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Clinical Trials in the USA / NCT03432520
Active, not recruiting Observational

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

NCT03432520 · tracked via the Priya Life Science USA tracker
Sponsor
Spark Therapeutics, Inc
Phase
Observational
Started
2018-08-14
Last updated
2024-11-29

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

SPK-8011SPK-8016

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

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

Study 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.

Eligibility

Sex
MALE
Min age
18 Years
Max age
—
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.

Primary outcome measure(s)

  • 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

Trial sites (12)

FacilityCityRegionStatus
Boston Children's Hospital Boston Massachusetts
Mississippi Center for Advanced Medicine Madison Mississippi
Truman Medical Centers Kansas City Missouri
Oregon Health & Science University Portland Oregon
Pennsylvania State University Milton S. Hershey Medical Center Hershey Pennsylvania
The Children's Hospital of Philadelphia Philadelphia Pennsylvania
Thomas Jefferson University Philadelphia Pennsylvania
Hemophilia Center of Western Pennsylvania Pittsburgh Pennsylvania
Virginia Commonwealth University Richmond Virginia
Royal Prince Alfred Hospital Department of Cell & Molecular Therapies Sydney New South Wales
The Alfred Hospital & Monash Medical Centre Melbourne Victoria
St. Michael's Hospital Toronto Ontario

More Spark Therapeutics, Inc. trials in the USA

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis Active, not recruiting
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Active, not recruiting
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 Active, not recruiting

Other trials for the same condition

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A Recruiting
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively Recruiting
ASC618 Gene Therapy in Hemophilia A Patients Recruiting
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A Recruiting
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03432520 on ClinicalTrials.gov ↗ ← All trials in the USA