Investigating muscle signaling and metabolism in ALS and Kennedy Disease
Cell Signaling, Reinnervation and Metabolism in Kennedy Disease and Amyotrophic Lateral Sclerosis (ALS)
This study is testing how muscle signaling and metabolism work in people with ALS and Kennedy Disease to find new ways to help manage these conditions.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 1 site (Paris) |
| Trial ID | NCT05107349 on ClinicalTrials.gov |
What this trial studies
This study aims to explore the molecular mechanisms behind collateral reinnervation in patients with amyotrophic lateral sclerosis (ALS) and Kennedy Disease. It will involve muscle biopsies from ALS patients, long-term survivors, and healthy controls to assess the role of insulin signaling pathways in maintaining muscle function and innervation. The research will correlate muscle innervation levels with the activation of insulin signaling pathways and glucose metabolism, which are believed to influence disease progression. By understanding these mechanisms, the study seeks to identify new therapeutic targets for ALS.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 to 80 with ALS who have had more than 5 years of disease progression without requiring respiratory support.
Not a fit: Patients with advanced ALS requiring respiratory support or gastrostomy feeding may not benefit from this study.
Why it matters
Potential benefit: If successful, this research could lead to new treatment strategies that improve muscle function and prolong survival in ALS patients.
How similar studies have performed: While the specific approach of this study is novel, previous research has indicated that understanding muscle signaling may provide insights into ALS progression.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Twenty patients with ALS, including 10 patients with more than 5 years of disease progression without requiring respiratory support or gastrostomy feeding. All ALS patients will fulfill the following inclusion criteria: * Aged 18 to 80 (inclusive) * Possible, probable (clinically or laboratory) or definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria (Brooks et al.,2000) * Ten patients with Spinal-bulbar muscular atrophy (SBMA), aged 18 to 80 * Ten healthy subjects: Control muscle specimens (deltoid) will be obtained from 10 adult patients (aged 18-80) without neuromuscular disease, undergoing shoulder surgery for local joint or bone disease in the local department of orthopaedic surgery
Where this trial is running
Paris
- Pitié-Salpêtrière Hospital — Paris, France (Recruiting)
Study contacts
- Principal investigator: Gaelle Bruneteau, MD, PhD — APHP - Hôpital Pitié-Salpêtrière
- Study coordinator: Gaelle Bruneteau, MD, PhD
- Email: nathalie.cormand@aphp.fr
- Phone: 0142162467
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.