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Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 10 years following exposure to SB-318, SB-913, or SB-FIX.
Non-interventional, multi-center, long-term follow-up (LTFU) study of subjects dosed with SB-318 in the clinical study SB-318-1502, SB-913 in the clinical study SB-913-1602, and SB-FIX in clinical study SB-FIX-1501. All subjects dosed in the studies and completed at least 52 weeks post-infusion follow-up in their primary protocol will be offered to participate. Subjects who enroll will be monitored for a total of up to 10 years following exposure to the respective investigational products.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Subjects who received SB-318 | Subjects who received SB-318 in clinical study SB-318-1502 |
| |
| Subjects who received SB-913 | Subjects who received SB-913 in clinical study SB-913-1602. |
| |
| Subjects who received SB-FIX | Subjects who received SB-FIX in clinical study SB-FIX |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SB-318 | Biological | No study drug is administered in this study. Subject who received SB-318 in a previous trial will be evaluated in this trial for long-term safety. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Long-term safety | Long-term safety of SB-318, SB-913, and SB-FIX by assessment of any newly-diagnosed or worsening of existing medical conditions. | 10 years |
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Inclusion Criteria:
Exclusion Criteria:
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All subjects who have received SB-318 in Study Protocol SB-318-1502, SB-913 in Study Protocol SB-913-1602 or SB-FIX in Study Protocol SB-FIX-1501 and who have consented to participate in this Long Term Follow-up study.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor, MD | Sangamo Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | United States | ||
| Ann & Robert H. Lurie Children's Hospital of Chicago |
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| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| D008059 | Mucopolysaccharidosis I |
| D016532 | Mucopolysaccharidosis II |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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plasma, serum
| SB-913 | Biological | No study drug is administered in this study. Subject who received SB-913 in a previous trial will be evaluated in this trial for long-term safety. |
|
| SB-FIX | Biological | No study drug is administered in this study. Subject who received SB-FIX in a previous trial will be evaluated in this trial for long-term safety. |
|
| Chicago |
| Illinois |
| 60611 |
| United States |
| New York University Grossman School of Medicine | New York | New York | 10016 | United States |
| University of North Carolina | Chapel Hill | North Carolina | 27514 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |