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This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.
This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 study. Participants will be followed for up to a total of 10 years post infusion (including the time on the dosing study).
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SPK-8011 | Genetic | Observational long-term safety follow-up study of participants previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study | ||
| SPK-8016 | Genetic | Observational long-term safety follow-up study of participants previously treated with SPK-8016 in any Spark-sponsored SPK-8016 study |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | Up to 9 years | |
| Number of Participants with Adverse Events of Special Interest (AESIs) | Up to 9 years | |
| Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development | Up to 9 years | |
| Number of Participants with Clinical Laboratory Abnormalities | Up to 9 years | |
| Number of Participants with Vital Sign Abnormalities | Up to 9 years | |
| Number of Participants with Physical Exam Abnormalities | Up to 9 years |
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Inclusion Criteria
Participants are eligible to be included in the study only if all of the following criteria apply:
Exclusion Criteria
Participants are excluded from the study if any of the following criteria apply:
1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.
Genetic males
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Males with hemophilia A, who received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored study.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trial Director | Spark Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States | ||
| Mississippi Center for Advanced Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34788507 | Derived | George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205. |
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| Madison |
| Mississippi |
| 39110 |
| United States |
| Truman Medical Centers | Kansas City | Missouri | 64108 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Pennsylvania State University Milton S. Hershey Medical Center | Hershey | Pennsylvania | 10733 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | United States |
| Hemophilia Center of Western Pennsylvania | Pittsburgh | Pennsylvania | 15213 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| Royal Prince Alfred Hospital Department of Cell & Molecular Therapies | Sydney | New South Wales | 2050 | Australia |
| The Alfred Hospital & Monash Medical Centre | Melbourne | Victoria | Australia |
| St. Michael's Hospital | Toronto | Ontario | M5B 1W8 | Canada |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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