Testing a new gene therapy to improve blood clotting in patients with severe hemophilia A.
A Phase I Clinical Trial Testing Feasibility of Hematopoietic Stem Cell Gene Therapy Using Platelet Factor VIII to Safely Improve Hemostasis for Severe Hemophilia A with Inhibitory Antibodies
This study is testing a new gene therapy for people with severe hemophilia A to see if it can help their bodies make a key protein that helps stop bleeding, which could mean fewer trips for treatments and less hassle with current therapies.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project ID | NIH-10599276 on NIH RePORTER |
What this research studies
This research investigates a novel gene therapy approach aimed at treating severe hemophilia A, a condition characterized by a significant deficiency in a crucial blood clotting factor. The therapy involves using hematopoietic stem cells to deliver platelet factor VIII, which could potentially enhance the body's ability to stop bleeding. Patients will be monitored for safety and effectiveness as they receive this treatment, which may reduce the need for frequent infusions of traditional clotting factor therapies. The study aims to address challenges faced by patients, such as the development of inhibitory antibodies that complicate treatment.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with severe hemophilia A who have developed inhibitory antibodies against standard treatments.
Not a fit: Patients with mild hemophilia or those who do not have inhibitory antibodies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve the quality of life for patients with severe hemophilia A by reducing bleeding episodes and the need for frequent treatments.
How similar studies have performed: Previous research has shown success in using gene therapy for hemophilia, indicating potential for this novel approach to also yield positive outcomes.
Where this research is happening
Milwaukee, United States
- Medical College of Wisconsin — Milwaukee, United States (Active)
Researchers
- Principal investigator: Wilcox, David Allen — Medical College of Wisconsin
- Study coordinator: Wilcox, David Allen
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.