RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)

Quick Facts

Study Start:2024-02-22
Study Completion:2025-11-28
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05996003

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:4 Years to 14 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Male ≥ 4 years and \<15 years of age
  2. * Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame
  3. * Able to walk independently without assistive devices
  4. * Ability to complete the TTSTAND without assistance in \<20 seconds
  5. * 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.
  6. * Other inclusion criteria may apply.
  1. * Has a body weight of \<20 kg at the time of informed consent (applies to participants screening for Part 1 only)
  2. * Evidence of symptomatic cardiomyopathy
  3. * 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
  4. * 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
  5. * Surgery within the 3 months prior to the first dose of study drug or planned during the study duration
  6. * Previously treated in an interventional study of NS-089/NCNP-02
  7. * Having taken any gene therapy or other exon-skipping oligonucleotide
  8. * Other exclusion criteria may apply.

Contacts and Locations

Study Contact

Trial info
CONTACT
1-866-677-6276
trialinfo@nspharma.com

Study Locations (Sites)

Children's Hospital Colorado
Aurora, Colorado, 80045
United States
Rare Disease Research
Atlanta, Georgia, 30329
United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Shriners Hospital for Children
Portland, Oregon, 97239
United States
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, 15224
United States
UT Southwestern/Children's Health
Dallas, Texas, 75207
United States
Virginia Commonwealth University Health System
Richmond, Virginia, 23298
United States

Collaborators and Investigators

Sponsor: NS Pharma, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-02-22
Study Completion Date2025-11-28

Study Record Updates

Study Start Date2024-02-22
Study Completion Date2025-11-28

Terms related to this study

Additional Relevant MeSH Terms

  • Duchenne Muscular Dystrophy