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

NCT05145062 | Active Not Recruiting FDA Drug

• 1 other identifier: LTS16653
• Study Type: observational
• Target: 8
• Locations: 1 country
• Sites: 7

Brief Summary

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

Conditions

Blood and Lymphatic Diseases

Keywords

Sickle Cell Disease (SCD), Beta-thalassemia (TDT)

Outcome Measures

Primary Outcomes (2)

• Adverse Events

  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

  Duration from first dose of study medication to death

  Up to 15 years

Secondary Outcomes (5)

• Change in hemoglobin levels

  Up to 15 years

• Change in hemolysis markers

  Up to 15 years

• Frequency of severe vaso-occlusive crises

  Up to 15 years

• Frequency and severity of SCD-related clinical events

  Up to 15 years

• Red blood cell transfusions

  Up to 15 years

Study Arms (2)

BIVV003 Cohort

All participants treated in parent and future studies with BIVV003

Drug: BIVV003

ST-400 Cohort

All participants treated in parent studies with ST-400

Drug: ST-400

Interventions

BIVV003 DRUG

Solution for intravenous administration

Also known as: SAR445136

BIVV003 Cohort

ST-400 DRUG

Solution for intravenous administration

ST-400 Cohort

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• 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.

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sangamo Therapeutics: lead

Study Sites (7)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Location

University of California Davis Health System

Sacramento, California, 95817, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Lymphatic Diseases; Anemia, Sickle Cell; beta-Thalassemia

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Thalassemia

Study Officials

• Medical Monitor

  Sangamo Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 22, 2021
• First Posted: December 6, 2021
• Study Start: December 21, 2021
• Primary Completion (Estimated): July 14, 2038
• Study Completion (Estimated): July 14, 2038
• Last Updated: November 12, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)