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A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH 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.
Age
6 - 18 years
Sex
MALE
Healthy Volunteers
No
David Geffen School of Medicine at UCLA/UCLA Neurology
Los Angeles, California, United States
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States
Ronald Reagan UCLA Pharmacy
Los Angeles, California, United States
UCLA (David Geffen School of Medicine), Department of Orthopedic Surgery
Los Angeles, California, United States
UCLA Clinical & Translational Research Center
Los Angeles, California, United States
University of California, Davis Medical Center
Sacramento, California, United States
University of Iowa ICTS, Clinical Research Unit
Iowa City, Iowa, United States
Kennedy Krieger Institute Out-patient Center
Baltimore, Maryland, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Start Date
October 13, 2016
Primary Completion Date
November 22, 2018
Completion Date
November 22, 2018
Last Updated
November 23, 2020
59
ACTUAL participants
PF-06252616
BIOLOGICAL
Lead Sponsor
Pfizer
NCT07037862
NCT07160634
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.
View ClinicalTrials.gov Terms and ConditionsNCT06450639