Clinical trial • Phase IV • Musculoskeletal

BMN 351 for Duchenne muscular dystrophy

Phase IV trial of BMN 351 for Duchenne muscular dystrophy. open-label, none/not specified-controlled. 8 participants.

Overview

Trial Therapeutic Area
Musculoskeletal
Trial Disease
Duchenne muscular dystrophy
Trial Stage
Phase IV
Drug Modality
Oligonucleotide
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
06-11-2025
First CTIS Authorization Date
16-03-2026

Trial design

open-label, none/not specified-controlled Phase IV trial across 4 sites in Italy, Spain, Netherlands.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
8
Trial Duration For Participant
297

Eligibility

Recruits 8 paediatric patients.

Vulnerable Population
Vulnerable population selected: minors/children are included. Multiple child assent and parental/guardian consent documents are provided (e.g. Child assent 4-11 years, Child assent 6-11, Child assent 12-15, Child assent 12-17, Mature Minor Assent, Parent/Parental-Guardian ICF, Adult Parent Guardian ICF). Consent is handled via parental/guardian informed consent for minors and age-appropriate assent forms for children; documentation available in multiple languages (English, Italian, Spanish, Dutch as evidenced by lay synopses and country-specific ICF documents).

Inclusion criteria

  • {"criterion_text":"- Participants must have completed 351-201 or 351-203 studies without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active"}

Exclusion criteria

  • {"criterion_text":"- Have known coagulation disorder"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiogram","definition_or_measurement_approach":"Safety assessments through recording incidence of AEs/SAEs/AESI and periodic physical examinations, laboratory safety tests, ECGs and echocardiograms"}

Secondary endpoints

  • {"endpoint_text":"- NSAA (total score and selected individual components, i.e., timed 10MWRT and TTR) at baseline and subsequent Q24W visits","definition_or_measurement_approach":"NSAA total score and selected components (timed 10 metre walk/run test (10MWRT) and time to rise (TTR)) assessed at baseline and every 24 weeks (Q24W)"}
  • {"endpoint_text":"- Timed 4SC at baseline and subsequent Q24W visits","definition_or_measurement_approach":"Timed 4-stair climb (4SC) measured at baseline and every 24 weeks (Q24W)"}
  • {"endpoint_text":"- SV95C (4-week monitoring periods preceding baseline and subsequent Q24W visits)","definition_or_measurement_approach":"SV95C measured during 4-week monitoring periods preceding baseline and each Q24W visit"}
  • {"endpoint_text":"- PUL 2.0 at baseline and subsequent Q24W visits","definition_or_measurement_approach":"Performance of Upper Limb (PUL) 2.0 assessment at baseline and every 24 weeks (Q24W)"}

Recruitment

Planned Sample Size
8
Recruitment Window Months
68
Consent Approach
Informed consent obtained from participants or their parent/legal guardian as appropriate. Age-specific assent documents are provided (examples: Child assent 4-11 years, Child assent 6-11, Child assent 12-15, Child assent 12-17, Mature Minor Assent). Parental/guardian consent forms (Parental-Guardian ICF, Parent ICF) and adult guardian forms are available. Country-specific subject information and consent forms are provided (documents available for Italy, Spain, Netherlands and English-language materials).

Geography

Total Number Of Sites
4
Total Number Of Participants
5

Italy

Earliest CTIS Part Ii Submission Date
20-11-2025
Latest Decision Or Authorization Date
16-03-2026
Processing Time Days
116
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
UOSD Centro Clinico Nemo Pediatrico
Contact Person Name
Marika Pane
Site Name
Centro Clinico Nemo
Department Name
Centro Clinico NeMo
Contact Person Name
Valeria Sansone

Spain

Earliest CTIS Part Ii Submission Date
06-03-2026
Latest Decision Or Authorization Date
20-03-2026
Processing Time Days
14
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Hospital Viamed Santa Angela de la Cruz
Department Name
Pediatric Neurology
Contact Person Name
Marcos Madruga Garrido
Contact Person Email
mapolgra@yahoo.es

Netherlands

Earliest CTIS Part Ii Submission Date
12-03-2026
Latest Decision Or Authorization Date
16-03-2026
Processing Time Days
4
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Leids Universitair Medisch Centrum (LUMC)
Department Name
Neurology
Contact Person Name
Erik Niks
Contact Person Email
E.H.Niks@lumc.nl

Sponsor

Primary sponsor

Full Name
Biomarin Pharmaceutical Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Medpace Finland Oy
Responsibilities
codes 1;2;6
Name
Suvoda LLC
Responsibilities
code 3
Name
Medidata Solutions Inc.
Responsibilities
code 7

Third parties

  • {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"Translations and interpreter services","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code 7","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Evidera Limited","duties_or_roles":"Embedded interviews","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Clario Medical Imaging Inc.","duties_or_roles":"ECG imaging","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"ATOM International Limited","duties_or_roles":"Cinical evaluator oversight","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"France","full_name":"Sysnav","duties_or_roles":"SV95C device management","organisation_type":"Pharmaceutical company"}
  • {"country":"Canada","full_name":"Agada Biosciences Inc.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"Laboratory sample storage","organisation_type":"Pharmaceutical company"}
  • {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"codes 1;2;6","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Mde Healthcare Services Limited","duties_or_roles":"Patient travel and reimbursement support","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Emvenio Clinical Research LLC","duties_or_roles":"Home health","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Ardena Bioanalysis B.V.","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Exon 51 specific phosphorothioate oligonucleotide
Active Substance
BMN 351
Modality
Oligonucleotide
Routes Of Administration
Intravenous infusion
Route
Intravenous infusion
Authorisation Status
Authorised
Frequency
Weekly
Maximum Dose
12 mg/kg (max daily dose amount)

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