ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 94 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02907619
Recruitment Status : Recruiting
First Posted : September 20, 2016
Last Update Posted : May 16, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

September 14, 2016
September 20, 2016
May 16, 2018
October 13, 2016
March 13, 2023   (Final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT02907619 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
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.
Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
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
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
Intervention: Biological: PF-06252616
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
105
Same as current
March 13, 2023
March 13, 2023   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
6 Years to 18 Years   (Child, Adult)
No
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com
Canada,   Italy,   Japan,   United Kingdom,   United States
 
 
NCT02907619
B5161004
2016-001615-21 ( EudraCT Number )
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
May 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP