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NCT06833489: ARNseq-Musc

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Recruiting now NA Last updated 18 February 2025
What this trial tests

NA trial testing ARN extraction from muscle biopsies in Rare Genetic Muscle Diseases in 50 participants. Currently enrolling.

Timeline
1 March 2025
Primary endpoint
1 September 2026
1 March 2027

Quick facts

Lead sponsorAssistance Publique Hopitaux De Marseille
PhaseNA
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposediagnostic
Enrollment50
Start date1 March 2025
Primary completion1 September 2026
Estimated completion1 March 2027
Sites1 location across France

Drugs / interventions tested

Conditions studied

Sponsor

Assistance Publique Hopitaux De Marseille — full company profile →

Who can join

Eligibility, any sex, with Rare Genetic Muscle Diseases or Muscular Dystrophy, Duchenne. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases. The researchers hypothesise that some of these misdiagnosed patients carry pathogenic RNA (transcript) disrupting variants that were not identified by DNA sequencing. In fact, DNA sequencing analyses can be negative despite the presence of a pathogenic variant that disrupts RNA splicing or expression, causing a genetic disease. For this reason, RNA sequencing can provide a diagnosis in patients who have not been diagnosed by DNA sequencing, thus putting an end to diagnostic wandering. Thus, as a descriptive prevalence study, the objectives are first to determine the rate of positive diagnoses made by the RNAseq approach in patients with muscle diseases that have not yet been diagnosed, and then to identify the genomic characteristics of the pathogenic variants identified in patients by RNAseq analysis, in order to facilitate the identification of this type of variant in future patients. 50 patients will be included in this study during 2 years.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

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Other Assistance Publique Hopitaux De Marseille trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06833489.

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