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

Research Study of Long-term Safety and Efficacy After Gene Therapy in Severe Sickle Cell Disease and Transfusion-dependent Beta-thalassemia

Not Recruiting
18 years - 45 years
All
Phase N/A
13 participants needed
1 Location

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: 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.

This study investigates the long-term safety and efficacy of an investigational gene therapy in individuals with severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Beta-thalassemia is a genetic disorder that affects the body's ability to produce normal hemoglobin, often requiring regular blood transfusions.

The study involves observing participants who have previously received the investigational gene therapy, specifically BIVV003 for SCD and ST-400 for TDT, to assess long-term effects. An observational study is a type of research study where data is collected by observing participants receiving routine care, without changing their treatment, which may focus on people using specific medications or having certain conditions to better understand how treatments work.

  • Who can participate: Participants who have received treatment with BIVV003 or ST-400 in previous related studies are eligible. They must be capable of giving informed consent. Participants unable to comply with study procedures or deemed unsuitable by the Investigator are excluded.
  • Study details: Participants will be observed over a long period to assess the safety and effectiveness of the investigational gene therapy they received earlier. This involves routine follow-up and monitoring without altering their treatment.
  • Study timelines: The study will last up to 15 years.
Updated on 10 Feb 2025. Study ID: NCT05145062