PF-06252616 in Boys with Duchenne Muscular Dystrophy

• Research type

  Research Study

• Full title

  A PHASE 2 RANDOMISED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY

• IRAS ID

  161739

• Contact name

  Marwa Ali

• Contact email

  marwa.ali@parexel.com

• Sponsor organisation

  Pfizer Inc

• Eudract number

  2014-002072-92

• Clinicaltrials.gov Identifier

  113,863, FDA IND Number

• Duration of Study in the UK

  3 years, 0 months, 0 days

• Research summary

  Duchenne muscular dystrophy (DMD) is a genetic disease of muscles which predominantly affects boys. DMD is characterised by muscle degeneration throughout the body including the heart. Most boys lose the ability to walk between the ages of 10 and 14. Heart and breathing problems become more common with age. Boys with DMD usually die in their late teens or early twenties.

  There is no treatment that can stop or reverse the progression of DMD. Disease management consists primarily of alleviating the symptoms including physical therapy, orthotics, surgery and use of wheelchair. Glucocorticoid therapy may delay the course of the disease; however there are some severe side effects.

  Myostatin is a naturally occurring protein that prevents muscles from growing too large. The study drug PF-06252616 is an engineered protein, called a monoclonal antibody, and it blocks myostatin which may result in an increase in muscle strength and bulk.

  Pfizer is sponsoring a study to look at how the bodies of boys between 6 and less than 10 years old and diagnosed with DMD respond to PF-06252616. This will be a phase 2, randomised, placebo-controlled, double-blind, multiple ascending dose study. Study participants will receive PF-06252616 and may receive placebo, which does not contain any drug. Neither the study doctor nor the study participant will know whether PF-06252616 or placebo is given. After safety reviews, the study participant may be moved to higher dose levels of PF-06252616.

  The study participants will be randomly assigned to one of three treatment arms divided into two treatment periods of 48 weeks each:

  Treatment arm 1:
  Period 1: PF-06252616 at the dose levels 5, 20, and 40 mg/kg
  Period 2: Highest tolerated dose of PF-06252616 received in Period 1

  Treatment arm 2:
  Period 1: PF-06252616 at the dose levels 5, 20, and 40 mg/kg
  Period 2: Placebo

  Treatment arm 3:
  Period 1: Placebo
  Period 2: PF-06252616 at the dose levels 5, 20, and 40 mg/kg

  Therefore, all participants will receive at least 48 weeks of treatment with PF-06252616 over the 2 years of dosing in the study.

  This study will take place across North America, Asia and Europe. Approximately 105 study participants will be recruited worldwide, with approximately 8 in the UK.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  14/YH/1282

• Date of REC Opinion

  30 Dec 2014

• REC opinion

  Further Information Favourable Opinion