Assessing swallowing difficulties in spinal muscular atrophy patients
Implementation of Flexible Endoscopic Evaluation of Swallowing (FEES) and Standardized FEES Scores in the Diagnostic Work-up of Dysphagia in Spinal Muscular Atrophy - DYS-SMA Trial
This study is testing how swallowing difficulties affect people with spinal muscular atrophy and whether new treatments like Risdiplam can help improve their swallowing over time.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 79 (estimated) |
| Sex | All |
| Sponsor | University of Giessen Academic / other |
| Locations | 1 site (Giessen, Hesse) |
| Trial ID | NCT04773470 on ClinicalTrials.gov |
What this trial studies
This project aims to evaluate the frequency and severity of dysphagia and bulbar dysfunction in patients with spinal muscular atrophy (SMA) types 1, 2, and 3 using Flexible Endoscopic Evaluation of Swallowing (FEES) and validated dysphagia scores. The study will track changes in swallowing function over time in newly diagnosed patients and those starting treatment with new SMA therapies, particularly Risdiplam. Data will be compared between groups receiving different treatments to understand the impact on swallowing difficulties.
Who should consider this trial
Good fit: Ideal candidates include patients with genetically confirmed 5q-linked spinal muscular atrophy who are being followed at the neuromuscular center in Giessen.
Not a fit: Patients with physical illnesses that could interfere with assessments or those in poor general condition may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management and treatment strategies for dysphagia in SMA patients.
How similar studies have performed: While there have been studies on dysphagia in SMA, this specific approach using FEES in conjunction with new SMA therapies is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: All patients with genetically determined 5q-linked SMA followed at the neuromuscular center Gießen - Informed and written consent signed by the patient or a legal guardian Exclusion Criteria: * Physical illness that, according to its type and severity, could interfere with the planned assessments, could have an influence on the parameters to be investigated or could endanger the patient or test person during the course of the investigation. * Pregnancy or lactation and a positive pregnancy test * Acute suicidal tendency or external danger * Poor general condition
Where this trial is running
Giessen, Hesse
- University Hospital Giessen and Marburg, Campus Giessen — Giessen, Hesse, Germany (Recruiting)
Study contacts
- Principal investigator: Samra Hamzic, MA — University Giessen
- Study coordinator: Samra Hamzic, MA
- Email: samra.hamzi@neuro.med.uni-giessen.de
- Phone: +4964198559233
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.