Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
- Conditions
- Blood and Lymphatic Diseases
- Interventions
- Registration Number
- NCT05145062
- Lead Sponsor
- 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
- Detailed Description
The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 12
- 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)
- 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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description ST-400 Cohort ST-400 All participants treated in parent studies with ST-400 BIVV003 Cohort BIVV003 All participants treated in parent and future studies with BIVV003
- Primary Outcome Measures
Name Time Method Adverse Events 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
Overall Survival Up to 15 years Duration from first dose of study medication to death
- Secondary Outcome Measures
Name Time Method Change in hemoglobin levels 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)
Change in hemolysis markers 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
Frequency of severe vaso-occlusive crises 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
Frequency and severity of SCD-related clinical events Up to 15 years Percentage of participants with SCD-related clinical events (e.g., acute renal failure, acute stroke) in the BIVV003 cohort
Red blood cell transfusions Up to 15 years Number and total volume of red blood transfusions in the BIVV003 and ST-400 cohorts
Trial Locations
- Locations (7)
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
Karmanos Cancer Institute
🇺🇸Detroit, Michigan, United States
Henry Ford Health System
🇺🇸Detroit, Michigan, United States
University of California Davis Health System
🇺🇸Sacramento, California, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States