New study aims to map rare muscle disease progression

NCT ID NCT06210672

What the study statuses mean

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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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 ยท Last updated Jun 27, 2026

Summary

This study was designed to track the natural course of gamma-sarcoglycanopathy (LGMDR5), a rare muscle-weakening disease, over two years. Researchers planned to measure changes in muscle strength, walking ability, and daily function in patients aged 6 to 35. The goal was to better understand the disease to help design future treatments. However, the study was withdrawn before enrolling any participants.

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.

Started

Jan 2025

Finished

Sep 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Who is studied

The study will enroll 30 ambulant pediatric patients less than 12 years of age (cohort 1) and 20 non-ambulant adolescent or young adults less than 35 years of age (cohort 2)

Ages

6 to 35 years

Sex

Anyone

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: * Male and female patients 6 to less than 35 years of age * Ambulant patients should be less than 12 years of age and able to walk/run 10 meters (item 29 of NSAD) in less than 15 sec * Confirmed diagnosis of LGMDR5 (genotyping) * FVC \> 40% Exclusion Criteria: * Subjects with co-morbidity which may interfere with LGMDR5 natural evolution and/or evaluation of outcome measures * Need of non-invasive ventilation \> 16h per 24h or any invasive ventilation * Left ventricular ejection fraction (LVEF) \< 30% or prior heart failure decompensation requiring hospitalization * Past participation in a gene therapy or cell therapy trial

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Hedi Chaker Hospital Child Neurology Department

    Sfax, Tunisia

  • Hopital Raymond Poincare

    Garches, 92380, France

  • National Institute Mongi Ben Hmida of Neurology

    Tunis, Tunisia