NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Launched by NS PHARMA, INC. · Aug 10, 2023

Keywords

ClinConnect Summary

This clinical trial, called NS-089/NCNP-02-201, is investigating a new treatment for boys aged 4 to 15 years who have Duchenne Muscular Dystrophy (DMD), a genetic condition that affects muscle strength. The treatment involves a drug known as NS-089/NCNP-02, which is given through an intravenous (IV) infusion once a week. The study has two parts: the first part will include six boys, and in the second part, an additional 14 boys will join to help researchers learn more about how well the treatment works for DMD caused by specific gene mutations.

To be eligible for this trial, boys must be able to walk independently and have a confirmed mutation in the dystrophin gene that allows for a specific type of treatment called "exon skipping." They also need to have been on a stable dose of glucocorticoids (a type of medication) for at least three months. Participants can expect to visit the study site regularly for their weekly infusions and will be monitored closely throughout the study. This trial is currently recruiting participants, and it’s a great opportunity for boys with DMD to potentially access a new treatment option while contributing to important research.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Male ≥ 4 years and \<15 years of age
• • Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame
• • Able to walk independently without assistive devices
• • Ability to complete the TTSTAND without assistance in \<20 seconds
• • Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.
• • Other inclusion criteria may apply.
• Exclusion Criteria:
• • Has a body weight of \<20 kg at the time of informed consent (applies to participants screening for Part 1 only)
• • Evidence of symptomatic cardiomyopathy
• • Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug
• • Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer
• • Surgery within the 3 months prior to the first dose of study drug or planned during the study duration
• • Previously treated in an interventional study of NS-089/NCNP-02
• • Having taken any gene therapy.
• • Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP or is expected to receive exon skipping oligonucleotide prior to completion of study.
• • Other exclusion criteria may apply.