Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol

Not Recruiting

Trial ID: NCT01890798


This is a single arm, open-label continued access protocol of drisapersen for the treatment of male subjects with Duchenne muscular dystrophy (DMD) having dystrophin mutations correctable by drisapersen-induced DMD Exon 51 skipping. The purpose of this continued access protocol is to offer pre-approval access to drisapersen for the treatment of subjects with DMD who previously participated in eligible drisapersen studies. The protocol will collect safety data required to assure subject safety and periodic efficacy data on muscle function.

Official Title

A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study

Stanford Investigator(s)

Yuen So, MD, PhD
Yuen So, MD, PhD

Professor of Neurology


Inclusion Criteria:

   - Prior DMD114876 subjects: Subjects who completed both the 24 week double-blind
   treatment and 24 week post-treatment phases in study DMD114876, OR Subjects who
   withdrew from the treatment portion of study DMD114876 due to meeting laboratory
   safety stopping criteria may be eligible to enroll in the extension study if: 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 GSK Medical Lead.

   - Prior DMD115501 subjects:

   - Active subjects who entered into open-label extension study DMD115501. Subjects are
   required to be withdrawn from DMD115501 to participate.

   - Prior DMD114044 Subjects: US citizens who completed study DMD114044 in another country
   and who want to return to the US to participate, upon agreement by a physician
   conducting this protocol, OR Unites States citizens who participated in DMD114044 but
   who had to withdraw from the study due to meeting laboratory safety stopping criteria
   may be eligible to enroll if: 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 GSK Medical Lead and upon
   agreement of physician conducting this protocol

   - Prior DMD114349 Subjects: US citizens who participated in and completed study
   DMD114044 in another country and who entered into the ongoing open-label extension
   study DMD114349 in a country outside the US who wish to withdraw from DMD114349 and
   return to the US to participate in this protocol, upon agreement by a physician
   conducting this protocol. Subjects are required to withdraw from DMD114349 to
   participate in this protocol.

   - Baseline platelets of 150 x 109/Liters (L) or greater and no history of

   - Continued use of glucocorticosteroids for a minimum of 60 days prior to protocol entry
   with a reasonable expectation that the subject will remain on steroids for the
   duration of the protocol. Changes to or cessation of glucocorticosteroids will be at
   the discretion of the physician conducting this protocol in consultation with the
   subject/parent. The GSK Medical Lead must be notified in a timely manner.

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

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

Exclusion Criteria:

   - Subject had a serious adverse experience or who met safety stopping criteria that
   remains unresolved from protocol DMD114876, which in the opinion of the physician
   conducting this protocol could have been attributable to study medication, and which
   is ongoing. Once resolved, subject may be eligible to enroll following consultation
   with the GlaxoSmithKline (GSK) Medical Lead.

   - Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment
   with investigational drugs, except for drisapersen, within 28 days of the first
   administration of drisapersen.


drug: Drisapersen

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Shirley Paulose