An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
NCT02907619
ABOUT THIS STUDY
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
Pfizer Clinical Trials Contact Center
1-800-718-1021
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.
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.
NEED INFO?
Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative

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Descriptive Information | |||||||
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Brief Title ICMJE | An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy | ||||||
Official Title ICMJE | A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHY | ||||||
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. | ||||||
Detailed Description | Not Provided | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 2 | ||||||
Study Design ICMJE | Allocation: N/A Masking: None (Open Label) Primary Purpose: Treatment | ||||||
Condition ICMJE | Duchenne Muscular Dystrophy | ||||||
Intervention ICMJE | 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 | ||||||
Study Arms ICMJE | 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 | ||||||
Publications * | Wagner KR, Abdel-Hamid HZ, Mah JK, Campbell C, Guglieri M, Muntoni F, Takeshima Y, McDonald CM, Kostera-Pruszczyk A, Karachunski P, Butterfield RJ, Mercuri E, Fiorillo C, Bertini ES, Tian C, Statland J, Sadosky AB, Purohit VS, Sherlock SP, Palmer JP, Binks M, Charnas L, Marraffino S, Wong BL. Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy. Neuromuscul Disord. 2020 Jun;30(6):492-502. doi: 10.1016/j.nmd.2020.05.002. Epub 2020 May 19. Erratum in: Neuromuscul Disord. 2021 Feb;31(2):167-168. | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||||
Recruitment Information | |||||||
Recruitment Status ICMJE | Terminated | ||||||
Actual Enrollment ICMJE | 59 | ||||||
Original Estimated Enrollment ICMJE | 105 | ||||||
Actual Study Completion Date ICMJE | November 22, 2018 | ||||||
Actual Primary Completion Date | November 22, 2018 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Years to 18 Years (Child, Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | Canada, Italy, Japan, United Kingdom, United States | ||||||
Removed Location Countries | |||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT02907619 | ||||||
Other Study ID Numbers ICMJE | B5161004 2016-001615-21 ( EudraCT Number ) | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Pfizer | ||||||
Study Sponsor ICMJE | Pfizer | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
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PRS Account | Pfizer | ||||||
Verification Date | October 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |