Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

November 7, 2025 updated by: Sangamo Therapeutics

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 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 Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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

Study Type

Observational

Enrollment (Actual)

8

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital
      • Sacramento, California, United States, 95817
        • University of California Davis Health System
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Children's Healthcare of Atlanta
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Karmanos Cancer Institute
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health System
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years to 41 years (Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All participants who received BIVV003 in one of the primary parent or future studies or ST-400 in the ST-400-01 parent study and agree to participate.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
BIVV003 Cohort
All participants treated in parent and future studies with BIVV003
Drug: BIVV003
Solution for intravenous administration
Other Names:
  • SAR445136
ST-400 Cohort
All participants treated in parent studies with ST-400
Drug: ST-400
Solution for intravenous administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events
Time Frame: Up to 15 years
Number of participants with serious adverse events and adverse events related to BIVV003 or ST-400, including new malignancy, new incidence or exacerbation of neurologic or rheumatologic or autoimmune or hematologic disorder, or new incidence of infection potentially related to BIVV003 or ST-400
Up to 15 years
Overall Survival
Time Frame: Up to 15 years
Duration from first dose of study medication to death
Up to 15 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in hemoglobin levels
Time Frame: Up to 15 years
Long-term change in levels of hemoglobin F, hemoglobin S and total hemoglobin (BIVV003 cohort), long term change in levels of hemoglobin F and total hemoglobin (ST-400 cohort)
Up to 15 years
Change in hemolysis markers
Time Frame: Up to 15 years
Long-term change in markers of hemolysis, including reticulocyte count, lactate dehydrogenase, haptoglobin, and serum bilirubin, over time in the BIVV003 cohort
Up to 15 years
Frequency of severe vaso-occlusive crises
Time Frame: Up to 15 years
Percentage of participants with severe vaso-occlusive crises, including acute pain crisis, acute chest syndrome, priapism, and splenic sequestration, in the BIVV003 cohort
Up to 15 years
Frequency and severity of SCD-related clinical events
Time Frame: Up to 15 years
Percentage of participants with SCD-related clinical events (e.g., acute renal failure, acute stroke) in the BIVV003 cohort
Up to 15 years
Red blood cell transfusions
Time Frame: Up to 15 years
Number and total volume of red blood transfusions in the BIVV003 and ST-400 cohorts
Up to 15 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sangamo Therapeutics

Investigators

  • Study Director: Medical Monitor, Sangamo Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 21, 2021

Primary Completion (Estimated)

July 14, 2038

Study Completion (Estimated)

July 14, 2038

Study Registration Dates

First Submitted

November 22, 2021

First Submitted That Met QC Criteria

November 22, 2021

First Posted (Actual)

December 6, 2021

Study Record Updates

Last Update Posted (Actual)

November 12, 2025

Last Update Submitted That Met QC Criteria

November 7, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LTS16653

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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