Clinical Trial: Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Phase II Study of Nonsense Readthrough Compound NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy Patients (NORTH POL

Brief Summary: Duchenne Muscular Dystrophy (DMD) is inherited neuromuscular disorders due to mutation in the gene that encodes critical muscle protein called dystrophin. Currently, there is no effective treatment option for the disease. A pharmacological approach by promoting mRNA translation regardless of the presence of premature stop codons by nonsense mutation, called the readthrough strategy, has been developing recently for DMD with nonsense mutation. NPC-14 is a candidate compound for the readthrough strategy, since effective readthrough activities were demonstrated in nonclinical studies. This study is a phase II study designed to assess safety, tolerability, and efficacy of NPC-14 in ambulant DMD patients with nonsense mutation that were confirmed by whole genome analysis. These goals will be accomplished by monitoring adverse events by physical examination, cardiac, pulmonary, auditory, balance, and laboratory tests as safety endpoints, and dystrophin expression in muscle biopsy as primary efficacy endpoint, muscle function (NSAA, timed test, muscle strength (QMT, MMT) , dairy activities by lifecorder), and biomarkers as secondary efficacy endpoints. The study is a randomized, double blind, placebo-controlled study in 21 DMD patients. After screening, eligible patients are allocated dynamically to weekly NPC-14 or a placebo (saline) in a 2:1 ratio and will receive study drugs for 36 weeks.

Detailed Summary:
Sponsor: Kobe University

Current Primary Outcome:

  • Safety and tolerability (Adverse events) [ Time Frame: Up to 38 weeks (36 weeks treatment period and 2 weeks follow up period) ]
  • Change of dystrophin expression rate in muscle tissues from the baseline assessment [ Time Frame: At 37 weeks (1 week after from 36 weeks treatment period) ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • North Star Ambulatory Assessment [ Time Frame: At 36 weeks ]
  • Timed test (6MWT, time to walk/run 10 meters, time to climb/descent four steps, time to rise from the floor) [ Time Frame: At 36 weeks ]
  • Muscle strength (MMT, QMT) [ Time Frame: At 36 weeks ]
  • Dairy activities [ Time Frame: At 36 weeks ]
  • Biomarkers (CK, ALD) [ Time Frame: At 36 weeks ]


Original Secondary Outcome: Same as current

Information By: Kobe University

Dates:
Date Received: August 1, 2013
Date Started: August 2013
Date Completion: October 2015
Last Updated: September 2, 2015
Last Verified: September 2015