Improving clinical trials for Charcot-Marie-Tooth disease
Accelerate Clinical Trials in CMT (ACTCMT) Study
['FUNDING_U01'] · UNIVERSITY OF ROCHESTER · NIH-10576824
This study is looking for ways to improve treatments for adults with Charcot-Marie-Tooth disease by testing new ways to measure how the condition affects them, so they can help find better therapies together.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF ROCHESTER (nih funded) |
| Locations | 1 site (ROCHESTER, UNITED STATES) |
| Trial ID | NIH-10576824 on ClinicalTrials.gov |
What this research studies
This research focuses on Charcot-Marie-Tooth disease (CMT), a rare inherited condition that affects the peripheral nerves, leading to weakness and sensory loss. The study aims to accelerate clinical trials by validating new functional outcome measures and biomarkers for adults with CMT1A, a common form of the disease. By leveraging a multicenter network, the research will gather data to support the development of effective therapies, including promising antisense oligonucleotide treatments. Patients will be involved in assessments that help define the progression of their condition and the effectiveness of new treatments.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with CMT1A who are experiencing symptoms such as weakness, imbalance, and sensory loss.
Not a fit: Patients with forms of Charcot-Marie-Tooth disease other than CMT1A may not benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments and improved quality of life for patients with Charcot-Marie-Tooth disease.
How similar studies have performed: Previous research has shown promise in developing therapies for CMT, but this specific approach to validating functional outcomes and biomarkers is relatively novel.
Where this research is happening
ROCHESTER, UNITED STATES
- UNIVERSITY OF ROCHESTER — ROCHESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: HERRMANN, DAVID N — UNIVERSITY OF ROCHESTER
- Study coordinator: HERRMANN, DAVID N
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.
Conditions: Burn injury, Charcot-Marie-Tooth Disease, Charcot Marie Disorder, Charcot Marie Tooth Disorder