Assessment of children with inherited non-Duchenne myopathies at Sohag University Hospital
Clinical and Functional Assessment of Patients With Inherited Non-Duchenne Myopathies in Sohag University Hospital
This study looks at children with inherited non-Duchenne myopathies to see how the condition affects them and to help improve their diagnosis and care.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 30 (estimated) |
| Ages | 2 Years to 18 Years |
| Sex | All |
| Sponsor | Sohag University Academic / other |
| Locations | 3 sites (Sohag and 2 other locations) |
| Trial ID | NCT06574919 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the clinical and functional characteristics of children diagnosed with inherited non-Duchenne myopathies at Sohag University Hospital. The study will include children aged 2 to 18 years who have a confirmed diagnosis based on clinical features, laboratory tests, and genetic testing. The research will take place over one year, focusing on understanding the progression and impact of these myopathies. The findings may help improve early diagnosis and management strategies for affected patients.
Who should consider this trial
Good fit: Ideal candidates are children aged 2 to 18 years with a confirmed diagnosis of non-Duchenne myopathies.
Not a fit: Patients with a confirmed diagnosis of Duchenne Muscular Dystrophy or other specified neuromuscular disorders may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance early diagnosis and tailored management for children with inherited non-Duchenne myopathies.
How similar studies have performed: While there have been advancements in understanding muscular myopathies, this specific observational approach is relatively novel and untested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * • Age between 2 and 18 years * Confirmed diagnosis of non-DMD based on clinical features, laboratory investigations, and genetic testing. * Patients and their parents agree to participate in the study. Exclusion Criteria: * • Children with a confirmed diagnosis of DMD * Children with incomplete medical records or unavailable clinical data. * Children with endocrinal ,nutritional critical care and inflammatory myopathies. * Other neuromuscular disorders that affect AHC or nerves as SMA, congenital myasthenia Gravis and neuropathic. * Other acquired neuromuscular disorders as Guillain Barre syndrome and toxic myopathy. * Patients and their parents refuse to participate in the study.
Where this trial is running
Sohag and 2 other locations
- Sohag university Hospital — Sohag, Egypt (Recruiting)
- Sohag University — Sohag, Egypt (Recruiting)
- Sohag University Hospital — Sohag, Egypt (Recruiting)
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.