Status:
RECRUITING
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Lead Sponsor:
NS Pharma, Inc.
Collaborating Sponsors:
Nippon Shinyaku Co., Ltd.
Conditions:
Duchenne Muscular Dystrophy
Exon 44
Eligibility:
MALE
4-14 years
Phase:
PHASE2
Brief Summary
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 skip...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
February 22 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 11 2026
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT05996003
Start Date
February 22 2024
End Date
September 11 2026
Last Update
December 3 2025
Active Locations (22)
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1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
2
Rare Disease Research, LLC - FL
Kissimmee, Florida, United States, 34746
3
Rare Disease Research
Atlanta, Georgia, United States, 30329
4
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611