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New hope for duchenne? experimental drug BMN 351 enters human testing

NCT ID NCT06280209

Disease control Sponsor: BioMarin Pharmaceutical 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial is testing a drug called BMN 351 in 18 boys aged 4 to 10 with Duchenne muscular dystrophy who have a specific genetic change. The drug is designed to help the body produce a shorter but still useful version of the muscle protein dystrophin. The main goal is to check the drug's safety and how the body processes it, not yet to prove it works.

What this could mean

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

Active substance
BMN 351 (an antisense oligonucleotide designed to skip exon 51 in the dystrophin gene)
What this could lead to
If successful, this could lead to a treatment that helps boys with Duchenne muscular dystrophy produce a shorter but functional version of the dystrophin protein, potentially slowing muscle decline.
What could go wrong
This is an early phase 1/2 trial with only 18 participants, so safety and dosing are still being established. It is not yet known if BMN 351 will meaningfully improve muscle function or quality of life.

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.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

18 people

The number who actually took part.

Started

Jan 2024

Expected to finish

Apr 2027

An estimate. End dates often move.

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.

Ages

4 to 10 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: * Age 4 to 10 * Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping * Able to walk * Not requiring assistance from a ventilator to breathe * Currently on consistent doses of steroid treatment for the last 12 weeks Exclusion Criteria: * The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function. * Treatment with an exon skipping therapy within 12 weeks prior to the first visit. * Any history of treatment with gene therapy

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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.

Duchenne muscular dystrophy

Contacts and locations

Locations

  • Fondazione Serena ETS - Centro Clinico NeMO Milano

    Milan, Italy

  • Great Ormond Street Hospital NHS Foundation Trust

    London, WC1N 3JH, United Kingdom

  • Hospital Sant Joan de Deu

    Barcelona, 08950, Spain

  • Hospital Viamed Santa Angela De la Cruz

    Seville, 41013, Spain

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZA, Netherlands

  • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    Rome, Italy

  • Yeditepe University Kosuyolu Hospital

    Istanbul, Turkey (Türkiye)

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