Extension Study of Drisapersen in DMD Subjects

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02636686
• Date of registration: 09/12/2015
• Prospective Registration: No
• Primary sponsor: BioMarin Pharmaceutical
• Public title: Extension Study of Drisapersen in DMD Subjects
• Scientific title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.
• Date of first enrolment: November 27, 2015
• Recruitment status: No longer available
• URL: https://clinicaltrials.gov/show/NCT02636686
• Study type: Expanded Access

Countries of recruitment

Argentina, Australia, Belgium, Bulgaria, Czech Republic, Czechia, France, Germany, Israel, Italy, Japan, Korea, Republic of, Netherlands, Norway, Poland, Russian Federation, Spain, Taiwan, Turkey, United Kingdom, United States

Contacts

• Name: Derry Ridgway, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Any subject who has been previously treated with an exon 51 skipping antisense
oligonucleotide (drisapersen or eteplirsen) and is not eligible for another ongoing
drisapersen study. Subjects who withdrew from the previous studies due to meeting
laboratory safety stopping criteria may be eligible to enroll if:

2. The laboratory parameters that led to stopping have resolved; benefit of further
treatment with drisapersen outweighs the risk to the individual subject; and following
consultation with the Medical Monitor.

3. Subjects with DMD mutation/deletion within the dystrophin gene and correctable by
drisapersen-induced DMD exon 51 skipping.

4. Male subjects age >5 at screening in whom the investigator considers treatment with
drisapersen is likely to lead to improvement or prevent worsening of the condition.

5. Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a
reasonable expectation that the subject will remain on glucocorticoids for the
duration of this study. Changes to or cessation of glucocorticoids will be at the
discretion of the investigator conducting this study in consultation with the
subject/parent and Medical Monitor.

6. Willing and able to comply with all study requirements and procedures (with the
exception of those assessments requiring a subject to be ambulant, for those subjects
who have lost ambulation).

7. Able to give informed assent and/or consent in writing by the subject and/or
parent(s)/legal guardian (according to local regulations)

Exclusion Criteria:

1. Subjects who have previously been treated with drisapersen and who had a serious
adverse experience or who met safety stopping criteria that remains unresolved, which
in the opinion of the investigator could have been attributable to drisapersen. Once
resolved, subject may be eligible to enter the study following investigator
consultation with the Medical Monitor.

2. Use of anticoagulants, anti-thrombotics or antiplatelet agents within 28 days of the
first re-dosing of drisapersen. Chronic use of anticoagulants, anti-thrombotics or
antiplatelet 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.

3. Participation in any investigational clinical trial within 3 months prior to start or
during this study (except for other drisapersen studies). If subjects have
participated in any other study within the last 6 months this should be discussed with
the Medical Monitor prior to start of this study.

4. 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)

5. 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.

6. A platelet count under the lower limit of normal (LLN) at start of this study. A
re-test is possible at a later stage, and if within normal range, the subject may
enter the study.



Age minimum: 5 Years
Age maximum: 80 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Drisapersen

Secondary ID(s)

BMN-051-302

Source(s) of Monetary Support

Please refer to primary and secondary sponsors