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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:
The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BIVV003 Cohort | All participants treated in parent and future studies with BIVV003 |
| |
| ST-400 Cohort | All participants treated in parent studies with ST-400 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BIVV003 | Drug | Solution for intravenous administration |
|
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events | 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 | Up to 15 years |
| Overall Survival | Duration from first dose of study medication to death | Up to 15 years |
| Measure | Description | Time Frame |
|---|---|---|
| Change in hemoglobin levels | 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) | Up to 15 years |
| Change in hemolysis markers |
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Inclusion Criteria:
Exclusion Criteria:
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Sangamo Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | United States | ||
| University of California Davis Health System |
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| ST-400 | Drug | Solution for intravenous administration |
|
Long-term change in markers of hemolysis, including reticulocyte count, lactate dehydrogenase, haptoglobin, and serum bilirubin, over time in the BIVV003 cohort
| Up to 15 years |
| Frequency of severe vaso-occlusive crises | Percentage of participants with severe vaso-occlusive crises, including acute pain crisis, acute chest syndrome, priapism, and splenic sequestration, in the BIVV003 cohort | Up to 15 years |
| Frequency and severity of SCD-related clinical events | Percentage of participants with SCD-related clinical events (e.g., acute renal failure, acute stroke) in the BIVV003 cohort | Up to 15 years |
| Red blood cell transfusions | Number and total volume of red blood transfusions in the BIVV003 and ST-400 cohorts | Up to 15 years |
| Sacramento |
| California |
| 95817 |
| United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Karmanos Cancer Institute | Detroit | Michigan | 48201 | United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| ID | Term |
|---|---|
| D008206 | Lymphatic Diseases |
| D000755 | Anemia, Sickle Cell |
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013789 | Thalassemia |
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