Testing a new treatment for Becker muscular dystrophy
Pilot Trial of Vamorolone for the Treatment of Becker Muscular Dystrophy
This study is looking at how safe and well-tolerated a new anti-inflammatory medication called vamorolone is for people with Becker muscular dystrophy, and it will involve 30 participants over six months to see how it affects their health and physical abilities.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-10437669 on NIH RePORTER |
What this research studies
This research investigates the safety and tolerability of vamorolone, a novel anti-inflammatory steroid, in patients with Becker muscular dystrophy (BMD). The trial will involve 30 ambulatory patients over a 24-week period, where participants will receive vamorolone at a dosage of 6 mg/kg/day. Throughout the trial, various biomarkers and physical performance tests will be monitored to assess the drug's effects and potential benefits. The goal is to gather data that could support further studies on vamorolone for treating BMD.
Who could benefit from this research
Good fit: Ideal candidates for this research are ambulatory patients diagnosed with Becker muscular dystrophy.
Not a fit: Patients with non-ambulatory Becker muscular dystrophy or those not meeting the trial's inclusion criteria may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new treatment option that improves the quality of life for patients with Becker muscular dystrophy.
How similar studies have performed: Previous research has shown promise for vamorolone in treating Duchenne muscular dystrophy, indicating potential for success in similar applications.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Clemens, Paula R — University of Pittsburgh at Pittsburgh
- Study coordinator: Clemens, Paula R
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.