A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
Public ClinicalTrials.gov record NCT02310763. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular Dystrophy. Three intravenous (IV) dose levels will be investigated in a within subject dose escalating fashion. Subjects will be randomly assigned to 1 of 3 sequence groups for approximately 96 weeks (2 periods of 48 weeks each). In period 1, two of the sequence groups will receive PF-06252616 and one sequence group will receive placebo. In period 2, the placebo group will switch to PF-06252616 and the two remaining sequence groups will either receive placebo or PF-06252616. Efficacy will be based on an observed mean change from baseline on function (4 stair climb) of PF-06252616 as compared to the placebo at the end of period 1. Period 2 provides an opportunity to evaluate PK. Subjects will receive monthly IV infused doses of either PF-06252616 or placebo and will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional evaluations (pulmonary function testing, 4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing and the six minute walk test), pharmacokinetic testing and pharmacodynamic testing to evaluate changes in muscle volume (MRI).
Study identification
- NCT ID
- NCT02310763
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 121 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Years to 15 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 23, 2014
- Primary completion
- Apr 29, 2018
- Completion
- Nov 22, 2018
- Last update posted
- Dec 6, 2020
2014 – 2018
United States locations
- U.S. sites
- 30
- U.S. states
- 14
- U.S. cities
- 17
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Ronald Reagan UCLA Medical Center | Los Angeles | California | 90095 | — |
| Ronald Reagan UCLA Pharmacy | Los Angeles | California | 90095 | — |
| UCLA (David Geffen School of Medicine) | Los Angeles | California | 90095 | — |
| University of California, Davis Medical Center | Sacramento | California | 95817 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Shriners Hospitals for Children - Tampa | Tampa | Florida | 33612 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| University of Iowa ICTS | Iowa City | Iowa | 52242 | — |
| KU Clinical Research Center, Clinical and Translational Science Unit(CTSU) | Fairway | Kansas | 66205 | — |
| University of Kansas-Clinical Research Center, Investigational Pharmacy | Fairway | Kansas | 66205 | — |
| University of Kansas Medical Center, Landon Center on Aging | Kansas City | Kansas | 66160 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Kennedy Krieger Institute Out-patient center | Baltimore | Maryland | 21205 | — |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | — |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287 | — |
| Johns Hopkins Investigational Drug Service | Baltimore | Maryland | 21287 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | 55455 | — |
| St Louis Children's Hospital | St Louis | Missouri | 63110 | — |
| Duke University Medical Center,Lenox Baker Children's Hospital | Durham | North Carolina | 27705 | — |
| Duke University, Investigational Drug Pharmacy | Durham | North Carolina | 27710 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | — |
| Center for Clinical and Translational Sciences | Salt Lake City | Utah | 84108 | — |
| Utah Center for Advanced Imaging and Research (UCAIR) | Salt Lake City | Utah | 84108 | — |
| Investigational Drug Services | Salt Lake City | Utah | 84112 | — |
| University of Utah, Department of Neurology | Salt Lake City | Utah | 84112 | — |
| University of Utah Medical Center | Salt Lake City | Utah | 84132 | — |
| University of Utah School of Medicine | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 36 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02310763, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 6, 2020 · Synced Sep 12, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02310763 live on ClinicalTrials.gov.