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New hope for DMD: Long-Term trial of weekly infusions begins

NCT ID NCT07573631

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 This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 Jul 24, 2026 · Updated 2 times

Summary

This study is for people with Duchenne muscular dystrophy (DMD) who have already taken part in a previous trial of BMN 351. It tests the long-term safety of weekly intravenous infusions of BMN 351 and whether it helps maintain physical function. Only 18 participants will be enrolled, and they must continue taking stable doses of corticosteroids. The goal is to see if BMN 351 can be a safe, long-term treatment option.

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 given intravenously)
What this could lead to
If successful, this could support BMN 351 as a long-term treatment to slow muscle decline in Duchenne muscular dystrophy.
What could go wrong
This is a small, early-phase extension study with only 18 participants, so results may not apply broadly. Long-term safety and effectiveness are still unproven.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 18 people

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

Started

Apr 2026

Expected to finish

Dec 2031

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 years and older

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: * Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study * Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consistent dose regimen throughout 351-202 or 351-203 except for modifications to accommodate changes in weight * Transition to the equivalent dose of vamorolone is permitted in 351-202 where approved in participating countries. * Willing and able to adhere to the study visit schedule and other protocol requirements * Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active * Contraceptive use by males should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. * Willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure * Willing and able to provide written, signed informed consent as parent or guardian after the nature of the study has been explained and prior to performance of any research-related procedure Exclusion Criteria: * Have known coagulation disorder * Are taking any prohibited medications * any approved exon skipping therapy within 12 weeks prior to baseline or with any gene therapy for the treatment of DMD at any time * anti-coagulants, anti-thrombotics, or anti-platelet agents * immunosuppressants

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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.

Duchene muscular dystrophy Duchenne muscular dystrophy (DMD)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    6 sites in 5 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Fondazione Serena ETS - Centro Clinico NeMO Milano

    Milan, Italy, Italy

  • Great Ormond Street Hospital NHS Foundation Trust

    London, United Kingdom, WC1N 3JH, United Kingdom

  • Hospital Viamed Santa Angela De la Cruz

    Seville, Spain, 41013, Spain

  • Leids Universitair Medisch Centrum

    Leiden, Netherlands, 2333 ZA, Netherlands

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

    Rome, Italy, Italy

  • Yeditepe University Kosuyolu Hospital

    Istanbul, Turkey, Turkey (Türkiye)

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