Phase 2 N=16 Treatment

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

Bottom Line

View on ClinicalTrials.gov: NCT03167255 ↗

Enrolled (actual)

Serious AEs

18.8%

Results posted

Nov 2022

Primary outcome: Primary: Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls — 0.17; -0.38; -0.11; 0.69 seconds

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: NS-065/NCNP-01 (Drug)
Age: Pediatric · 4+ yrs
Sex: Male
Sponsor: NS Pharma, Inc.
Primary completion: Oct 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls	0.17; -0.38; -0.11; 0.69; -0.60; 0.07	—
PRIMARY Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls	0.03; 0.04; 0.04; 0.03; 0.04; 0.04	—
PRIMARY Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.	8; 8; 16; 0; 1; 1	—
SECONDARY Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls	-0.41; -1.35; -0.88; -0.87; -0.74; -0.82	—
SECONDARY Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls	0.15; 0.53; 0.34; 0.35; 0.28; 0.32	—
SECONDARY Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls	-0.37; 0.38; 0.01; -0.26; 0.28; 0.02	—
SECONDARY Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls	0.04; 0.01; 0.02; 0.05; 0.01; 0.03	—
SECONDARY Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls	2.53; -0.02; 1.27; 1.91; 0.14; 1.05	—
SECONDARY Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls	15.15; 13.03; 14.30; 9.90; 16.94; 13.32	—
SECONDARY Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls	0.56; 1.86; 1.09; 1.63; 2.34; 1.96	—
SECONDARY Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls	-0.11; 0.57; 0.21; 0.57; 0.63; 0.60	—
SECONDARY Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls	0.55; 0.88; 0.70; 0.48; 0.93; 0.68	—
SECONDARY Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls	-0.35; 2.04; 0.82; 0.77; 1.20; 1.03	—
SECONDARY Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls	1.25; 1.10; 1.15; 0.04; 1.11; 0.52	—

Summary

This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

Eligibility Criteria

Inclusion Criteria

Completed Study NS-065/NCNP-01-201 through Week 25.
Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures.
Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.

Exclusion Criteria

Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-01.
Patient had a treatment which was made for the purpose of dystrophin or its related protein induction after completion of Study NS-065/NCNP-01-201.
Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201.
Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03167255). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.