Recruiting
Phase 2

NS-089/NCNP-02-201

Sponsor:

NS Pharma, Inc.

Code:

NCT05996003

Conditions

Duchenne Muscular Dystrophy

Exon 44

DMD

Eligibility Criteria

Sex: Male

Age: 4 - 14

Healthy Volunteers: Not accepted

Interventions

NS-089/NCNP-02

Study Details

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 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.

Conditions

Duchenne Muscular Dystrophy

Exon 44

DMD

Study ID

NCT05996003

Start date

Feb 22, 2024

Status verified date

Mar, 2026

Completion date

Sep 11, 2026

Anticipated

Primary completion date

Sep 11, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 4 - 14

Healthy Volunteers: Not accepted

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 received exon skipping oligonucleotide within 1 year prior to the first dose of IP
  • Other exclusion criteria may apply.

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

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)

Interventions

NS-089/NCNP-02

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

Primary outcome measure

  • Adverse Event and Adverse Drug Reaction [ Time Frame: through study completion, up to follow-up phone call for Part 2 ]
  • Plasma pharmacokinetic (PK) parameters [ Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Maximum plasma concentration (Cmax) of NS-089/NCNP-02 ]
  • Plasma pharmacokinetic (PK) parameters [ Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Time of the maximum plasma concentration (Tmax) of NS-089/NCNP-02 ]
  • Plasma pharmacokinetic (PK) parameters [ Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Terminal half-life (T1/2) of NS-089/NCNP-02 ]
  • Plasma pharmacokinetic (PK) parameters [ Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to the last time point (AUC0-t) of NS-089/NCNP-02 ]
  • Plasma pharmacokinetic (PK) parameters [ Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Area under the concentration-time curve from time 0 to infinity (AUC0-∞) of NS-089/NCNP-02 ]
  • Plasma pharmacokinetic (PK) parameters [ Time Frame: [Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Total body clearance (CLtot) of NS-089/NCNP-02 ]
  • Plasma pharmacokinetic (PK) parameters [ Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] The volume in the terminal state (Vz) of NS-089/NCNP-02 ]
  • Urine pharmacokinetic parameters [ Time Frame: Day1, Week4 for each dose for Part 1, Day1 and Week24 for Part 2] Urinary excretion of NS-089/NCNP-02 ]
  • Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot). [ Time Frame: Baseline, Week25 ]

Central Contacts and Locations

Central contacts

Trial info

1-866-677-6276trialinfo@nspharma.com

Locations

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Hannah Kleiner

hannah.kleiner@childrenscolorado.org

Rare Disease Research, LLC - FL

Recruiting

Kissimmee, Florida, United States, 34746

Contacts

Marcial Almaraz

marcial.almaraz@rarediseaseresearch.com

Rare Disease Research

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

Julia Zhu

julia.zhu@rarediseaseresearch.com

Ann and Robert H. Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Raven Hill

rhill@luriechildrens.org

Alka Maheshwari

amaheshwari@luriechildrens.org

University of Kansas Medical Center (KUMC)

Recruiting

Kansas City, Kansas, United States, 66160

Contacts

Rebecca Clay

rclay@kumc.edu

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Angela Edmondson

Angela.Edmondson@cchmc.org

The Children's Hospital of Philadelphia (CHOP)

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Karen Monono

mononok@email.chop.edu

University of Pittsburgh School of Medicine

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contacts

Vibha Chauhan

vibha.chauhan@chp.edu

UT Southwestern/Children's Health

Recruiting

Dallas, Texas, United States, 75207

Contacts

Deniece Winston

Deniece.Winston@UTSouthwestern.edu

Virginia Commonwealth University Health System

Recruiting

Richmond, Virginia, United States, 23298

Contacts

Andrea Jewell

andrea.jewell@vcuhealth.org

London Health Sciences Centre

Recruiting

London, Ontario, Canada

Contacts

Rhiannon Hicks

rhiannon.hicks@lhsc.on.ca

More Information

Sponsor

NS Pharma, Inc.

Last update posted

Mar 6, 2026

Last verified

Mar, 2026

Keywords

  • Exon 44 Skipping
  • DMD
  • Brogidirsen

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by NS Pharma, Inc. on 2026-03-06.