NCT05145062

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 8 (actual)
• Sponsor: Sangamo Therapeutics · Industry
• Sex: All
• Age: 18 Years – 45 Years
• Healthy volunteers: Not accepted

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

Detailed description

The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.

Conditions

• Blood and Lymphatic Diseases

Interventions

Type	Name	Description
DRUG	BIVV003	Solution for intravenous administration
DRUG	ST-400	Solution for intravenous administration

Timeline

• Start date: 2021-12-21
• Primary completion: 2038-07-14
• Completion: 2038-07-14
• First posted: 2021-12-06
• Last updated: 2025-11-12

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study