🏛️ Verified NIH Record • Updated 2024-11-26
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A (NCT03432520)
A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
🔵 Active, Not Recruiting Interventional Protocol 📊 Observational Clinical Research Sponsor: Spark Therapeutics, Inc.
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Boston Children's Hospital — Boston, Massachusetts
Age range: 18 Years
🧪 Interventions in This Study
- SPK-8011 (GENETIC)
- SPK-8016 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (12 Sites)
- 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
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Hemophilia A
- 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" — Little Rock, Arkansas
- Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A — Los Angeles, California
- Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A — Milwaukee, Wisconsin
- A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A — Gainesville, Florida
- A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A — Gilbert, Arizona
- Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults — Mountain View, California
Explore by condition: All Hemophilia A clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.