NCT03003533CompletedPhase 1/2
A Gene Transfer Study for Hemophilia A
Gene-transfer, Open-label, Dose-escalation Study of SPK-8011 [Adeno-associated Viral Vector With B-domain Deleted Human Factor VIII Gene] in Individuals With Hemophilia A
Source: ClinicalTrials.gov · Last updated 30 Dec 2024
Brief summary (registry, in English)
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 25 (actual)
- First submitted
- 16 Dec 2016
- Start date
- 26 Jan 2017
- Primary completion date
- 5 Dec 2023
- Lead sponsor
- Spark Therapeutics, Inc.
- Diseases
- Haemophilia
- NCT number
- NCT03003533
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 25
- From 26 Jan 2017 to the primary completion date, 5 Dec 2023
- 16 sites in 5 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Currently on antiviral therapy for hepatitis B or C
- Have detectable antibodies reactive with AAV-Spark200 capsid
- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational product within the last 12 weeks
Other illness
- Evidence of active hepatitis B or C
- Have significant underlying liver disease
Lab values
- Have serological evidence\* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 and who are on an antiretroviral drug regimen (\* participants who are HIV+ and stable with CD4 count \>200/mm3 and undetectable viral load are eligible to enroll)
Who the study is looking for
- Males age 18 years or older
- Confirmed diagnosis of hemophilia A as evidenced by their medical history with baseline FVIII activity levels \<=2%
- Have received \>150 exposure days (EDs) to FVIII concentrates or cryoprecipitate
- Have no prior history of allergic reaction to any FVIII product
- Have no measurable inhibitor against FVIII as assessed by the central laboratory and have no prior history of inhibitors to FVIII protein and no clinical signs or symptoms of decreased response to FVIII administration
- Agree to use reliable barrier contraception
Age in the registry: 18 years to - · Sex: Male · Does not accept healthy volunteers
Sites (16)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Royal Prince Alfred Hosptial Coordinator of this site? Confirm its status | Camperdown | Australia | Unknown status |
| The Alfred Hospital Coordinator of this site? Confirm its status | Melbourne | Australia | Unknown status |
| McMaster University Medical Centre and Juravinski Hospital Coordinator of this site? Confirm its status | Hamilton | Canada | Unknown status |
| Chaim Sheba Center Coordinator of this site? Confirm its status | Ramat Gan | Israel | Unknown status |
| Mahidol University - Ramathibody Hospital Coordinator of this site? Confirm its status | Bangkok | Thailand | Unknown status |
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| University of Florida Health Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| Pennsylvania State University / Milton S. Hershey Medical Center Coordinator of this site? Confirm its status | Hershey | United States | Unknown status |
| Mississippi Center for Advanced Medicine Coordinator of this site? Confirm its status | Madison | United States | Unknown status |
| Weill Cornell Medicine-Comprehensive Center for Hemophilia and Coagulation Disorders Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Jefferson University Hospitals Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Hemophilia Center of Western Pennsylvania Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| Oregon Health Science University Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| Virginia Commonwealth University School of Medicine Coordinator of this site? Confirm its status | Richmond | United States | Unknown status |
| University of California Davis - Hemostasis and Thrombosis Center Coordinator of this site? Confirm its status | Sacramento | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.