Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Completed
• Trial end date: 2018-04-01
• Target enrollment: 0
• Participant gender: Male

Summary

The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NS Pharma, Inc.
• Collaborators: Cooperative International Neuromuscular Research Group; Nippon Shinyaku Co Ltd; Nippon Shinyaku Co., Ltd.; Therapeutic Research in Neuromuscular Disorders Solutions

Criteria

Inclusion Criteria:

• Male ≥ 4 years and <10 years of age

• Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame;

• Able to walk independently without assistive devices;

• Ability to complete the time to stand, time to run/walk and time to climb assessments;

• Stable dose of glucocorticoid for at least 3 months

Exclusion Criteria:

• Acute illness within 4 weeks prior to the first dose of study medication;

• Evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];

• Severe allergy or hypersensitivity to medications;

• Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;

• Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;

• Patient is taking any other investigational drug currently or within 3 months prior to the start of study treatment; or

• Patient has had surgery within the 3 months prior to the first anticipated administration of study medication or surgery is planned for anytime during the duration of the study;

• Patient has previously participated in this study or any other study during which NS-065/NCNP-01 was administered.