Clinical Trial: An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Multicenter, Open-label Extension Study To Evaluate The Long Term Safety Of Pf-06252616 In Boys With Duchenne Muscular Dystrophy

Brief Summary: This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo comparator. Subjects will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional capacity evaluations (4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing, six minute walk test and pulmonary function tests) and pharmacokinetic testing.

Detailed Summary:
Sponsor: Pfizer

Current Primary Outcome:

  • Incidence and/or rate of intolerability or dose limiting treatment related adverse events [ Time Frame: Baseline up to 4 years ]
  • Incidence and/or rate, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs. [ Time Frame: Baseline up to 4 years ]
  • Incidence and magnitude of abnormal laboratory findings. [ Time Frame: Baseline up to 4 years ]
  • Abnormal and clinically relevant changes in liver MRI and physical examinations. [ Time Frame: Baseline up to 4 years ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Mean change from baseline in functional capacity assessments [ Time Frame: Baseline up to 4 years ]
  • Mean change from baseline in pulmonary function tests [ Time Frame: Baseline up to 4 years ]
  • Mean change from baseline in muscle strength measured by myometry [ Time Frame: Baseline up to 4 years ]
  • Pharmacokinetic: Trough serum concentrations for all subjects receiving active drug. [ Time Frame: Baseline up to 4 years ]
  • Immunogenicity: Incidence of neutralising and anti-drug antibodies [ Time Frame: Baseline up to 4 years ]


Original Secondary Outcome: Same as current

Information By: Pfizer

Dates:
Date Received: September 14, 2016
Date Started: October 13, 2016
Date Completion: March 29, 2021
Last Updated: May 17, 2017
Last Verified: May 2017