Duchenne Muscular DystrophyPhase 1/2NCT06280209
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
A Phase 1/2 study testing BMN 351 in people with Duchenne Muscular Dystrophy, run by BioMarin Pharmaceutical at 7 sites in 5 countries.
Trial summary
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping. Read more about this study
- Who is it for?
- Ages 4 to 10 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- BMN 351
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 7 sitesin 5 countries
- Study dates
- 3 Jan 2024to 14 Apr 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 10 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
7 sites in 5 countries.
7 sites in 5 countries
Italy2 sites
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome
Spain2 sites
Hospital Sant Joan de Deu
Barcelona, 08950
Hospital Viamed Santa Angela De la Cruz
Seville, 41013
Turkey (Türkiye)1 site
Yeditepe University Kosuyolu Hospital
Istanbul
United Kingdom1 site
Great Ormond Street Hospital NHS Foundation Trust
London, WC1N 3JH
About this study
Official title: A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
Read the detailed description
This is Phase 1/2, open-label, multi-center study consisting of 2 parts to evaluate the safety and tolerability of BMN 351 at escalating doses in participants with Duchenne Muscular Dystrophy (DMD) with genetic mutations amenable to exon 51 skipping.
Participants will be assigned to one of three groups called cohorts (Cohort 1, 2 or 3). Cohort 1 participants are further divided into Cohort 1A and Cohort 1B. In Cohort 1A, 3 participants will receive increasing doses once every 2 weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351. In part 2, the participants in cohort 1A will transition to once weekly dosing. The participants in Cohort 1B, 2, and 3 will initiate low, medium, and high doses of BMN 351 and continue once weekly dosing at that same dose. The study will enroll approximately 18 participants.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- BMN 351
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMN 351Drug
Anti-sense Oligonucleotide BMN 351 will be administered intravenously.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1A | Experimental | Cohort 1A will consist of both a single ascending dose (SAD) part and a multiple ascending dose (MAD). BMN 351 will be administered once every 2 weeks during the SAD portion of the study for up to 8 weeks and once weekly during the MAD portion for up to 89 weeks. Receives: Drug: BMN 351 |
| Cohort 1B | Experimental | BMN 351 low dose will be administered once weekly for up to 97 weeks Receives: Drug: BMN 351 |
| Cohort 2 | Experimental | BMN 351 medium dose will be administered once weekly for up to 73 weeks Receives: Drug: BMN 351 |
| Cohort 3 | Experimental | BMN 351 high dose will be administered once weekly for up to 48 weeks Receives: Drug: BMN 351 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters).
The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
Measured: Up to 97 weeks.
Secondary outcomes (1)
Pharmacokinetics (PK) concentration of BMN 351 in plasma, urine and muscle approximately every 8 weeks for up to 97 weeks.
Serial measurements of plasma and urine PK predose approximately hourly up to 24 hours post-infusion. Muscle PK will be measured at 13 weeks or 25 weeks only post dosing.
Measured: Serial measurements pre and post infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2024
- Study start3 Jan 2024
- Primary completion14 Apr 2027 (estimated)
- Study completion14 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06280209
- Sponsor's protocol ID
- 351-201
- Other IDs
- 2023-506737-30-00, 2023
- Status last verified
- Jun 2026
- First submitted
- 26 Jan 2024
- Last updated
- 25 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06280209
- Acronym / sponsor ID
- 351-201
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 3 Jan 2024 (actual)
- Primary completion
- 14 Apr 2027 (estimated)
- Study completion
- 14 Apr 2027 (estimated)
- First posted
- 28 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
Detailed description
This is Phase 1/2, open-label, multi-center study consisting of 2 parts to evaluate the safety and tolerability of BMN 351 at escalating doses in participants with Duchenne Muscular Dystrophy (DMD) with genetic mutations amenable to exon 51 skipping.
Participants will be assigned to one of three groups called cohorts (Cohort 1, 2 or 3). Cohort 1 participants are further divided into Cohort 1A and Cohort 1B. In Cohort 1A, 3 participants will receive increasing doses once every 2 weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351. In part 2, the participants in cohort 1A will transition to once weekly dosing. The participants in Cohort 1B, 2, and 3 will initiate low, medium, and high doses of BMN 351 and continue once weekly dosing at that same dose. The study will enroll approximately 18 participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1A | Experimental | Drug: BMN 351Cohort 1A will consist of both a single ascending dose (SAD) part and a multiple ascending dose (MAD). BMN 351 will be administered once every 2 weeks during the SAD portion of the study for up to 8 weeks and once weekly during the MAD portion for up to 89 weeks. |
| Cohort 1B | Experimental | Drug: BMN 351BMN 351 low dose will be administered once weekly for up to 97 weeks |
| Cohort 2 | Experimental | Drug: BMN 351BMN 351 medium dose will be administered once weekly for up to 73 weeks |
| Cohort 3 | Experimental | Drug: BMN 351BMN 351 high dose will be administered once weekly for up to 48 weeks |
Who may qualify
- Ages
- 4 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BMN 351Anti-sense Oligonucleotide BMN 351 will be administered intravenously. | Drug | Cohort 1A, Cohort 1B, Cohort 2, Cohort 3 |
Primary outcome
To evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters).
Time frame: Up to 97 weeks.
The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
1 secondary outcome
Pharmacokinetics (PK) concentration of BMN 351 in plasma, urine and muscle approximately every 8 weeks for up to 97 weeks.
Time frame: Serial measurements pre and post infusion.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BMN 351 in people with Duchenne Muscular Dystrophy, run by BioMarin Pharmaceutical at 7 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 25 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Italy, Spain, Netherlands, Turkey (Türkiye), United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters). (time frame: Up to 97 weeks.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jun 2026.