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New study tracks muscle decline in rare muscular dystrophy

NCT ID NCT06390566

Knowledge-focused Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 25 people with limb-girdle muscular dystrophy type 2A (LGMD2A), a rare genetic disease that causes progressive muscle weakness. Researchers will measure how muscle strength changes over time and how it affects quality of life. The goal is to better understand the disease's natural course, which could help design future treatments.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 25 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2024

Expected to finish

May 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

patients with LGMD2A already followed at the Reference Center for Neuromuscular Diseases at Henri Mondor Hospital

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Confirmed diagnosis of autosomal recessive LGMD2A (two pathogenic mutations in the calpain 3 gene) * Ability to participate in the tests and examinations planned by the study (manual muscle tests, motor scales) * Informed and having signed a consent form * Affiliate to a social security scheme in France (beneficiary or entitled person). Exclusion Criteria: * Patient with another disease likely to significantly interfere with the interpretation of the natural history of LGMD2A * Participant in a clinical trial with an investigational product within 3 months preceding inclusion * Patient unable or unwilling to comply with protocol requirements * Patient under guardianship, curatorship or legal protection * Pregnant or breastfeeding woman * Patient deprived of liberty * Adult patient unable to express consent * Refusal to participate

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Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2A limb-girdle muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Limb girdle muscular dystrophy

Contacts and locations

Locations

  • Hôpital Henri Mondor

    Créteil, Île-de-France Region, 94130, France

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