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An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
study id #: NCT02907619
condition: Duchenne Muscular Dystrophy
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.
mechanism of action: Monoclonal anti-myostatin to promote muscle growth and improve function
last updated: January 07, 2019
start date: October 13, 2016
estimated completion: November 22, 2018
phase of development: Phase 2
size / enrollment: 105
- 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 ]
- 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 ]
• Subjects with Duchenne muscular dystrophy who enrolled and completed study B5161002.
• 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.
• Subjects and their legal guardians/caregivers who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
• Subject have;
-Adequate hepatic function on screening laboratory assessments
-GLDH less than 20 units/liter (2 x upper limit of normal [ULN])
-Iron content estimate on the liver MRI within the normal range.
• Unwilling or unable (eg, metal implants) to undergo examination with closed MRI.
• 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.
• 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.
• Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation.
• Participation in other studies involving investigational drug(s), with the exception of B5161002.
• History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of this investigational product.
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