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Can tracking muscle changes unlock better duchenne treatments?

NCT ID NCT05833633

Knowledge-focused Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 03, 2026 · Last updated Aug 04, 2026 · Updated 1 time

Summary

This study follows boys with Duchenne muscular dystrophy who have small mutations in their genes, a group that is less understood than others. Over one year, researchers will use muscle MRI, genetic tests, and motor and respiratory assessments to see how the disease progresses. The goal is to better define the natural history of these patients and to understand how they might respond to mutation-specific therapies like Ataluren. By learning more about these mutations, the study could help tailor future treatments for this specific group.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Muscle MRI, genetic tests, and functional motor and respiratory assessments
What this could lead to
If successful, this could lead to better understanding of how Duchenne muscular dystrophy progresses in boys with small mutations, potentially improving treatment strategies and care.
What could go wrong
This is a small observational study with only 17 participants, so findings may not apply to all patients. It does not test a new treatment directly, and results may take time to influence clinical practice.

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

17 people

The number who actually took part.

Started

Mar 2022

Finished

Jun 2025

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

All 25 patients had genetically proven DMD diagnosis with a small mutation genotype; the sample includes both ambulant and non ambulant patients; Ten patients of the 25 are in treatment with Atarulen.

Ages

4 to 30 years

Sex

Male participants only

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: \- DMD diagnosis confirming a small mutation genotype. Exclusion Criteria: * DMD patient enrolled in other clinical trials using genetic approach * impossibility to perform MRI without sedation * presence of severe cognitive or behavioral problems

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

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

Muscular dystrophy, Duchenne

Contacts and locations

Locations

  • Claudia Brogna

    Rome, 00168, Italy

More trials for these conditions

Other studies related to the condition(s) this trial covers.