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

N/AActive Not RecruitingNCT03432520

Spark Therapeutics, Inc.29 enrolled

Overview

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).

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hemophilia A

Interventions

SPK-8011GENETIC

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

SPK-8016GENETIC

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

Eligibility

Sex: MALEMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (12)

Boston Children's Hospital

Boston, Massachusetts, United States

Mississippi Center for Advanced Medicine

Madison, Mississippi, United States

Truman Medical Centers

Kansas City, Missouri, United States

Outcomes

Primary Outcomes

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Up to 9 years

Number of Participants with Adverse Events of Special Interest (AESIs)

Time frame: Up to 9 years

Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development

Time frame: Up to 9 years

Number of Participants with Clinical Laboratory Abnormalities

Time frame: Up to 9 years

Number of Participants with Vital Sign Abnormalities

Time frame: Up to 9 years

Number of Participants with Physical Exam Abnormalities

Time frame: Up to 9 years

Data from ClinicalTrials.gov

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