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 on Long-term Safety and Efficacy of Investigational Gene Therapy in Severe Blood Disorders

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.

Updated on 01 Feb 2022. Study ID: NCT05145062

This study investigates the long-term safety and efficacy of investigational gene therapy in individuals with severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Sickle cell disease is a condition where red blood cells become misshapen and can block blood flow, causing pain and other complications. Beta-thalassemia is a blood disorder that reduces the production of hemoglobin, leading to anemia. The study focuses on participants who have previously received treatment with investigational gene therapy products in other studies.

Participants in this observational study will undergo regular follow-ups to monitor the long-term effects of the investigational gene therapy. 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. This study will not involve any changes to the participants' usual treatment plans.

  • Who can participate: Participants must have received treatment with the investigational gene therapy in a previous study and be capable of providing informed consent.
  • Study details: Participants will be observed over a long period to assess the safety and effectiveness of the investigational gene therapy. No changes will be made to their existing treatment plans.
  • Study timelines: The study will last up to 15 years.

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