NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Recruiting
Phase 2
Interventional Study
Duchenne Muscular Dystrophy
Exon 44
DMD
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 4 – 14
- Sex
- Male
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 20 (estimated)
- Sponsor
- NS Pharma, Inc. · Industry
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About This Trial
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…
Trial Locations
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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 assista…
Contacts