Blood and Lymphatic Diseases
Conditions
Keywords
Sickle Cell Disease (SCD), Beta-thalassemia (TDT)
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.
Interventions
DRUGBIVV003
Solution for intravenous administration
DRUGST-400
Solution for intravenous administration
Sponsors
Sangamo Therapeutics
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 45 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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) |
| Red blood cell transfusions | Up to 15 years | Number and total volume of red blood transfusions in the BIVV003 and ST-400 cohorts |
| 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 |
| 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 |
Countries
United States
Outcome results
None listed