THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYS

• Research type

  Research Study

• Full title

  A MULTICENTRE, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHY

• IRAS ID

  210410

• Contact name

  Amritpalq Rai

• Contact email

  amritpal.rai@parexel.com

• Sponsor organisation

  Pfizer Inc

• Eudract number

  2016-001615-21

• Duration of Study in the UK

  4 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 PF06252616 is an engineered protein, called a monoclonal antibody, and it blocks myostatin which may result in an increase in muscle strength and bulk.

  This is an open label extension (OLE) study to the previously approved main research study (B5161002)which will look primarily at the long term safety of the study drug, PF-06252616, which the participants have been receiving in the main research study.

  All participants who enrolled and completed through Week 97 of the main study, B5161002, will be eligible for enrollment in the OLE study.

  All participants enrolled in the study will be given the study drug, which will be given as an Intravenous Infusion(IV).

  Participants individual dose will be based on the maximum tolerated dose from the main study (dose of drug that had no/limited unwanted side effects). Participants will be dosed with one of three PF-06252616 dose levels:
  •5 mg/kg
  •20 mg/kg
  •40 mg/kg
  Dose levels in this OLE study may be adjusted depending on safety and other information from the main study.

  The study drug will be administered once a month (approximately every 4 weeks).

  Depending on when participants begin the extension study, participation in the study may be as long as 4 years. Participants will receive monthly infusions of the study drug, and will take part in other assessments.

  In Year 1, they will need to visit the study site approximately 14 times (including the screening visit). In Years 2, 3 and 4, they will need to visit the study once a month (50 times over 4 years)

  To get the best test results, some testing will need to be carried out in a certain order at about the same time of day. Visits which require both imaging and functional testing may be conducted over 2 days.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  16/YH/0461

• Date of REC Opinion

  12 Dec 2016

• REC opinion

  Further Information Favourable Opinion