An Observational Long-term Safety and Efficacy Follow-up Study After Ex-vivo Gene Therapy With BIVV003 in Severe Sickle Cell Disease (SCD) and ST-400 in Transfusion-dependent Beta-thalassemia (TDT) With Autologous Hematopoietic Stem Cell Transplant
Study Details
Study Description
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 | Phase |
|---|---|---|
Detailed Description
The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| BIVV003 Cohort All participants treated in parent and future studies with BIVV003 |
Drug: BIVV003
Solution for intravenous administration
Other Names:
|
| ST-400 Cohort All participants treated in parent studies with ST-400 |
Drug: ST-400
Solution for intravenous administration
|
Outcome Measures
Primary Outcome Measures
- 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
- 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
Eligibility Criteria
Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Investigational Site Number :8400001 | Detroit | Michigan | United States | 48201 |
Sponsors and Collaborators
- Sangamo Therapeutics
Investigators
- Study Director: Medical Monitor, Sangamo Therapeutics
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- LTS16653