Who is sponsoring NORTH POLE DMD?

NORTH POLE DMD is sponsored by Kobe University.

What condition does NORTH POLE DMD study?

NORTH POLE DMD studies Muscular Dystrophy, Duchenne.

What drugs are being tested in NORTH POLE DMD?

Interventions: NPC-14, Placebo.

What is the status of NORTH POLE DMD?

NORTH POLE DMD is currently UNKNOWN.

Last reviewed 2015-09-02 · How we verify

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

NCT01918384 Phase 2 UNKNOWN

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.

Details

Lead sponsor	Kobe University
Phase	Phase 2
Status	UNKNOWN
Enrolment	21
Start date	2013-08
Completion	2015-10

Conditions

Muscular Dystrophy, Duchenne

Interventions

NPC-14
Placebo

Primary outcomes

Safety and tolerability (Adverse events) — 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 — At 37 weeks (1 week after from 36 weeks treatment period)

Countries

Japan