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

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)

Facility	City	Region
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

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A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Active, not recruiting

Other trials for the same condition

ASC618 Gene Therapy in Hemophilia A Patients Recruiting

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency) Recruiting

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle" Active, not recruiting

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) Enrolling by invitation

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