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Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

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. Identifier: NCT02958202
Recruitment Status : Terminated (Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.)
First Posted : November 8, 2016
Last Update Posted : January 26, 2018
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: BMN 044 IV 6 mg/kg Drug: BMN 044 IV 9 mg/kg Drug: BMN 044 SC 6 mg/kg Phase 2

Detailed Description:

This is a phase 2 multi center, multi national, open label, long term extension study. Up to approximately 50 male subjects with Duchenne Muscular Dystrophy (DMD) who have previously been treated with BMN 044 will be enrolled. Subjects will receive either IV infusions or SC injections at pre-defined doses.

Safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy assessments will be conducted at regular intervals throughout the study.

Subjects will be permitted to continue in this study until the subject meets any of the defined withdrawal criteria, BioMarin decides to halt the clinical development of BMN 044, or BMN 044 receives marketing authorization.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : April 2016
Actual Primary Completion Date : September 2016
Actual Study Completion Date : September 2016

Arm Intervention/treatment
Experimental: BMN 044 IV 6 mg/kg
Weekly intravenous (IV) dosing with 6 mg/kg
Drug: BMN 044 IV 6 mg/kg
Other Name: PRO044

Experimental: BMN 044 IV 9 mg/kg
Weekly intravenous (IV) dosing with 9 mg/kg
Drug: BMN 044 IV 9 mg/kg
Other Name: PRO044

Experimental: BMN 044 SC 6 mg/kg
Weekly subcutaneous (SC) dosing with 6 mg/kg
Drug: BMN 044 SC 6 mg/kg
Other Name: PRO044

Primary Outcome Measures :
  1. Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing [ Time Frame: Through study completion, an average of 1 year ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02958202

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Universitair Ziekenhuis Leuven
Leuven, Belgium
S.Anna Hospital
Ferrara, Italy
Policlinico Univsersitario Agostino Gemelli
Rome, Italy
Leiden University Medical Center
Leiden, Netherlands, 2333 ZA
Drottning Silvias Barn- ochungdomssjukhus
Goteborg, Sweden
Sponsors and Collaborators
BioMarin Pharmaceutical
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Study Director: Paulatsya Joshi BioMarin Pharmaceutical
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Responsible Party: BioMarin Pharmaceutical Identifier: NCT02958202    
Other Study ID Numbers: BMN-044-201
2015-003681-87 ( EudraCT Number )
First Posted: November 8, 2016    Key Record Dates
Last Update Posted: January 26, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by BioMarin Pharmaceutical:
Duchenne Muscular Dystrophy
BMN 044
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked