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

Phase 1

• Conditions: Duchenne muscular dystrophy; MedDRA version: 20.0Level: 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-IT
• Lead Sponsor: BIOMARIN PHARMACEUTICAL INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2016-09-12
• Study Start: 2021-01-19
• Last Update Posted: 10 August 2021

Recruitment & Eligibility

• Status: A
• Sex: Male
• Target Recruitment: 7

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 researchrelated
procedures.
Are the trial subjects under 18? yes
Number of subjects for this age range:
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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified