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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02958202 |
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Date of registration:
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27/10/2016 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
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Scientific title:
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A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy |
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Date of first enrolment:
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April 2016 |
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Target sample size:
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7 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT02958202 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Belgium
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Italy
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Netherlands
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Sweden
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Contacts
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Name:
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Paulatsya Joshi |
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Address:
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Telephone:
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Email:
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Affiliation:
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BioMarin Pharmaceutical |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Subjects previously treated with BMN 044 or a comparator treatment in a BMN 044
Sponsored Study or Investigator Initiated Trial and who are not eligible for another
ongoing BMN 044 study.
- Continued use of glucocorticosteroids for a minimum of 60 days prior to study entry
with a reasonable expectation that the subject will remain on glucocorticosteroids for
the duration of this study.
- Willing and able to comply with all study requirements and procedures.
- Willing and able to provide written, signed informed consent, or in the case of
subjects under the age of 18 years(or 16 years, depending on the region), provide
written assent (if required) and written informed consent by a legally authorized
representative after the nature of the study has been explained, and prior to the
conduct of any research-related procedures.
Exclusion Criteria:
- Subjects who have previously been treated with BMN 044 who had a serious adverse
experience or met safety stopping criteria, that remains unresolved, which in the
opinion of the Investigator could have been attributable to BMN 044.
- History of significant medical disorder which may confound the interpretation of
safety data
- Acute illness within 4 weeks prior to the first dose of BMN 044 (Week 1) which may
interfere with the measurements.
- Symptomatic cardiomyopathy.
- Baseline aPTT above the upper limit of normal (ULN).
- Baseline platelet count below the lower limit of normal (LLN).
- Use of anti coagulants, anti thrombotics or anti platelet agents within 28 days of the
baseline visit.
- Prior use of any investigational product (other than BMN 044) or investigational
medical device must be discussed with the Medical Monitor prior to screening.
- Current or history of drug and/or alcohol abuse.
Age minimum:
5 Years
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Drug: BMN 044 IV 9 mg/kg
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Drug: BMN 044 IV 6 mg/kg
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Drug: BMN 044 SC 6 mg/kg
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Primary Outcome(s)
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Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing
[Time Frame: Through study completion, an average of 1 year]
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Secondary ID(s)
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2015-003681-87
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BMN-044-201
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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