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NCT Number: NCT07138963

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.

Recruiting

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Key information

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Ain Shams University

Cairo, 11591, Egypt

Location status: Recruiting

Location contact

Alice K Abdel Aleem, MD

SUB_INVESTIGATOR

Maha Z Ramadan, MD

SUB_INVESTIGATOR

Nagia A Fahmy, MD

SUB_INVESTIGATOR

Nermine S Elsayed, MD

SUB_INVESTIGATOR

Nouran M Sabry, MSc

CONTACT

[email protected]

00201092289982

Radwa M Soliman, MD

SUB_INVESTIGATOR

About this study

Congenital Muscular dystrophies (CMDs) and Congenital Myopathies (CMs) constitute the two most important groups of congenital muscle diseases with early onset whether at birth or early infancy.

CMDs and CMs as a group encompass great clinical and genetic heterogeneity so that achieving an accurate genetic diagnosis has become increasingly challenging, even in the era of next generation sequencing. However, it has become clear that there is overlap between CMDs and CMs on the clinical, pathological and genetic level.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients with clinical criteria of Congenital Myopathies (CMs) and Congenital Muscular dystrophies (CMDs) with different modes of inheritance.
  • Age: patients below age of 18 years.
  • Gender: Both males and females are included
  • Genetically confirmed CMs and CMDs.

Exclusion criteria

  • Patients above 18 years.
  • Spinal muscular atrophy (SMA),and root lesions.
  • Congenital myasthenic syndromes
  • Dystrophinopathies,Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy (LGMD)
  • .Metabolic myopathies
  • .Inflammatory muscle diseases

Treatment and study plan

Genetic Testing and Muscle Biopsy

Diagnostic Test

Comprehensive diagnostic assessment including clinical examination, electromyography (EMG), muscle biopsy for histopathological evaluation, and genetic testing to determine phenotype-genotype correlation in congenital myopathies and muscular dystrophies.

Primary outcomes

  1. Phenotype and genotype of congenital myopathies (CM) and congenital muscular dystrophies (CMD) patients

    Time frame: Two years

    Correlation of the most common clinical presentations and complications among Egyptian patients with congenital myopathies (CM) and congenital muscular dystrophies (CMD) of different genotypes.

Secondary outcomes

  1. Common facial features

    Time frame: Two years

    Identification and comparison of typical dysmorphic facial features associated with different genetic subtypes of congenital myopathies (CM) and congenital muscular dystrophies (CMD).

  2. Response to physiotherapy

    Time frame: Two years

    Response to physiotherapy will be recorded.

  3. Prognosis of same genotype across different age groups

    Time frame: Two years

    The prognosis of the same genotype across different age groups will be recorded.

Study contacts

Contact information is provided by the study sponsor or research team.

Nouran M Sabry, MSc

CONTACT

[email protected]

00201092289982

Sponsors and collaborators

Lead sponsor

Ain Shams University

Other

Registry information

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Aug 24, 2025
Registry last updated
Aug 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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