Frequently asked questions – FAQs

This page lists some commonly asked questions about the trial.

Please click on the questions below to learn more about each one.

What is the trial?

A multi-center, phase 2 clinical trial evaluating the safety and tolerability, efficacy, pharmacokinetics (how the body processes the drug) and pharmacodynamics (effects the drug has on the body) of a new investigational drug, PF-06252616, in approximately 105 boys diagnosed with Duchenne Muscular Dystrophy (DMD) who are 6 to 15 years of age and are able to walk.

What was the rationale for choosing the 4 stair climb as the primary endpoint versus the Six-minute Walk Test?

There are several reasons that the 4 stair climb was selected as the primary research endpoint in this trial versus the six-minute walk test which has been used in other DMD studies. Based on what’s currently known and suspected regarding how the investigational drug, PF-06252616, works, functional strength may be a more appropriate measure to evaluate the drug’s efficacy. Climbing the stairs requires more focused leg strength than walking on a flat surface. Also, in previous glucocorticoid steroid studies, the 4 stair climb was used to prove efficacy which was documented as clinically meaningful.

What happens when the trial ends?

If safety and efficacy are demonstrated in this Phase 2 trial, participants who complete this trial may also be invited to participate in an open-label trial (an extension of this trial where the participants and their families will know they are receiving the investigational study drug).

Where is the trial being conducted?

Currently, trial sites are planned in the US, Canada, Japan, the United Kingdom, Italy, Poland, Australia, and Bulgaria.

Reasonable travel and accommodations will be provided or reimbursed.

In cases where a participant is already enrolled and a site is added that is closer to their home, unfortunately, transfer to the new site will not be possible in an effort to preserve the consistency and integrity of the already-collected clinical trial data.