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

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ClinicalTrials.gov Identifier: NCT02907619
Recruitment Status : Completed
First Posted : September 20, 2016
Last Update Posted : December 12, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief 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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Biological: PF-06252616 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 105 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-label Extension Study To Evaluate The Long Term Safety Of Pf-06252616 In Boys With Duchenne Muscular Dystrophy
Actual Study Start Date : October 13, 2016
Actual Primary Completion Date : November 14, 2018
Actual Study Completion Date : November 14, 2018


Arm Intervention/treatment
Experimental: PF-06252616
Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002
Biological: PF-06252616
Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002




Primary Outcome Measures :
  1. Incidence and/or rate of intolerability or dose limiting treatment related adverse events [ Time Frame: Baseline up to 4 years ]
  2. 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 ]
  3. Incidence and magnitude of abnormal laboratory findings. [ Time Frame: Baseline up to 4 years ]
  4. Abnormal and clinically relevant changes in liver MRI and physical examinations. [ Time Frame: Baseline up to 4 years ]

Secondary Outcome Measures :
  1. Mean change from baseline in functional capacity assessments [ Time Frame: Baseline up to 4 years ]
  2. Mean change from baseline in pulmonary function tests [ Time Frame: Baseline up to 4 years ]
  3. Mean change from baseline in muscle strength measured by myometry [ Time Frame: Baseline up to 4 years ]
  4. Pharmacokinetic: Trough serum concentrations for all subjects receiving active drug. [ Time Frame: Baseline up to 4 years ]
  5. Immunogenicity: Incidence of neutralising and anti-drug antibodies [ Time Frame: Baseline up to 4 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02907619


  Hide Study Locations
Locations
United States, California
David Geffen School of Medicine at UCLA/UCLA Neorology
Los Angeles, California, United States, 90095
Ronald Reagan UCLA Medical Center
Los Angeles, California, United States, 90095
Ronald Reagan UCLA Pharmacy
Los Angeles, California, United States, 90095
UCLA (David Geffen School of Medicine), Department of Orthopedic Surgery
Los Angeles, California, United States, 90095
UCLA Clinical & Translational Research Center
Los Angeles, California, United States, 90095
University of California, Davis Medical Center
Sacramento, California, United States, 95817
United States, Iowa
University of Iowa ICTS, Clinical Research Unit
Iowa City, Iowa, United States, 52242
United States, Maryland
Kennedy Krieger Institute Out-patient Center
Baltimore, Maryland, United States, 21205
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Johns Hopkins Investigational Drug Service
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Minnesota
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55455
United States, Missouri
St. Louis Childrens's Hospital
Saint Louis, Missouri, United States, 63110
United States, North Carolina
Duke Neurology Clinical Research
Durham, North Carolina, United States, 27705
Duke University Medical Center, Lenox Baker Children's Hospital
Durham, North Carolina, United States, 27705
Duke University, Investigational Drug Pharmacy
Durham, North Carolina, United States, 27710
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, Utah
Center for Clinical and Translational Sciences
Salt Lake City, Utah, United States, 84108
Utah Center for Advanced Imaging and Research (UCAIR)
Salt Lake City, Utah, United States, 84108
Investigational Drug Services
Salt Lake City, Utah, United States, 84112
Utah Program for Inherited Neuromuscular Disorder
Salt Lake City, Utah, United States, 84112
University of Utah Hospital
Salt Lake City, Utah, United States, 84132
University of Utah School of Medicine
Salt Lake City, Utah, United States, 84132
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Canada, British Columbia
UBC Children's and Women's Health Centre of British Columbia
Vancouver, British Columbia, Canada, V6H 3V4
Canada, Ontario
Children's Hospital- London Health Sciences Centre
London, Ontario, Canada, N6A 5W9
Canada, Quebec
Centre de Readaptation Marie Enfant (CRME)
Montreal, Quebec, Canada, H1T 1C9
CHU Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Italy
UOC Farmacia - Istituto Gianna Gaslini,
Genova, Italy, 16147
UOC Medicina Fisica Riabilitativa - Istituto G. Gaslini
Genova, Italy, 16147
UOC Neurologia Pediatrica e Malattie Muscolari Istituto Gianna Gaslini
Genova, Italy, 16147
UOC Radiologia - Istituto Gianna Gaslini,
Genova, Italy, 16147
UOS Dipartimentale Endocrinologia Clinica Sperimentale - Ist.
Genova, Italy, 16147
Dipartimento Pediatrico Universitario-Ospedaliero Endocrinologia
Rome, Italy, 00150
Farmacia Ospedaliera, IRCCS Ospedale Pediatrico Bambino Gesu
Rome, Italy, 00165
IRCCS Ospedale Pediatrico Bambino Gesu - Centro Trials
Rome, Italy, 00165
U.O. Malattie Neuromuscolari e Neurodegenerative, IRCCS Ospedale Pediatrico Bambino Gesu
Rome, Italy, 00165
Japan
Hyogo college of medicine hospital
Nishinomiya-shi, Hyogo, Japan, 6638501
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan, 187-8551
United Kingdom
Dubowitz Neuromuscular Centre, Institute of Child Health
London, United Kingdom, WC1N 3JH
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom, NE1 4LP
Institute of Genetic Medicine,Muscle Team
Newcastle-upon-Tyne, United Kingdom, NE1 3BZ
Clinical Research Facility
Newcastle-upon-Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT02907619     History of Changes
Other Study ID Numbers: B5161004
2016-001615-21 ( EudraCT Number )
First Posted: September 20, 2016    Key Record Dates
Last Update Posted: December 12, 2018
Last Verified: December 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Pfizer:
Duchenne muscular dystrophy, myostatin, open-label

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked