Clinical Trials Logo
  1. Home
  2. Duchenne Muscular Dystrophy
  3. NCT02310763

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

Duchenne Muscular Dystrophy Clinical Trial

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

Clinical Trial 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).

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02310763
Study type Interventional
Source Pfizer
Contact
Status Terminated
Phase Phase 2
Start date November 24, 2014
Completion date November 23, 2018
View Details
See also
  Status Clinical Trial Phase
Completed NCT05575648 - Dual Task in Duchenne Muscular Dystrophy N/A
Terminated NCT03907072 - Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy Phase 2/Phase 3
Completed NCT04335942 - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor N/A
Active, not recruiting NCT04906460 - Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) Phase 1/Phase 2
Active, not recruiting NCT02500381 - Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) Phase 3
Enrolling by invitation NCT05967351 - A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study Phase 3
Recruiting NCT03067831 - Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy Phase 1/Phase 2
Recruiting NCT01834040 - Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy Phase 1/Phase 2
Completed NCT02246478 - A Study of TAS-205 for Duchenne Muscular Dystrophy Phase 1
Active, not recruiting NCT01772043 - Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping N/A
Terminated NCT01168908 - Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Phase 2
Completed NCT00758225 - Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) Phase 2
Completed NCT03680365 - Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
Recruiting NCT03513367 - The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
Recruiting NCT05712447 - Duchenne Muscular Dystrophy Video Assessment Registry
Recruiting NCT01484678 - Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Completed NCT03319030 - Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
Terminated NCT01753804 - A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. N/A
Completed NCT02530905 - Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients Phase 1
Terminated NCT04708314 - An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy Phase 4