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NCT Number: NCT05145062

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Blood and Lymphatic Diseases Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Lymphatic Diseases Thalassemia beta-Thalassemia

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Observational

Primary location

UCSF Benioff Children's Hospital, Oakland, California, United States

Loading trial locations.

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

BIVV003

Drug

Solution for intravenous administration

Other names: SAR445136

ST-400

Drug

Solution for intravenous administration

Primary outcomes

  1. 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

  2. Overall Survival

    Time frame: Up to 15 years

    Duration from first dose of study medication to death

Secondary outcomes

  1. 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)

  2. 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

  3. 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

  4. 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

  5. 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

Sponsors and collaborators

Lead sponsor

Sangamo Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2038
Study completion
2038
First posted
Dec 6, 2021
Registry last updated
Nov 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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