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

Primary Purpose

Duchenne Muscular Dystrophy

Status

Terminated

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

BMN 044 IV 6 mg/kg

BMN 044 IV 9 mg/kg

BMN 044 SC 6 mg/kg

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne Muscular Dystrophy, DMD, Dystrophin, BMN 044, PRO044, BioMarin

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

Universitair Ziekenhuis Leuven
S.Anna Hospital
Policlinico Univsersitario Agostino Gemelli
Leiden University Medical Center
Drottning Silvias Barn- ochungdomssjukhus

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

BMN 044 IV 6 mg/kg

BMN 044 IV 9 mg/kg

BMN 044 SC 6 mg/kg

Arm Description

Weekly intravenous (IV) dosing with 6 mg/kg

Weekly intravenous (IV) dosing with 9 mg/kg

Weekly subcutaneous (SC) dosing with 6 mg/kg

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Full Information

NCT ID

NCT02958202

First Posted

October 27, 2016

Last Updated

January 23, 2018

Sponsor

BioMarin Pharmaceutical

1. Study Identification

Unique Protocol Identification Number

NCT02958202

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

January 2018

Overall Recruitment Status

Terminated

Why Stopped

Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.

Study Start Date

April 2016 (undefined)

Primary Completion Date

September 2016 (Actual)

Study Completion Date

September 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

BioMarin Pharmaceutical

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

7 (Actual)

8. Arms, Groups, and Interventions

Arm Title

BMN 044 IV 6 mg/kg

Arm Type

Experimental

Arm Description

Weekly intravenous (IV) dosing with 6 mg/kg

Arm Title

BMN 044 IV 9 mg/kg

Arm Type

Experimental

Arm Description

Weekly intravenous (IV) dosing with 9 mg/kg

Arm Title

BMN 044 SC 6 mg/kg

Arm Type

Experimental

Arm Description

Weekly subcutaneous (SC) dosing with 6 mg/kg

Intervention Type

Drug

Intervention Name(s)

BMN 044 IV 6 mg/kg

Other Intervention Name(s)

PRO044

Intervention Type

Drug

Intervention Name(s)

BMN 044 IV 9 mg/kg

Other Intervention Name(s)

PRO044

Intervention Type

Drug

Intervention Name(s)

BMN 044 SC 6 mg/kg

Other Intervention Name(s)

PRO044

Primary Outcome Measure Information:

Title

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

Time Frame

Through study completion, an average of 1 year

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Paulatsya Joshi

Organizational Affiliation

BioMarin Pharmaceutical

Official's Role

Study Director

Facility Information:

Facility Name

Universitair Ziekenhuis Leuven

City

Leuven

Country

Belgium

Facility Name

S.Anna Hospital

City

Ferrara

Country

Italy

Facility Name

Policlinico Univsersitario Agostino Gemelli

City

Rome

Country

Italy

Facility Name

Leiden University Medical Center

City

Leiden

ZIP/Postal Code

2333 ZA

Country

Netherlands

Facility Name

Drottning Silvias Barn- ochungdomssjukhus

City

Goteborg

Country

Sweden

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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