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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05145062
Recruitment Status : Enrolling by invitation
First Posted : December 6, 2021
Last Update Posted : December 19, 2022
Information provided by (Responsible Party):
Sangamo Therapeutics

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

Condition or disease Intervention/treatment
Blood and Lymphatic Diseases Drug: BIVV003 Drug: ST-400

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

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Study Type : Observational
Estimated Enrollment : 12 participants
Observational Model: Cohort
Time Perspective: Prospective
Official 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
Actual Study Start Date : December 21, 2021
Estimated Primary Completion Date : August 10, 2037
Estimated Study Completion Date : August 10, 2037

Group/Cohort Intervention/treatment
BIVV003 Cohort
All participants treated in parent and future studies with BIVV003
Drug: BIVV003
Solution for intravenous administration
Other Name: SAR445136

ST-400 Cohort
All participants treated in parent studies with ST-400
Drug: ST-400
Solution for intravenous administration

Primary Outcome Measures :
  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 Outcome Measures :
  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

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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05145062

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United States, Michigan
Karmanos Cancer Institute
Detroit, Michigan, United States, 48201
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Sangamo Therapeutics
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Study Director: Medical Monitor Sangamo Therapeutics
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Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT05145062    
Other Study ID Numbers: LTS16653
First Posted: December 6, 2021    Key Record Dates
Last Update Posted: December 19, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sangamo Therapeutics:
Sickle Cell Disease (SCD), Beta-thalassemia (TDT)
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Lymphatic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn