Date
December 21, 2021
Primary Completion Date
July 14, 2038
Completion Date
July 14, 2038
Last Updated
November 12, 2025
8
ACTUAL participants
BIVV003
DRUG
ST-400
DRUG
Lead Sponsor
Sangamo Therapeutics
Data Source & Attribution
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Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
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