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 Treatments for Sickle Cell Disease and Beta-Thalassemia

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 effectiveness of investigational treatments for people with severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Sickle cell disease is a condition where red blood cells, which carry oxygen, become misshapen and can block blood flow. Beta-thalassemia is a blood disorder that reduces the production of hemoglobin, leading to a lack of oxygen in many parts of the body. The study aims to understand how these investigational treatments affect the body over a long period.

Participants in this study will have previously received an investigational treatment in a prior study. They will undergo regular follow-ups to monitor the long-term effects of the treatment on their health. The study involves various procedures to check the safety and effectiveness of the treatments over time.

  • Who can participate: Participants must have received the investigational treatment in a prior study and be able to give informed consent.
  • Study details: Participants will be monitored over a long period to assess the safety and effectiveness of the investigational treatments. A placebo is not used in this study since all participants have already received the investigational treatment in previous studies.
  • Study Timelines: The study will last up to 15 years.

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