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Trial record 41 of 1003 for:    BMD

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.
 
ClinicalTrials.gov 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
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date  ICMJE October 27, 2016
First Posted Date  ICMJE November 8, 2016
Last Update Posted Date January 26, 2018
Study Start Date  ICMJE April 2016
Actual Primary Completion Date September 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 9, 2016)
Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing [ Time Frame: Through study completion, an average of 1 year ]
Original Primary Outcome Measures  ICMJE
 (submitted: November 4, 2016)
Long term safety and tolerability of BMN 044 [ Time Frame: Through study completion, an average of 1 year ]
Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing
Change History Complete list of historical versions of study NCT02958202 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
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 Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
Official Title  ICMJE 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
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: BMN 044 IV 6 mg/kg
    Other Name: PRO044
  • Drug: BMN 044 IV 9 mg/kg
    Other Name: PRO044
  • Drug: BMN 044 SC 6 mg/kg
    Other Name: PRO044
Study Arms  ICMJE
  • Experimental: BMN 044 IV 6 mg/kg
    Weekly intravenous (IV) dosing with 6 mg/kg
    Intervention: Drug: BMN 044 IV 6 mg/kg
  • Experimental: BMN 044 IV 9 mg/kg
    Weekly intravenous (IV) dosing with 9 mg/kg
    Intervention: Drug: BMN 044 IV 9 mg/kg
  • Experimental: BMN 044 SC 6 mg/kg
    Weekly subcutaneous (SC) dosing with 6 mg/kg
    Intervention: Drug: BMN 044 SC 6 mg/kg
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: November 4, 2016)
7
Original Actual Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE September 2016
Actual Primary Completion Date September 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Italy,   Netherlands,   Sweden
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02958202
Other Study ID Numbers  ICMJE BMN-044-201
2015-003681-87 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party BioMarin Pharmaceutical
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Paulatsya Joshi BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP