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NCT02907619

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

Last Updated: 2020-11-23

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

59 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-10-13

Study Completion Date

2018-11-22

Brief Summary

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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 Description

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Conditions

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Duchenne Muscular Dystrophy

Keywords

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Duchenne muscular dystrophy, myostatin, open-label

Study Design

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Allocation Method

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PF-06252616

Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002

Group Type EXPERIMENTAL

PF-06252616

Intervention Type BIOLOGICAL

Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002

Interventions

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PF-06252616

Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.

Minimum Eligible Age

6 Years

Maximum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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David Geffen School of Medicine at UCLA/UCLA Neurology

Los Angeles, California, United States

Site Status

Ronald Reagan UCLA Medical Center

Los Angeles, California, United States

Site Status

Ronald Reagan UCLA Pharmacy

Los Angeles, California, United States

Site Status

UCLA (David Geffen School of Medicine), Department of Orthopedic Surgery

Los Angeles, California, United States

Site Status

UCLA Clinical & Translational Research Center

Los Angeles, California, United States

Site Status

University of California, Davis Medical Center

Sacramento, California, United States

Site Status

University of Iowa ICTS, Clinical Research Unit

Iowa City, Iowa, United States

Site Status

Kennedy Krieger Institute Out-patient Center

Baltimore, Maryland, United States

Site Status

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Johns Hopkins Investigational Drug Service

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, United States

Site Status

(For Drug deliveries) IDS Pharmacy

Minneapolis, Minnesota, United States

Site Status

St. Louis Childrens's Hospital

St Louis, Missouri, United States

Site Status

Duke University Medical Center, Lenox Baker Children's Hospital

Durham, North Carolina, United States

Site Status

Duke University, Investigational Drug Pharmacy

Durham, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status