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

NCT02958202 | Terminated Phase 2 DMC

• 2 other identifiers: BMN-044-2012015-003681-87
• Study Type: interventional
• Target: 7
• Locations: 4 countries
• Sites: 5

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; DMD; Dystrophin; BMN 044; PRO044; BioMarin

Outcome Measures

Primary Outcomes (1)

• Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing

  Through study completion, an average of 1 year

Study Arms (3)

BMN 044 IV 6 mg/kg

EXPERIMENTAL

Weekly intravenous (IV) dosing with 6 mg/kg

Drug: BMN 044 IV 6 mg/kg

BMN 044 IV 9 mg/kg

EXPERIMENTAL

Weekly intravenous (IV) dosing with 9 mg/kg

Drug: BMN 044 IV 9 mg/kg

BMN 044 SC 6 mg/kg

EXPERIMENTAL

Weekly subcutaneous (SC) dosing with 6 mg/kg

Drug: BMN 044 SC 6 mg/kg

Interventions

BMN 044 IV 6 mg/kg DRUG

Also known as: PRO044

BMN 044 IV 6 mg/kg

BMN 044 IV 9 mg/kg DRUG

Also known as: PRO044

BMN 044 IV 9 mg/kg

BMN 044 SC 6 mg/kg DRUG

Also known as: PRO044

BMN 044 SC 6 mg/kg

Eligibility Criteria

• Age: 5 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (5)

Universitair Ziekenhuis Leuven

Leuven, Belgium

Location

S.Anna Hospital

Ferrara, Italy

Location

Policlinico Univsersitario Agostino Gemelli

Rome, Italy

Location

Leiden University Medical Center

Leiden, 2333 ZA, Netherlands

Location

Drottning Silvias Barn- ochungdomssjukhus

Gothenburg, Sweden

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Paulatsya Joshi

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 27, 2016
• First Posted: November 8, 2016
• Study Start: April 1, 2016
• Primary Completion: September 1, 2016
• Study Completion: September 1, 2016
• Last Updated: January 26, 2018

Record last verified: 2018-01

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1); IT (2); SE (1); BE (1)