NCT06280209

Brief 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.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for phase_1

Timeline
4mo left

Started Jan 2024

Typical duration for phase_1

Geographic Reach
6 countries

8 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress85%
Jan 2024Sep 2026

Study Start

First participant enrolled

January 3, 2024

Completed
23 days until next milestone

First Submitted

Initial submission to the registry

January 26, 2024

Completed
1 month until next milestone

First Posted

Study publicly available on registry

February 28, 2024

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2026

Last Updated

April 8, 2026

Status Verified

April 1, 2026

Enrollment Period

2.7 years

First QC Date

January 26, 2024

Last Update Submit

April 7, 2026

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

  • 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.

    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 pre and post infusion.

Other Outcomes (4)

  • To evaluate the immune response to BMN 351.

    Up to 97 weeks

  • To evaluate the effect of BMN 351 on physical function.

    Change from baseline at week 25.

  • To evaluate the effect of BMN 351 on physical function.

    Change from baseline at week 25.

  • +1 more other outcomes

Study Arms (4)

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.

Drug: BMN 351

Cohort 1B

EXPERIMENTAL

BMN 351 low dose will be administered once weekly for up to 97 weeks

Drug: BMN 351

Cohort 2

EXPERIMENTAL

BMN 351 medium dose will be administered once weekly for up to 73 weeks

Drug: BMN 351

Cohort 3

EXPERIMENTAL

BMN 351 high dose will be administered once weekly for up to 48 weeks

Drug: BMN 351

Interventions

BMN 351DRUG

Anti-sense Oligonucleotide BMN 351 will be administered intravenously.

Cohort 1ACohort 1BCohort 2Cohort 3

Eligibility Criteria

Age4 Years - 10 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Children's Hospital LHSC

London, Ontario, N6A 5W9, Canada

RECRUITING

Fondazione Serena ETS - Centro Clinico NeMO Milano

Milan, Italy

RECRUITING

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

Rome, Italy

RECRUITING

Leids Universitair Medisch Centrum

Leiden, 2333 ZA, Netherlands

RECRUITING

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

RECRUITING

Hospital Viamed Santa Angela De la Cruz

Seville, 41013, Spain

RECRUITING

Yeditepe University Kosuyolu Hospital

Istanbul, Turkey (Türkiye)

RECRUITING

Great Ormond Street Hospital NHS Foundation Trust

London, WC1N 3JH, United Kingdom

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 26, 2024

First Posted

February 28, 2024

Study Start

January 3, 2024

Primary Completion (Estimated)

September 30, 2026

Study Completion (Estimated)

September 30, 2026

Last Updated

April 8, 2026

Record last verified: 2026-04

Locations

NL(1)ES(2)TU(1)GB(1)CA(1)IT(2)