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

Study Overview

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

Study details

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

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Blood and Lymphatic Diseases
  • Age: Between 18 Years - 45 Years
  • Gender: All

Inclusion Criteria:
  • Received treatment with BIVV003 or ST-400 in one of the parent studies (ACT16222, ST- 400-01) or any future studies with BIVV003
  • Capable of giving signed informed consent (and if applicable assent)
Exclusion Criteria:
  • Unable to comply with study visit schedule or study procedures
  • Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the participant unsuitable for participation in the study The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Updated on 09 Mar 2024. Study ID: NCT05145062