An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
Status: | Completed |
---|---|
Conditions: | Neurology, Orthopedic |
Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
Healthy: | No |
Age Range: | 6 - 18 |
Updated: | 1/6/2019 |
Start Date: | October 13, 2016 |
End Date: | November 22, 2018 |
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.
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.
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.
We found this trial at
27
sites
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Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
Cincinnati, Ohio 45229
1-513-636-4200
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
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3000 Erwin Road
Durham, North Carolina 27705
Durham, North Carolina 27705
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10833 Le Conte Avenue
Los Angeles, California 90095
Los Angeles, California 90095
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10833 Le Conte Avenue
Los Angeles, California 90095
Los Angeles, California 90095
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2450 Riverside Avenue
Minneapolis, Minnesota 55454
Minneapolis, Minnesota 55454
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2315 Stockton Blvd.
Sacramento, California 95817
Sacramento, California 95817
(916) 734-2011
University of California, Davis Medical Center UC Davis Medical Center serves a 65,000-square-mile area that...
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