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A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

NCT02310763 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 121

Last updated 2020-12-07

Study results available
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Summary

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).

Conditions

Interventions

BIOLOGICAL

PF-06252616

PF-06252616 IV Infusion, 3 dose levels (5mg/kg, 20 mg/kg and 40 mg/kg) will be investigated within each subject

DRUG

Placebo

Sponsors & Collaborators

Principal Investigators

  • Pfizer CT.gov Call Center · Pfizer

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE

Eligibility

Min Age
6 Years
Max Age
15 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-11-24
Primary Completion
2018-04-30
Completion
2018-11-23
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Bulgaria
  • Canada
  • Italy
  • Japan
  • Poland
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02310763 on ClinicalTrials.gov