Trial record 1 of 24 for:
Duchenne netherlands dmd
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01037309 |
Recruitment Status :
Completed
First Posted : December 23, 2009
Results First Posted : April 9, 2015
Last Update Posted : October 16, 2018
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Sponsor:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
BioMarin Pharmaceutical
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Tracking Information | |||||||||||||
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First Submitted Date ICMJE | December 21, 2009 | ||||||||||||
First Posted Date ICMJE | December 23, 2009 | ||||||||||||
Results First Submitted Date ICMJE | September 1, 2014 | ||||||||||||
Results First Posted Date ICMJE | April 9, 2015 | ||||||||||||
Last Update Posted Date | October 16, 2018 | ||||||||||||
Study Start Date ICMJE | December 2009 | ||||||||||||
Actual Primary Completion Date | May 2013 (Final data collection date for primary outcome measure) | ||||||||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | |||||||||||||
Current Secondary Outcome Measures ICMJE |
PRO044 Pharmacokinetic Cmax (μg/mL) Following Subcutaneous Administration [ Time Frame: Week 1, Week 5 ] Pharmacokinetic population evaluated for maximum plasma concentration (Cmax)
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Original Secondary Outcome Measures ICMJE | Not Provided | ||||||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||||||
Descriptive Information | |||||||||||||
Brief Title ICMJE | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | ||||||||||||
Official Title ICMJE | A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy | ||||||||||||
Brief Summary | The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein. | ||||||||||||
Detailed Description | To assess the effect of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To assess the safety and tolerability of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To determine the pharmacokinetics of PRO044 at different dose levels after subcutaneous and intravenous administration in subjects with Duchenne muscular dystrophy. | ||||||||||||
Study Type ICMJE | Interventional | ||||||||||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Duchenne Muscular Dystrophy | ||||||||||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||||||
Recruitment Status ICMJE | Completed | ||||||||||||
Actual Enrollment ICMJE |
18 | ||||||||||||
Original Estimated Enrollment ICMJE |
12 | ||||||||||||
Actual Study Completion Date ICMJE | October 2013 | ||||||||||||
Actual Primary Completion Date | May 2013 (Final data collection date for primary outcome measure) | ||||||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 5 Years to 16 Years (Child) | ||||||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||||||||
Listed Location Countries ICMJE | Netherlands, Belgium, Italy, Sweden | ||||||||||||
Removed Location Countries | |||||||||||||
Administrative Information | |||||||||||||
NCT Number ICMJE | NCT01037309 | ||||||||||||
Other Study ID Numbers ICMJE | PRO044-CLIN-01 | ||||||||||||
Has Data Monitoring Committee | Yes | ||||||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||||||||||
Responsible Party | BioMarin Pharmaceutical | ||||||||||||
Study Sponsor ICMJE | BioMarin Pharmaceutical | ||||||||||||
Collaborators ICMJE | Not Provided | ||||||||||||
Investigators ICMJE |
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PRS Account | BioMarin Pharmaceutical | ||||||||||||
Verification Date | September 2018 | ||||||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |