A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

Phase 2

Completed

• Conditions: 10028302; 10029317; Duchenne muscular dystrophy; Duchenne's disease

• Registration Number: NL-OMON43779
• Lead Sponsor: BioMarin Parmaceutical Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 29 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

1. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with BMN 045 confirmed by a state-of-the-art DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or HRMCA (High-Resolution Melting Curve Analysis)
2. Ambulant boys aged at least 5 years on the day of first dosing able to walk for at
least 230 meters in the 6 minute walking distance (6MWD) at the first screening
visit and also at the baseline visit. In addition, 2 of the 3 pre-treatment 6MWD
tests (screen 1, screen 2, baseline) must be within ± 30 meters of each other prior
to first BMN 045 administration
3. Adequate quality for biopsy (confirmed with MRI) of the lateral head of the gastrocnemius muscle.
4. Life expectancy of at least 3 years after inclusion in the study.
5. Glucocorticosteroid use which is stable for at least 3 months prior to first BMN 045 administration. Subjects must have been receiving glucocorticosteroids for at least 6 months prior to the first BMN 045 administration.

Exclusion Criteria

1. Known presence of dystrophin in >=5% of fibres in a pre-study diagnostic muscle biopsy (i.e. historic muscle biopsy taken prior to written informed consent for this study).
2. Current or history of liver disease or impairment
3. Current, or history of, renal disease or impairment.
4. at least two aPTT above ULN within the last month
5. Screening platelet count below the lower limit of normal (LLN).
6. Acute illness within 4 weeks prior to first dose of BMN 045 which may interfere with the study assessments.
7. Severe mental retardation or behavioural problems which, in the opinion of the investigator, prohibit participation in this study
8.Severe cardiomyopathy which in the opinion of the investigator prohibits participation in this study. If a subject has a left ventricular ejection fraction <45% at screening, the investigator should discuss inclusion of the subject with the Medical Monitor.
9. Expected need for daytime mechanical ventilation within the next year.
10. Use of anticoagulants, antithrombotics or antiplatelet agents.
11. Use of idebenone or other forms of coenzyme Q10 within 1 month prior to the start of the screening for the study.
12. Use of nutritional or herbal supplements which, in the opinion of the investigator, may influence muscle performance, within 1 month of the study
13. Use of any other investigational product or participation in another trial with
an investigational product, within 6 months prior to the start of the screening
for the study.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Change from baseline in 6MWD after 48 weeks of treatment phase at selected<br /><br>dose.</p><br>