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

Part of paid clinical trials in Aurora, Colorado.

Sponsor
NS Pharma, Inc.
Study ID
NCT05996003
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
MALE
Age
4 Years - 14 Years
Healthy Volunteers
Not accepted

Interventions

  • NS-089/NCNP-02 — DRUG
    Cohort 1: Part 1 Dose Level 1-3: a 4-week Treatment Phase at each treatment dose level Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1 Cohort 2: Part 2 Single Dose Level: a 24-week Treatment Phase at the MTD of Part 1

Study Details

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.

Key Dates

Start date
Feb 22, 2024
Status verified
Mar 2026
Primary completion
Sep 11, 2026
Completion
Sep 11, 2026

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: NS-089/NCNP-02
    Experimental: NS-089/NCNP-02 NS-089/NCNP-02 solution for infusion (Cohort 1) NS-089/NCNP-02 solution for infusion (Cohort 2)

Primary Outcome Measure

Adverse Event and Adverse Drug Reaction [ Time Frame: through study completion, up to follow-up phone call for Part 2 ]

Central Contacts

Locations (10)

FacilityCityStateZIPSite coordinators
Children's Hospital ColoradoAuroraColorado80045
Hannah Kleiner
[email protected]
Rare Disease Research, LLC - FLKissimmeeFlorida34746
Marcial Almaraz
[email protected]
Rare Disease ResearchAtlantaGeorgia30329
Ann and Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Alka Maheshwari
[email protected]
University of Kansas Medical Center (KUMC)Kansas CityKansas66160
Rebecca Clay
[email protected]
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229
Angela Edmondson
[email protected]
The Children's Hospital of Philadelphia (CHOP)PhiladelphiaPennsylvania19104
Karen Monono
[email protected]
University of Pittsburgh School of MedicinePittsburghPennsylvania15224
Vibha Chauhan
[email protected]
UT Southwestern/Children's HealthDallasTexas75207
Deniece Winston
[email protected]
Virginia Commonwealth University Health SystemRichmondVirginia23298
Andrea Jewell
[email protected]

Find similar trials in Aurora, CO

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Children's Hospital Colorado· Aurora, CORare Disease Research, LLC - FL· Kissimmee, FLRare Disease Research· Atlanta, GAAnn and Robert H. Lurie Children's Hospital of Chicago· Chicago, ILUniversity of Kansas Medical Center (KUMC)· Kansas City, KSCincinnati Children's Hospital Medical Center· Cincinnati, OH

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