Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

Conditions

Hemophilia A

Brief summary

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.

Detailed description

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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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

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10.1056/nejmoa2104205

Interventions

GENETICSPK-8011

Observational long-term safety follow-up study of participants previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study

GENETICSPK-8016

Observational long-term safety follow-up study of participants previously treated with SPK-8016 in any Spark-sponsored SPK-8016 study

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Age

18 Years to No maximum

Healthy volunteers

No

Inclusion criteria

Participants are eligible to be included in the study only if all of the following criteria apply: 1. Have received a single intravenous administration of SPK-8011 or SPK-8016 in either Study SPK-8011-101 or SPK-8016-101, respectively. 2. Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures.

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.

Design outcomes

Primary

Measure	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

Countries

Australia, Canada, United States

Outcome results

None listed