DMD: Phase 2 Trial of GSK Exon-Skipping Drug Opens in US

A phase 2 trial of GSK2402968, designed to treat Duchenne MD caused by specific mutations, is now recruiting at 14 sites

Article Highlights:
  • GSK2402968, in development by GlaxoSmithKline and Prosensa, is designed to allow DMD patients with certain mutations in the dystrophin gene to produce functional dystrophin protein through a strategy called exon skipping.
  • The phase 2 study will test two doses of GSK2402968 at 14 U.S. sites. The trial is recruiting 54 boys with DMD who meet certain criteria.
  • In 2011, a small study of GSK2402968 conducted in Europe showed encouraging results with respect to dystrophin protein production, walking distance and safety.
by Margaret Wahl on June 1, 2012 - 4:05pm

Update (April 9, 2013): The U.S.-based phase 2 trial of drisapersen stopped recruiting new participants in January 2013. Results are expected in early 2014.

Update (Aug. 15, 2012): GlaxoSmithKline's exon-skipping drug GSK2402968 has been given a new generic name, "drisapersen."

Pharmaceutical company GlaxoSmithKline has announced the opening of a U.S.-based phase 2 trial of its exon-skipping drug GSK2402968, designed to treat patients with Duchenne muscular dystrophy (DMD) who have specific mutations in the dystrophin gene.

The trial is recruiting 54 boys with DMD who meet certain criteria (see below), at study sites in California, Florida, Iowa, Kansas, Maryland, Minnesota, Missouri, New York, North Carolina, Ohio, Oregon and Texas.

Favorable results reported last year

GSK2402968 is designed to change the way muscle cells interpret genetic instructions for the muscle protein dystrophin, by coaxing them to ignore ("skip") a section of the instructions known as exon 51 and piece together the surrounding instructions.

Also known as PRO051/GSK2402968, the drug is in development by the Dutch company Prosensa and by GlaxoSmithKline.

In April 2011, investigators reported that a 48-week trial of the drug in Belgium and Sweden yielded encouraging results. They saw increased presence of the dystrophin protein in muscle fibers; an increase over baseline measurements of the distance participants could walk in six minutes; and no serious adverse events.

That trial included 12 boys with DMD who received weekly injections of GSK2402968.

New study open at 14 U.S. sites

The new trial is seeking 54 boys with DMD with the following characteristics:

  • at least 5 years old;
  • with a dystrophin mutation potentially treatable by skipping exon 51, which includes deletions in exons 13-50, 29-50, 43-50, 45-50, 47-50, 48-50, 49-50, 50, 52 and 52-63;
  • able to walk 75 meters (about 246 feet) in six minutes;
  • able to rise from the floor in seven seconds or less;
  • taking oral glucocorticoids (corticosteroids) for a minimum of six months prior to screening, with no significant change in daily dosage or dosing regimen for a minimum of three months prior to screening;
  • without significant heart disease; and
  • meeting other study criteria.

Participants will be assigned to receive one of two weekly dosage levels of GSK2402968, or a placebo.

For details and contact information, see A Clinical Study to Assess Two Doses of GSK2402968 in Subjects with Duchenne Muscular Dystrophy, or go to and enter NCT01462292 in the search box.

For more information

To learn more about exon skipping in general and GSK2402968 specifically, see:

About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to

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