Status:
NOT_YET_RECRUITING
Natural History Study for Patients With Nemaline Myopathy in Belgium
Lead Sponsor:
Centre Hospitalier Universitaire de Liege
Collaborating Sponsors:
Centre Hospitalier Régional de la Citadelle
SYSNAV
Conditions:
Nemaline Myopathy
Eligibility:
All Genders
Phase:
NA
Brief Summary
This is a prospective, longitudinal, observational study designed to characterize the natural history of Nemaline Myopathy (NM), a rare congenital neuromuscular disorder. The study will follow up to 1...
Detailed Description
Nemaline Myopathies (NM) are a group of rare, genetically and clinically heterogeneous congenital myopathies characterized by the presence of nemaline bodies (rods) on muscle biopsy. The estimated inc...
Eligibility Criteria
Inclusion
- Male or Female
- Any age
- Patient and/or parent or legal guardian must be willing and have the ability to provide written informed consent for participation in the study.
- Diagnosis of NM which in most cases includes having a disease-causing variant/s in one of the known NM causative genes and a consistent clinical phenotype.
Exclusion
- Any confirmed chronic or acute condition or disease affecting any system(s), which could interfere with the results of the study and/or the compliance with the study procedures. This will be subject to the clinical judgement of the Coordinating Investigator (CI) and/or the Principal Investigator (PI).
- Clinically significant medical finding on the physical examination other than NM that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures.
- Participants of ongoing (interventional) clinical trials that assess the efficacy of potential treatments will be excluded as assessments need to be done on the basis that represent the natural progression of NM.
- Safety concerns. This includes anything that might put the participant and/or their Parent(s) or Guardian(s) at risk through participating in the study potentially including but not limited to: Safeguarding concerns, Social Issues and Health issues.
- Ongoing pregnancy (for participants becoming pregnant during the trial, some assessments may be cancelled or postponed. This will be subject to the clinical judgement of the Coordinating Investigator and/or the Principal Investigator)
Key Trial Info
Start Date :
December 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2029
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT07201636
Start Date
December 1 2025
End Date
August 1 2029
Last Update
November 14 2025
Active Locations (1)
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1
Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, Belgium, 4000