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

NS Pharma

Status and phase

Enrolling

Phase 2

Conditions

DMD

Exon 44

Duchenne Muscular Dystrophy

Treatments

Drug: NS-089/NCNP-02

Study type

Interventional

Funder types

Industry

Identifiers

NCT05996003

NS-089/NCNP-02-201

Details and patient eligibility

About

This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly.

The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

Enrollment

20 estimated patients

Sex

Male

Ages

4 to 14 years old

Volunteers

No Healthy Volunteers

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.