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

January 23, 2018 updated by: BioMarin Pharmaceutical

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Actual)

7

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Leuven, Belgium
        • Universitair Ziekenhuis Leuven
  • Italy
      • Ferrara, Italy
        • S.Anna Hospital
      • Rome, Italy
        • Policlinico Univsersitario Agostino Gemelli
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Leiden University Medical Center
  • Sweden
      • Goteborg, Sweden
        • Drottning Silvias Barn- ochungdomssjukhus

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / 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 Names:
  • PRO044
EXPERIMENTAL: BMN 044 IV 9 mg/kg
Weekly intravenous (IV) dosing with 9 mg/kg
Drug: BMN 044 IV 9 mg/kg
Other Names:
  • PRO044
EXPERIMENTAL: BMN 044 SC 6 mg/kg
Weekly subcutaneous (SC) dosing with 6 mg/kg
Drug: BMN 044 SC 6 mg/kg
Other Names:
  • PRO044

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Investigators

  • Study Director: Paulatsya Joshi, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2016

Primary Completion (ACTUAL)

September 1, 2016

Study Completion (ACTUAL)

September 1, 2016

Study Registration Dates

First Submitted

October 27, 2016

First Submitted That Met QC Criteria

November 4, 2016

First Posted (ESTIMATE)

November 8, 2016

Study Record Updates

Last Update Posted (ACTUAL)

January 26, 2018

Last Update Submitted That Met QC Criteria

January 23, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BMN-044-201
  • 2015-003681-87 (EUDRACT_NUMBER)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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