Duchenne Muscular DystrophyPhase 2NCT03167255

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

A Phase 2 study testing NS-065/NCNP-01 in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 6 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 4 to 10 years, men only
Conditions the study is for: Duchenne Muscular Dystrophy
What is being tested?: NS-065/NCNP-01
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 16 participants
Where: 6 sitesin 2 countries
Study dates: 6 Jul 2017to 15 Nov 2021
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 4 to 10 years
Men only
No healthy volunteers
Child

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

6 sites in 2 countries.

Country

City, postcode or hospital

6 sites in 2 countries

United States5 sites

Lurie Children's Hospital
Chicago, Illinois, 60611
Duke University Medical Center
Durham, North Carolina, 27710
Children's Hospital of Richmond at VCU
Richmond, Virginia, 23298
UC Davis
Sacramento, California, 95817
Washington University
St Louis, Missouri, 63110

Other trials in United States

Canada1 site

Alberta Children's Hospital
Calgary, Alberta

Other trials in Canada

About this study

Official title: A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

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.

Read the detailed description

This is a Phase II, multicenter, 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. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.

Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.

Conditions: Duchenne Muscular Dystrophy
Being tested: NS-065/NCNP-01
Medical subject headings: Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NS-065/NCNP-01Drug
Received during weekly intravenous infusions

Study groups in NCT03167255
Study group	Role	What they receive
NS-065/NCNP-01 40mg/kg	Experimental	Patients receiving 40mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier. Receives: Drug: NS-065/NCNP-01
NS-065/NCNP-01 80mg/kg	Experimental	Patients receiving 80mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier. Receives: Drug: NS-065/NCNP-01

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND)
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
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.
Measured: Up to 192 weeks of treatment

Secondary outcomes (11)

Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW)
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls
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).
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB)
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls
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).
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls
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 score of 34 implying normal function.
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Six-Minute Walk Test (6MWT)
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
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 QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls
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 scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls
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 scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee 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 scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee 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 scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge.
Measured: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

6 Jul 201715 Nov 2021

First posted25 May 2017
Study start6 Jul 2017
Primary completion20 Oct 2021
Study completion15 Nov 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Model description: Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg
Masking: None (open label)
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03167255
Sponsor's protocol ID: NS-065/NCNP-01-202
Status last verified: Dec 2022
First submitted: 22 May 2017
Last updated: 28 Dec 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
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Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
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Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
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All Duchenne Muscular Dystrophy trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
ClinicalTrials.gov ID: NCT03167255
Acronym / sponsor ID: NS-065/NCNP-01-202
Conditions: Duchenne Muscular Dystrophy
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: NS Pharma, Inc.Industry (pharma, biotech, devices)
Collaborators: Nippon Shinyaku Co., Ltd., Cooperative International Neuromuscular Research Group, Therapeutic Research in Neuromuscular Disorders Solutions
Study start: 6 Jul 2017 (actual)
Primary completion: 20 Oct 2021 (actual)
Study completion: 15 Nov 2021 (actual)
First posted: 25 May 2017 (actual)
Results: Posted 18 Nov 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2
Model description: Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg

Arm	Type	Receives
NS-065/NCNP-01 40mg/kg	Experimental	Drug: NS-065/NCNP-01Patients receiving 40mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
NS-065/NCNP-01 80mg/kg	Experimental	Drug: NS-065/NCNP-01Patients receiving 80mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.

Who may qualify

Ages: 4 to 10 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (3 listed)

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.

Key exclusion criteria (4 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
NS-065/NCNP-01Received during weekly intravenous infusions	Drug	NS-065/NCNP-01 40mg/kg, NS-065/NCNP-01 80mg/kg

Primary outcome

Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Time frame: 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)
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Time frame: 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
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Time frame: 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.

11 secondary outcomes

Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202

Results and safety

Results posted to the registry on 18 Nov 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
NS-065/NCNP-01 40mg/kg	8	8
NS-065/NCNP-01 80mg/kg	8	8

Primary outcome: Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls

Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202. Least squares mean (Standard Error). Unit: seconds

Measure	NS-065/NCNP-01 40mg/kg n = 8	NS-065/NCNP-01 80mg/kg n = 8	Total NS-065/NCNP-01 Group n = 16
Week 37	0.17 (1.60)	-0.38 (1.59)	-0.11 (1.12)
Week 49	0.69 (1.60)	-0.60 (1.61)	0.07 (1.13)
Week 73	0.55 (1.63)	0.03 (1.62)	0.29 (1.14)
Week 109	0.77 (1.68)	0.30 (1.67)	0.54 (1.18)
Week 157	1.32 (1.80)	1.45 (1.72)	1.44 (1.23)
Week 205	1.62 (1.82)	3.58 (1.70)	2.71 (1.24)

Population analysed: The analysis population included all participants who received at least one dose of investigational drug. Number of participants analyzed signifies number of participants who were evaluable for this outcome measure.

Primary outcome: Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls

Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202. Least squares mean (Standard Error). Unit: rise/s

Measure	NS-065/NCNP-01 40mg/kg n = 8	NS-065/NCNP-01 80mg/kg n = 8	Total NS-065/NCNP-01 Group n = 16
Week 37	0.03 (0.03)	0.04 (0.03)	0.04 (0.02)
Week 49	0.03 (0.03)	0.04 (0.03)	0.04 (0.02)
Week 73	0.03 (0.03)	0.50 (0.03)	0.04 (0.02)
Week 109	0.04 (0.03)	0.04 (0.03)	0.04 (0.02)
Week 157	0.02 (0.04)	-0.05 (0.03)	-0.01 (0.02)
Week 205	-0.02 (0.03)	-0.07 (0.03)	-0.04 (0.02)

Primary outcome: Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.

Time frame: Up to 192 weeks of treatment. Count of participants. Unit: Participants

Measure	NS-065/NCNP-01 40mg/kg n = 8	NS-065/NCNP-01 80mg/kg n = 8	Total NS-065/NCNP-01 Group n = 16
Participants with TEAE	8	8	16
Participants with drug-related TEAE	0	1	1
Participants with CTCAE ≥ Grade 3	0	2	2
Participants with TEAEs leading to discontinuation	0	0	0
Participants with serious TEAE	1	2	3
Death	0	0	0

11 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 192 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
NS-065/NCNP-01 40mg/kg	1 of 8 (12.5%)	8 of 8 (100%)	0 of 8
NS-065/NCNP-01 80mg/kg	2 of 8 (25%)	8 of 8 (100%)	0 of 8
Total NS-065/NCNP-01 Group	3 of 16 (18.8%)	16 of 16 (100%)	0 of 16

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Dec 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Paula R. Clemens, MD study chair

University of Pittsburgh

6 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NS-065/NCNP-01 in people with Duchenne Muscular Dystrophy, run by NS Pharma, Inc. at 6 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Nov 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls (time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03167255.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Dec 2022.