A study to test if multiple injections of BMN 045 under the skin are safe and effective in people who suffer from Duchenne muscular dystrophy
- Conditions
- Duchenne muscular dystrophy resulting from a mutation correctable by BMN 045-induced DMD exon 45 skippingMedDRA version: 18.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2011-005040-10-GB
- Lead Sponsor
- BioMarin Pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 15
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).
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.
Adequate quality for biopsy (confirmed with MRI) of the lateral head of the gastrocnemius muscle. An alternative muscle may be considered for biopsy but only following discussion between the Principal Investigator and the Prosensa Medical Monitor.
Life expectancy of at least 3 years after inclusion in the study.
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.
Willing and able to adhere to the study visit schedule and other protocol requirements.
Written informed consent signed (by parent(s)/legal guardian and/or the subject, according to the local regulations).
In France, a subject will be eligible for inclusion in this study only if either affiliated to, or a beneficiary of, a social security category.
Are the trial subjects under 18? yes
Number of subjects for this age range: 45
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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).
Current or history of liver disease or impairment.
Current or history of renal disease or impairment.
At least two aPTT above ULN within the last month.
Screening platelet count below the lower limit of normal (LLN).
Acute illness within 4 weeks prior to first dose of BMN 045 which may interfere with the study assessments.
Severe mental retardation or behavioural problems which, in the opinion of the investigator, prohibit participation in this study.
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.
Expected need for daytime mechanical ventilation within the next year.
Use of anticoagulants, antithrombotics or antiplatelet agents.
Use of idebenone or other forms of coenzyme Q10 within 1 month prior to the start of the screening for the study.
Use of nutritional or herbal supplements which, in the opinion of the investigator, may influence muscle performance, within1 month of the study
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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method