Congenital Myopathies

3

Review clinical trials related to Congenital Myopathies. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

VirtualPark_Pediatric

The goal of this clinical trial is to assess the effects of a dual-task, multimodal training program on slowing functional motor decline in children and adolescents with neuromuscular disorders. The main questions it aims to answer are: Does Virtual Park slow functional motor decline in children and adolescents with neuromuscular diseases compared with standard therapy? Does Virtual Park contribute to increased engagement and improved neuropsychological function? If there is a comparison group, Researchers will compare dual-task cycling supported by the Virtual Park application to standard therapy to see whether it improves motor and neuropsychological functions and engagement. Participants will undergo two phases: * Phase 1: 3 months of standard therapy; * Phase 2: 3 months of experimental conditions in which patients undergo a rehabilitative intervention with Virtual Park, 3 times a week.

Participants needed: 22
Trial details
Age: 6-18Biological sex: AllType: InterventionalSponsor: Istituto di Sistemi e Tecnologie Industriali Intelligenti per il Manifatturiero AvanzatoUpdated: May 12, 2026Locations: 4
Eligibility criteria

availability of a definitive clinical or molecular genetic diagnosis of a neurom... [+3]

other concomitant genetic diseases; [+3]

Status: Recruiting

Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies

The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases. MicroRNAs (miRs) are small messengers that help control how cells grow and stay healthy. Some of these, like miR-1, are specifically found in muscles and the heart. Research shows that levels of miR-1 are often abnormal in people with muscle-wasting conditions, but more information are needed to understand how this relates to the severity of the disease. The main goal is to compare the blood levels of miR-1 between four different groups at different ages and severities: 1. Patients with Duchenne or Becker muscular dystrophy (DMD/DMB). 2. Patients with Myotonic Dystrophy Type 1 (Steinert's disease). 3. Patients with congenital myopathies. 4. Healthy volunteers (control group). The main objective is to assess if miR-1 levels can accurately show how a muscular disease is progressing.

Participants needed: 104
Trial details
Age: 2+Biological sex: AllType: InterventionalSponsor: University Hospital, Clermont-FerrandUpdated: Feb 25, 2026Locations: 1
Eligibility criteria

Age: Participants must be older than 2 years of age [+3]

Refusal to participate expressed by the subject or their parental authority. [+5]

Status: Recruiting

Phenotype - Genotype Correlation in a Sample of Egyptian Patients With Congenital Myopathies and Congenital Muscular Dystrophies

The aim of this study is to correlate the phenotype and genotype among a sample of Egyptian patients with Congenital myopathies and Congenital muscular dystrophies.

Participants needed: 25
Trial details
Age: 1-18Biological sex: AllType: ObservationalSponsor: Ain Shams UniversityUpdated: Aug 24, 2025Locations: 1
Eligibility criteria

Patients with clinical criteria of Congenital Myopathies (CMs) and Congenital Mu... [+3]

Patients above 18 years. [+5]