This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback
Trial record 1 of 1 for:    NCT02907619
Previous Study | Return to List | Next Study

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

This study is currently recruiting participants.
See Contacts and Locations
Verified June 2017 by Pfizer
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT02907619
First received: September 14, 2016
Last updated: June 6, 2017
Last verified: June 2017
  Purpose
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 Intervention Phase
Duchenne Muscular Dystrophy Biological: PF-06252616 Phase 2

Study Type: Interventional
Study Design: Masking: No masking
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

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 105
Actual Study Start Date: October 13, 2016
Estimated Study Completion Date: February 3, 2023
Estimated Primary Completion Date: February 3, 2023 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02907619

Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021

Locations
United States, Maryland
Kennedy Krieger Institute Out-patient Center Recruiting
Baltimore, Maryland, United States, 21205
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21287
Johns Hopkins Investigational Drug Service Recruiting
Baltimore, Maryland, United States, 21287
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Canada, Ontario
Children's Hospital- London Health Sciences Centre Recruiting
London, Ontario, Canada, N6A 5W9
Japan
National Center of Neurology and Psychiatry Recruiting
Kodaira, Tokyo, Japan, 187-8551
United Kingdom
Royal Victoria Infirmary Recruiting
Newcastle upon Tyne, United Kingdom, NE1 4LP
Institute of Genetic Medicine,Muscle Team Recruiting
Newcastle-upon-Tyne, United Kingdom, NE1 3BZ
Clinical Research Facility Recruiting
Newcastle-upon-Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT02907619     History of Changes
Other Study ID Numbers: B5161004
2016-001615-21 ( EudraCT Number )
Study First Received: September 14, 2016
Last Updated: June 6, 2017

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

ClinicalTrials.gov processed this record on June 22, 2017