autologous cd34-positive bcl11a-disrupted hematopoietic progenitor cells bivv003
autologous cd34-positive bcl11a-disrupted hematopoietic progenitor cells bivv003: 2 clinical trials tracked.
autologous cd34-positive bcl11a-disrupted hematopoietic progenitor cells bivv003 evidence translation
What we can confirm
2 tracked trials
What it means
What to watch
Prepared research question
What is verified about autologous cd34-positive bcl11a-disrupted hematopoietic progenitor cells bivv003, and what remains unknown?
Traceable citations · Unknowns marked
See linked entities and source evidence1 indications · 1 study sponsors
Related indications
Study sponsors
Clinical Trial Registry Records (2)
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 endpoint: Adverse Events
A Phase 1/2, Open-Label, Multicenter, Single-Arm Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
Primary endpoint: Percentage of Participants who are Alive at Post-transplantation Day 100