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

Duchenne Muscular Dystrophy Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02958202
• Other study ID #: BMN-044-201
• Secondary ID: 2015-003681-87
• Status: Terminated
• Phase: Phase 2
• First received: October 27, 2016
• Last updated: January 23, 2018
• Start date: April 2016
• Est. completion date: September 2016

Study information

• Verified date: January 2018
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 7
• Est. completion date: September 2016
• Est. primary completion date: September 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• BMN 044 IV 6 mg/kg

• BMN 044 IV 9 mg/kg

• BMN 044 SC 6 mg/kg

Locations

Country	Name	City	State
Belgium	Universitair Ziekenhuis Leuven	Leuven
Italy	S.Anna Hospital	Ferrara
Italy	Policlinico Univsersitario Agostino Gemelli	Rome
Netherlands	Leiden University Medical Center	Leiden
Sweden	Drottning Silvias Barn- ochungdomssjukhus	Goteborg

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

Belgium,  Italy,  Netherlands,  Sweden, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing		Through study completion, an average of 1 year