A study to assess the effect of BMN 044 in subjects with Duchenne muscular dystrophy (Extension study)

Phase 1

Conditions: Duchenne muscular dystrophy
MedDRA version: 18.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Registration Number: EUCTR2015-003681-87-BE

Lead Sponsor: BioMarin Pharmaceutical Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 50

Inclusion Criteria

1. 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. Subjects who withdrew from any previous BMN 044 study due to meeting laboratory safety stopping criteria may be eligible to enroll if the applicable laboratory parameter has resolved to be within normal limits or parent study baseline value, and benefit of further treatment with BMN 044 outweighs the risk to the individual subject, as agreed in consultation with the Medical Monitor.
2. 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. Changes to or cessation of glucocorticosteroids will be at the discretion of the Investigator conducting this study in consultation with the subject/parent and Medical Monitor. Subjects who have discontinued glucocorticosteroids in their previous BMN 044 study, following consultation with the Medical Monitor may be enrolled.
3. Willing and able to comply with all study requirements and procedures.
4. 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.

Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. 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. Once resolved, subject may be eligible to enter the study following Investigator consultation with the Medical Monitor.
2. History of significant medical disorder which may confound the interpretation of safety data (e.g. current or history of renal or liver disease/impairment, history of inflammatory illness, bleeding complications, mental retardation and/or behavioral problems).
3. Acute illness within 4 weeks prior to the first dose of BMN 044 (Week 1) which may interfere with the measurements.
4. Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction <45% at start of this study, the Investigator should discuss inclusion of subject in this study with the Medical Monitor.
5. Baseline aPTT above the upper limit of normal (ULN). A re test is possible at a later stage, and if within normal range, the subject may enter the study.
6. Baseline platelet count below the lower limit of normal (LLN). A re test is possible at a later stage, and if within normal range, the subject may enter the study.
7. Use of anti coagulants, anti thrombotics or anti platelet agents within 28 days of the baseline visit. Chronic use of anti coagulants, anti thrombotics or anti platelet agents is prohibited during the study. As needed dosing (pro re nata – PRN) may be acceptable (except for aspirin) following discussion with the Medical Monitor.
8. Prior use of any investigational product (other than BMN 044) or investigational medical device must be discussed with the Medical Monitor prior to screening.
9. Current or history of drug and/or alcohol abuse.