NCT03167255 is a Phase 2 clinical trial of NS-065/NCNP-01 in Duchenne Muscular Dystrophy, sponsored by NS Pharma, Inc..

Who is sponsoring NCT03167255?

NCT03167255 is sponsored by NS Pharma, Inc..

What drugs are being tested in NCT03167255?

Interventions in NCT03167255: NS-065/NCNP-01.

What condition does NCT03167255 study?

NCT03167255 studies Duchenne Muscular Dystrophy.

Is NCT03167255 still recruiting?

NCT03167255 is currently completed. Last updated 28 December 2022.

Are results published for NCT03167255?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2022-12-28 · How we verify

NCT03167255

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

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

Completed Phase 2 Results posted Last updated 28 December 2022

What this trial tests

Phase 2 trial testing NS-065/NCNP-01 in Duchenne Muscular Dystrophy in 16 participants. Completed in 15 November 2021.

Timeline

6 July 2017

Primary endpoint
20 October 2021

15 November 2021

Quick facts

Lead sponsor	NS Pharma, Inc.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	6 July 2017
Primary completion	20 October 2021
Estimated completion	15 November 2021
Sites	6 locations across Canada, United States

Drugs / interventions tested

NS-065/NCNP-01 — full drug profile →

Conditions studied

Duchenne Muscular Dystrophy — all drugs for Duchenne Muscular Dystrophy →

Sponsor

NS Pharma, Inc. — full company profile →

Who can join

Adults 4 to 10, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls Primary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND)

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.17	± 1.60
NS-065/NCNP-01 80mg/kg	-0.38	± 1.59
Total NS-065/NCNP-01 Group	-0.11	± 1.12

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.69	± 1.60
NS-065/NCNP-01 80mg/kg	-0.60	± 1.61
Total NS-065/NCNP-01 Group	0.07	± 1.13

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.55	± 1.63
NS-065/NCNP-01 80mg/kg	0.03	± 1.62
Total NS-065/NCNP-01 Group	0.29	± 1.14

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.77	± 1.68
NS-065/NCNP-01 80mg/kg	0.30	± 1.67
Total NS-065/NCNP-01 Group	0.54	± 1.18

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.32	± 1.80
NS-065/NCNP-01 80mg/kg	1.45	± 1.72
Total NS-065/NCNP-01 Group	1.44	± 1.23

Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.62	± 1.82
NS-065/NCNP-01 80mg/kg	3.58	± 1.70
Total NS-065/NCNP-01 Group	2.71	± 1.24

Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls Primary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.03	± 0.03
NS-065/NCNP-01 80mg/kg	0.04	± 0.03
Total NS-065/NCNP-01 Group	0.04	± 0.02

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.03	± 0.03
NS-065/NCNP-01 80mg/kg	0.04	± 0.03
Total NS-065/NCNP-01 Group	0.04	± 0.02

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.03	± 0.03
NS-065/NCNP-01 80mg/kg	0.50	± 0.03
Total NS-065/NCNP-01 Group	0.04	± 0.02

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.04	± 0.03
NS-065/NCNP-01 80mg/kg	0.04	± 0.03
Total NS-065/NCNP-01 Group	0.04	± 0.02

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.02	± 0.04
NS-065/NCNP-01 80mg/kg	-0.05	± 0.03
Total NS-065/NCNP-01 Group	-0.01	± 0.02

Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.02	± 0.03
NS-065/NCNP-01 80mg/kg	-0.07	± 0.03
Total NS-065/NCNP-01 Group	-0.04	± 0.02

Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. Primary · Up to 192 weeks of treatment

For adverse events (AEs) starting in study 201 (NCT02740972) which are not resolved at the time of enrollment into this study 202, any change in outcome or relatedness were reported in study 201. For AEs starting in study 201 which increase in severity or becomes serious after enrollment in this study 202, a new AE was reported in this study. Treatment-emergent AEs (TEAEs) were summarized by dose level. Coding was done by system organ class and preferred term (using the Medical Dictionary for Regulatory Activities (MedDRA)). Level of severity was assessed using the CTCAE grading system.

Participants with TEAE

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	8
NS-065/NCNP-01 80mg/kg	8
Total NS-065/NCNP-01 Group	16

Participants with drug-related TEAE

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0
NS-065/NCNP-01 80mg/kg	1
Total NS-065/NCNP-01 Group	1

Participants with CTCAE ≥ Grade 3

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0
NS-065/NCNP-01 80mg/kg	2
Total NS-065/NCNP-01 Group	2

Participants with TEAEs leading to discontinuation

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0
NS-065/NCNP-01 80mg/kg	0
Total NS-065/NCNP-01 Group	0

Participants with serious TEAE

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1
NS-065/NCNP-01 80mg/kg	2
Total NS-065/NCNP-01 Group	3

Death

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0
NS-065/NCNP-01 80mg/kg	0
Total NS-065/NCNP-01 Group	0

Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW)

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.41	± 1.06
NS-065/NCNP-01 80mg/kg	-1.35	± 1.05
Total NS-065/NCNP-01 Group	-0.88	± 0.75

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.87	± 1.06
NS-065/NCNP-01 80mg/kg	-0.74	± 1.06
Total NS-065/NCNP-01 Group	-0.82	± 0.75

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-1.15	± 1.06
NS-065/NCNP-01 80mg/kg	-0.68	± 1.05
Total NS-065/NCNP-01 Group	-0.91	± 0.75

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.67	± 1.06
NS-065/NCNP-01 80mg/kg	-0.25	± 1.05
Total NS-065/NCNP-01 Group	-0.46	± 0.75

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.13	± 1.07
NS-065/NCNP-01 80mg/kg	0.56	± 1.06
Total NS-065/NCNP-01 Group	0.35	± 0.75

Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.89	± 1.10
NS-065/NCNP-01 80mg/kg	2.84	± 1.05
Total NS-065/NCNP-01 Group	2.00	± 0.76

Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW) Velocity. The results were converted into velocity (meter/time).

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.15	± 0.17
NS-065/NCNP-01 80mg/kg	0.53	± 0.17
Total NS-065/NCNP-01 Group	0.34	± 0.12

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.35	± 0.17
NS-065/NCNP-01 80mg/kg	0.28	± 0.17
Total NS-065/NCNP-01 Group	0.32	± 0.12

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.44	± 0.17
NS-065/NCNP-01 80mg/kg	0.28	± 0.17
Total NS-065/NCNP-01 Group	0.36	± 0.12

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.33	± 0.17
NS-065/NCNP-01 80mg/kg	0.12	± 0.17
Total NS-065/NCNP-01 Group	0.23	± 0.12

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.24	± 0.18
NS-065/NCNP-01 80mg/kg	-0.02	± 0.17
Total NS-065/NCNP-01 Group	0.11	± 0.12

Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.14	± 0.18
NS-065/NCNP-01 80mg/kg	-0.23	± 0.17
Total NS-065/NCNP-01 Group	-0.06	± 0.13

Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB)

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.37	± 1.54
NS-065/NCNP-01 80mg/kg	0.38	± 1.54
Total NS-065/NCNP-01 Group	0.01	± 1.09

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.26	± 1.54
NS-065/NCNP-01 80mg/kg	0.28	± 1.56
Total NS-065/NCNP-01 Group	0.02	± 1.09

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.08	± 1.54
NS-065/NCNP-01 80mg/kg	0.44	± 1.54
Total NS-065/NCNP-01 Group	0.18	± 1.09

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.02	± 1.54
NS-065/NCNP-01 80mg/kg	1.08	± 1.54
Total NS-065/NCNP-01 Group	1.05	± 1.09

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	4.28	± 1.55
NS-065/NCNP-01 80mg/kg	2.95	± 1.55
Total NS-065/NCNP-01 Group	3.61	± 1.09

Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	3.00	± 1.66
NS-065/NCNP-01 80mg/kg	3.18	± 1.58
Total NS-065/NCNP-01 Group	3.16	± 1.14

Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB) Velocity. The results were converted into velocity (meter/time).

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.04	± 0.04
NS-065/NCNP-01 80mg/kg	0.01	± 0.04
Total NS-065/NCNP-01 Group	0.02	± 0.03

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.05	± 0.04
NS-065/NCNP-01 80mg/kg	0.01	± 0.04
Total NS-065/NCNP-01 Group	0.03	± 0.03

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.06	± 0.04
NS-065/NCNP-01 80mg/kg	0.03	± 0.04
Total NS-065/NCNP-01 Group	0.05	± 0.03

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.03	± 0.04
NS-065/NCNP-01 80mg/kg	-0.02	± 0.04
Total NS-065/NCNP-01 Group	0.00	± 0.03

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.04	± 0.04
NS-065/NCNP-01 80mg/kg	-0.07	± 0.04
Total NS-065/NCNP-01 Group	-0.01	± 0.03

Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.05	± 0.05
NS-065/NCNP-01 80mg/kg	-0.07	± 0.04
Total NS-065/NCNP-01 Group	-0.01	± 0.03

Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): North Star Ambulatory Assessment (NSAA) score The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	2.53	± 1.83
NS-065/NCNP-01 80mg/kg	-0.02	± 1.83
Total NS-065/NCNP-01 Group	1.27	± 1.29

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.91	± 1.83
NS-065/NCNP-01 80mg/kg	0.14	± 1.85
Total NS-065/NCNP-01 Group	1.05	± 1.29

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.41	± 1.83
NS-065/NCNP-01 80mg/kg	1.23	± 1.83
Total NS-065/NCNP-01 Group	0.83	± 1.29

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.91	± 1.83
NS-065/NCNP-01 80mg/kg	1.36	± 1.83
Total NS-065/NCNP-01 Group	1.14	± 1.29

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-1.31	± 1.85
NS-065/NCNP-01 80mg/kg	0.35	± 1.85
Total NS-065/NCNP-01 Group	-0.47	± 1.30

Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Six-Minute Walk Test (6MWT)

Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	15.15	± 26.84
NS-065/NCNP-01 80mg/kg	13.03	± 28.15
Total NS-065/NCNP-01 Group	14.30	± 19.29

Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	9.90	± 26.84
NS-065/NCNP-01 80mg/kg	16.94	± 29.70
Total NS-065/NCNP-01 Group	13.32	± 19.75

Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.73	± 26.84
NS-065/NCNP-01 80mg/kg	18.14	± 26.89
Total NS-065/NCNP-01 Group	8.72	± 18.87

Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-16.35	± 26.84
NS-065/NCNP-01 80mg/kg	18.51	± 26.89
Total NS-065/NCNP-01 Group	1.10	± 18.87

Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-41.61	± 28.08
NS-065/NCNP-01 80mg/kg	-27.76	± 29.71
Total NS-065/NCNP-01 Group	-34.86	± 20.28

Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Handgrip For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored

Handgrip, Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.56	± 1.28
NS-065/NCNP-01 80mg/kg	1.86	± 1.42
Total NS-065/NCNP-01 Group	1.09	± 0.94

Handgrip, Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.63	± 1.28
NS-065/NCNP-01 80mg/kg	2.34	± 1.37
Total NS-065/NCNP-01 Group	1.96	± 0.93

Handgrip, Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.85	± 1.32
NS-065/NCNP-01 80mg/kg	0.83	± 1.42
Total NS-065/NCNP-01 Group	1.39	± 0.96

Handgrip, Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	2.22	± 1.28
NS-065/NCNP-01 80mg/kg	2.00	± 1.42
Total NS-065/NCNP-01 Group	2.12	± 0.94

Handgrip, Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	2.94	± 1.55
NS-065/NCNP-01 80mg/kg	3.10	± 1.59
Total NS-065/NCNP-01 Group	3.06	± 1.10

Handgrip, Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	7.34	± 2.61
NS-065/NCNP-01 80mg/kg	4.05	± 1.74
Total NS-065/NCNP-01 Group	5.00	± 1.39

Dominant Side Handgrip, Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.22	± 1.28
NS-065/NCNP-01 80mg/kg	2.17	± 1.42
Total NS-065/NCNP-01 Group	1.03	± 0.95

Dominant Side Handgrip, Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.17	± 1.28
NS-065/NCNP-01 80mg/kg	2.70	± 1.37
Total NS-065/NCNP-01 Group	1.88	± 0.93

Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Flexors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a s

Elbow Flexors, Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.11	± 0.72
NS-065/NCNP-01 80mg/kg	0.57	± 0.78
Total NS-065/NCNP-01 Group	0.21	± 0.53

Elbow Flexors, Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.57	± 0.72
NS-065/NCNP-01 80mg/kg	0.63	± 0.78
Total NS-065/NCNP-01 Group	0.60	± 0.53

Elbow Flexors, Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.70	± 0.75
NS-065/NCNP-01 80mg/kg	0.82	± 0.81
Total NS-065/NCNP-01 Group	0.00	± 0.55

Elbow Flexors, Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.53	± 0.72
NS-065/NCNP-01 80mg/kg	0.30	± 0.78
Total NS-065/NCNP-01 Group	0.42	± 0.53

Elbow Flexors, Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.24	± 0.89
NS-065/NCNP-01 80mg/kg	1.05	± 0.85
Total NS-065/NCNP-01 Group	0.49	± 0.61

Elbow Flexors, Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.74	± 0.98
NS-065/NCNP-01 80mg/kg	0.07	± 0.91
Total NS-065/NCNP-01 Group	0.34	± 0.66

Dominant Side Elbow Flexors, Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.01	± 0.77
NS-065/NCNP-01 80mg/kg	0.39	± 0.83
Total NS-065/NCNP-01 Group	0.18	± 0.56

Dominant Side Elbow Flexors, Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.48	± 0.77
NS-065/NCNP-01 80mg/kg	-0.38	± 0.83
Total NS-065/NCNP-01 Group	0.08	± 0.56

Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls Secondary · Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Extensors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a

Elbow Extensors, Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.55	± 0.73
NS-065/NCNP-01 80mg/kg	0.88	± 0.78
Total NS-065/NCNP-01 Group	0.70	± 0.53

Elbow Extensors, Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.48	± 0.73
NS-065/NCNP-01 80mg/kg	0.93	± 0.78
Total NS-065/NCNP-01 Group	0.68	± 0.53

Elbow Extensors, Week 73

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.27	± 0.76
NS-065/NCNP-01 80mg/kg	0.96	± 0.81
Total NS-065/NCNP-01 Group	0.60	± 0.55

Elbow Extensors, Week 109

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.23	± 0.73
NS-065/NCNP-01 80mg/kg	-0.05	± 0.78
Total NS-065/NCNP-01 Group	0.63	± 0.53

Elbow Extensors, Week 157

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	1.56	± 0.91
NS-065/NCNP-01 80mg/kg	-0.01	± 0.86
Total NS-065/NCNP-01 Group	0.70	± 0.62

Elbow Extensors, Week 205

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	-0.06	± 1.01
NS-065/NCNP-01 80mg/kg	0.07	± 0.92
Total NS-065/NCNP-01 Group	0.06	± 0.67

Dominant Side Elbow Extensors, Week 37

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.68	± 0.76
NS-065/NCNP-01 80mg/kg	1.21	± 0.82
Total NS-065/NCNP-01 Group	0.92	± 0.55

Dominant Side Elbow Extensors, Week 49

Group	Value	95% CI
NS-065/NCNP-01 40mg/kg	0.54	± 0.76
NS-065/NCNP-01 80mg/kg	0.53	± 0.82
Total NS-065/NCNP-01 Group	0.53	± 0.55

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 192 weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

NS-065/NCNP-01 40mg/kg

Serious: 1/8 (13%)

Deaths: 0/8

NS-065/NCNP-01 80mg/kg

Serious: 2/8 (25%)

Deaths: 0/8

Total NS-065/NCNP-01 Group

Serious: 3/16 (19%)

Deaths: 0/16

Serious adverse events (3 terms)

Reaction	System	NS-065/NCNP-01 40mg/kg	NS-065/NCNP-01 80mg/kg	Total NS-065/NCNP-01 Group
Lower limb fracture	Injury, poisoning and procedural complications	—	—	—
Femur fracture	Injury, poisoning and procedural complications	—	—	—
Rhabdomyolysis	Musculoskeletal and connective tissue disorders	—	—	—

Other adverse events (100 terms — click to expand)

Reaction	System	NS-065/NCNP-01 40mg/kg	NS-065/NCNP-01 80mg/kg	Total NS-065/NCNP-01 Group
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—
Nasopharyngitis	Infections and infestations	—	—	—
Arthropod bite	Injury, poisoning and procedural complications	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—
Pyrexia	General disorders	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—
Nasal congestion	Respiratory, thoracic and mediastinal disorders	—	—	—
Headache	Nervous system disorders	—	—	—
Influenza	Infections and infestations	—	—	—
Contusion	Injury, poisoning and procedural complications	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	—	—	—
Constipation	Gastrointestinal disorders	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—
Upper respiratory tract infection	Infections and infestations	—	—	—
Body tinea	Infections and infestations	—	—	—
Ear infection	Infections and infestations	—	—	—
Pharyngitis streptococcal	Infections and infestations	—	—	—
Pneumonia	Infections and infestations	—	—	—
Respiratory tract infection viral	Infections and infestations	—	—	—
Sinusitis	Infections and infestations	—	—	—
Viral infection	Infections and infestations	—	—	—
Limb injury	Injury, poisoning and procedural complications	—	—	—
Lower limb fracture	Injury, poisoning and procedural complications	—	—	—
Thermal burn	Injury, poisoning and procedural complications	—	—	—
Respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	—	—	—
Abdominal pain upper	Gastrointestinal disorders	—	—	—
Haematochezia	Gastrointestinal disorders	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—
Ingrowing nail	Skin and subcutaneous tissue disorders	—	—	—
Anxiety	Psychiatric disorders	—	—	—
Bacterial infection	Infections and infestations	—	—	—
Bronchitis	Infections and infestations	—	—	—
Conjunctivitis	Infections and infestations	—	—	—
Coxsackie viral infection	Infections and infestations	—	—	—
Gastroenteritis	Infections and infestations	—	—	—
Hordeolum	Infections and infestations	—	—	—
Onychomycosis	Infections and infestations	—	—	—

Most-reported serious reactions: Lower limb fracture, Femur fracture, Rhabdomyolysis.

Data from ClinicalTrials.gov NCT03167255 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Duchenne muscular dystrophy: disease mechanism and therapeutic strategies.
Bez Batti Angulski A, Hosny N, Cohen H, Martin AA, et al · · 2023 · cited 109× · PMID 37435300 · DOI 10.3389/fphys.2023.1183101
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update.
Sun C, Shen L, Zhang Z, Xie X. · · 2020 · cited 109× · PMID 32717791 · DOI 10.3390/genes11080837
Muscle Wasting Diseases: Novel Targets and Treatments.
Furrer R, Handschin C. · · 2019 · cited 75× · PMID 30148697 · DOI 10.1146/annurev-pharmtox-010818-021041
Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches.
Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, et al · · 2019 · cited 62× · PMID 30621068 · DOI 10.3390/jpm9010001
Drug development progress in duchenne muscular dystrophy.
Deng J, Zhang J, Shi K, Liu Z. · · 2022 · cited 57× · PMID 35935842 · DOI 10.3389/fphar.2022.950651
Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.
Łoboda A, Dulak J. · · 2020 · cited 57× · PMID 32691346 · DOI 10.1007/s43440-020-00134-x
Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy.
Fortunato F, Rossi R, Falzarano MS, Ferlini A. · · 2021 · cited 53× · PMID 33671409 · DOI 10.3390/jcm10040820
Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Clemens PR, Rao VK, Connolly AM, Harper AD, et al · · 2022 · cited 39× · PMID 35634851 · DOI 10.3233/jnd-220811

Verify or expand the search:

Other recruiting trials for Duchenne Muscular Dystrophy

Currently open trials in the same condition.

NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients · Phase 2 · recruiting
NCT06817382 — A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Ma · Phase 1 · recruiting
NCT06402942 — Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy · NA · recruiting
NCT06450639 — A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) · Phase 2 · active not recruiting
NCT06692426 — Trial of Cell Based Therapy for DMD · Phase 1 · recruiting

Other NS Pharma, Inc. trials

Trials by the same sponsor.

NCT07441200 — Clinical Trial of NS-863 in Participants With Pulmonary Arterial Hypertension (PAH) · Phase 2 · not yet recruiting
NCT07441278 — A Clinical Trial of NS-863 in Participants With Pulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD · Phase 2 · not yet recruiting
NCT05996003 — NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) · Phase 2 · recruiting
NCT04854096 — Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis · Phase 2 · terminated
NCT04956289 — Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galact · Phase 2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03167255 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by NS Pharma, Inc.
Last refreshed: 28 December 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03167255.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing

NCT03167255

Quick facts

Drugs / interventions tested

Conditions studied

Sponsor

Who can join

Results — posted to ClinicalTrials.gov

Adverse events — posted to ClinicalTrials.gov

Serious adverse events (3 terms)

Sponsor's own description

Publications & conference data

Related trials

Other recruiting trials for Duchenne Muscular Dystrophy

Other NS Pharma, Inc. trials

Verify against primary sources