An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: PF-06252616
Intervention description: Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Masking: None (Open Label)
Enrollment: 59.0
Sex: Male
Minimum age: 6 Years
Maximum age: 18 Years
Healthy volunteers: No
Keywords: Duchenne muscular dystrophy, myostatin, open-label

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.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Subjects with Duchenne muscular dystrophy who enrolled and completed study B5161002.

          2. Signed and dated informed consent document (ICD) indicating that the subject's parent
             or legal guardian/caregiver has been informed of all pertinent aspects of the study.

          3. Subjects and their legal guardians/caregivers who are willing and able to comply with
             scheduled visits, treatment plan, laboratory tests, and other study procedures.

          4. Subject have;

               1. Adequate hepatic function on screening laboratory assessments

               2. GLDH less than 20 units/liter (2 x upper limit of normal [ULN])

               3. Iron content estimate on the liver MRI within the normal range.

        Exclusion Criteria:

          1. Unwilling or unable (eg, metal implants) to undergo examination with closed MRI.

          2. All male subjects who are able to father children and are sexually active and at risk
             for impregnating a female partner, who are unwilling or unable to use a highly
             effective method of contraception. In addition, all sexually active male subjects who
             are unwilling or unable to prevent potential transfer of and exposure to drug through
             semen to their partners by using a condom consistently and correctly. .

          3. Subjects who are investigational site staff members directly involved in the conduct
             of the study and their family members, site staff members otherwise supervised by the
             Investigator, or subjects who are related to Pfizer employees directly involved in the
             conduct of the study.

          4. Other severe acute or chronic medical or psychiatric condition or laboratory
             abnormality that may increase the risk associated with study participation.

          5. Participation in other studies involving investigational drug(s), with the exception
             of B5161002.

          6. History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or
             additives of this investigational product.