NL-OMON43779
Completed
Phase 2
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 - PRO045-CLIN-01
BioMarin Parmaceutical Inc.0 sites5 target enrollmentTBD
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Duchenne muscular dystrophy
- Sponsor
- BioMarin Parmaceutical Inc.
- Enrollment
- 5
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
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.
Outcomes
Primary Outcomes
Not specified
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