Date: 2018-08-30
Type of
information: Halting of the trial
phase: 2
Announcement: halting of the trial
Company: Pfizer (USA - NY)
Product: domagrozumab -PF-06252616
Action
mechanism:
- monoclonal antibody. PF-06252616 is an infused, anti-myostatin monoclonal antibody. Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it is believed that blocking the activity of myostatin may have potential therapeutic application in treating muscle wasting diseases such as DMD.
- PF-06252616 was granted Orphan Drug designation in July 2012 and Fast Track Designation in November 2012 by the FDA. The European Medicines Agency (EMA) granted the investigational candidate Orphan Medical Product designation in February 2013.
Disease: Duchenne muscular dystrophy (DMD)
Therapeutic
area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country:
Trial
details:
- The Phase 2 double-blind, placebo-controlled, multicenter clinical trial investigated the efficacy and safety of domagrozumab, administered in monthly IV doses, in 121 boys aged 6 to 15 with DMD, regardless of underlying mutation. It was designed as a two-year, placebo-controlled study (with the primary analysis after one year); all subjects used background corticosteroid therapy. The open-label extension study was
designed to evaluate long-term safety and efficacy of domagrozumab. (NCT02310763 and NCT02907619)
Latest
news:
- • On August 30, 2018, Pfizer announced that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and
efficacy study (B5161002) and an open-label extension study (B5161004). The Phase 2 study (B5161002), did not meet its primary efficacy endpoint, which was to demonstrate a difference in the mean change from baseline in 4 Stair Climb (in seconds) following one year of treatment with domagrozumab as compared to placebo in patients with DMD. Further evaluation of the totality of evidence including secondary endpoints did not support a significant treatment effect. The decision comes after a thorough review of data available at the time of the primary analysis, which evaluated all study participants after one year of treatment, as well as those participants who were in the trial beyond one year. The
studies were not terminated for safety reasons. Pfizer will continue to review the data to better understand any insights they may provide, and will share results with the scientific and patient community.
- • On December 17, 2014, Pfizer announced enrollment of the first patient in a multicenter Phase II clinical trial of the investigational compound PF-06252616 in boys with Duchenne muscular dystrophy (DMD). The phase 2 clinical trial will evaluate the safety, tolerability and efficacy of PF-06252616.
Is
general: Yes
