Gene therapy patients monitored for years to ensure lasting safety

NCT ID NCT05145062

Ongoing Disease control Sponsor: Sangamo Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 8 people who received a one-time gene therapy (BIVV003 for sickle cell disease or ST-400 for beta-thalassemia) to see if it remains safe and effective over many years. Researchers will track serious side effects, survival, and blood markers like hemoglobin levels. Participants must have already received the therapy in an earlier study.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major hematologic disorder lymphatic system disorder sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BLOOD AND LYMPHATIC DISEASES

Contacts and locations

Locations

Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States
Henry Ford Health System

Detroit, Michigan, 48202, United States
Karmanos Cancer Institute

Detroit, Michigan, 48201, United States
UCSF Benioff Children's Hospital

Oakland, California, 94609, United States
University of California Davis Health System

Sacramento, California, 95817, United States
University of Minnesota

Minneapolis, Minnesota, 55455, United States

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