Duchenne Muscular Dystrophy
Conditions
Brief 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.
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.
Interventions
DRUGNS-065/NCNP-01
Received during weekly intravenous infusions
Sponsors
Nippon Shinyaku Co., Ltd.
Cooperative International Neuromuscular Research Group
Therapeutic Research in Neuromuscular Disorders Solutions
NS Pharma, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg
Eligibility
Sex/Gender
MALE
Age
4 Years to 10 Years
Healthy volunteers
No
Inclusion criteria
1. Completed Study NS-065/NCNP-01-201 through Week 25. 2. Willing and able to comply with scheduled visits, investigational product administration plan, and study procedures. 3. Stable dose of glucocorticoid (GC), and is expected to remain on the stable dose for the duration of the study.
Exclusion criteria
1. Serious or severe adverse event in Study NS-065/NCNP-01-201 that precludes safe use of NS-065/NCNP-01. 2. 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. 3. Patient took any other investigational drugs after completion of Study NS-065/NCNP-01-201. 4. Patient was judged by the investigator and/or the Sponsor that it was not appropriate to participate in the extension study for other reasons.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | 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 | 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. | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | 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) |
| Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | 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) |
| Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | 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). |
| Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | 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). |
| Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | 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 score of 34 implying normal function. |
| Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | 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) |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | 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 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. |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | 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 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. |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | 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 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. |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | 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 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. |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | 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 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. |
Countries
Canada, United States
Participant flow
Recruitment details
Participants were enrolled in the study at 6 sites in the United States and Canada from July 6, 2017 and August 25, 2018.
Pre-assignment details
Participants who completed study: NS-065/NCNP-01-201 (NCT02740972) were eligible to enroll in this long-term extension study.
Participants by arm
| Arm | Count |
|---|---|
| NS-065/NCNP-01 40mg/kg NS-065/NCNP-01 40mg/kg dose once a week for up to 192 weeks.
Baseline of Study NS-065/NCNP-01-202: Defined as the measurement taken on Study NS-065/NCNP-01-201 Week 25. | 8 |
| NS-065/NCNP-01 80mg/kg NS-065/NCNP-01 80mg/kg dose once a week for up to 192 weeks.
Baseline of Study NS-065/NCNP-01-202: Defined as the measurement taken on Study NS-065/NCNP-01-201 Week 25. | 8 |
| Total | 16 |
Baseline characteristics
| Characteristic | NS-065/NCNP-01 40mg/kg | NS-065/NCNP-01 80mg/kg | Total |
|---|---|---|---|
| Age, Continuous | 8.0 years STANDARD_DEVIATION 1.75 | 7.8 years STANDARD_DEVIATION 2.11 | 7.9 years STANDARD_DEVIATION 1.88 |
| Body Mass Index(BMI) | 18.4 kg/m^2 STANDARD_DEVIATION 2.17 | 17.0 kg/m^2 STANDARD_DEVIATION 2.54 | 17.7 kg/m^2 STANDARD_DEVIATION 2.39 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 8 Participants | 6 Participants | 14 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Height | 115.9 cm STANDARD_DEVIATION 7.54 | 115.3 cm STANDARD_DEVIATION 9.96 | 115.6 cm STANDARD_DEVIATION 8.54 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 8 Participants | 7 Participants | 15 Participants |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 8 Participants | 8 Participants | 16 Participants |
| Weight | 25.0 kg STANDARD_DEVIATION 4.95 | 23.0 kg STANDARD_DEVIATION 6.72 | 24.0 kg STANDARD_DEVIATION 5.79 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 8 | 0 / 8 | 0 / 16 |
| other Total, other adverse events | 8 / 8 | 8 / 8 | 16 / 16 |
| serious Total, serious adverse events | 1 / 8 | 2 / 8 | 3 / 16 |
Outcome results
Primary
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
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 37 | 0.03 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 49 | 0.03 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 73 | 0.03 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 109 | 0.04 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 157 | 0.02 rise/s | Standard Error 0.04 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 205 | -0.02 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 205 | -0.07 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 37 | 0.04 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 109 | 0.04 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 157 | -0.05 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 49 | 0.04 rise/s | Standard Error 0.03 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 73 | 0.50 rise/s | Standard Error 0.03 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 49 | 0.04 rise/s | Standard Error 0.02 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 73 | 0.04 rise/s | Standard Error 0.02 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 205 | -0.04 rise/s | Standard Error 0.02 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 109 | 0.04 rise/s | Standard Error 0.02 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 37 | 0.04 rise/s | Standard Error 0.02 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | Week 157 | -0.01 rise/s | Standard Error 0.02 |
Primary
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)
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 37 | 0.17 seconds | Standard Error 1.6 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 49 | 0.69 seconds | Standard Error 1.6 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 73 | 0.55 seconds | Standard Error 1.63 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 109 | 0.77 seconds | Standard Error 1.68 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 157 | 1.32 seconds | Standard Error 1.8 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 205 | 1.62 seconds | Standard Error 1.82 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 205 | 3.58 seconds | Standard Error 1.7 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 37 | -0.38 seconds | Standard Error 1.59 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 109 | 0.30 seconds | Standard Error 1.67 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 157 | 1.45 seconds | Standard Error 1.72 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 49 | -0.60 seconds | Standard Error 1.61 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 73 | 0.03 seconds | Standard Error 1.62 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 49 | 0.07 seconds | Standard Error 1.13 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 73 | 0.29 seconds | Standard Error 1.14 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 205 | 2.71 seconds | Standard Error 1.24 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 109 | 0.54 seconds | Standard Error 1.18 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 37 | -0.11 seconds | Standard Error 1.12 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | Week 157 | 1.44 seconds | Standard Error 1.23 |
Primary
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.
Time frame: Up to 192 weeks of treatment
Population: 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with TEAEs leading to discontinuation | 0 Participants |
| NS-065/NCNP-01 40mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with TEAE | 8 Participants |
| NS-065/NCNP-01 40mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Death | 0 Participants |
| NS-065/NCNP-01 40mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with drug-related TEAE | 0 Participants |
| NS-065/NCNP-01 40mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with CTCAE ≥ Grade 3 | 0 Participants |
| NS-065/NCNP-01 40mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with serious TEAE | 1 Participants |
| NS-065/NCNP-01 80mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with serious TEAE | 2 Participants |
| NS-065/NCNP-01 80mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with TEAEs leading to discontinuation | 0 Participants |
| NS-065/NCNP-01 80mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with CTCAE ≥ Grade 3 | 2 Participants |
| NS-065/NCNP-01 80mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with drug-related TEAE | 1 Participants |
| NS-065/NCNP-01 80mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Death | 0 Participants |
| NS-065/NCNP-01 80mg/kg | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with TEAE | 8 Participants |
| Total NS-065/NCNP-01 Group | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Death | 0 Participants |
| Total NS-065/NCNP-01 Group | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with serious TEAE | 3 Participants |
| Total NS-065/NCNP-01 Group | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with TEAE | 16 Participants |
| Total NS-065/NCNP-01 Group | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with CTCAE ≥ Grade 3 | 2 Participants |
| Total NS-065/NCNP-01 Group | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with TEAEs leading to discontinuation | 0 Participants |
| Total NS-065/NCNP-01 Group | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | Participants with drug-related TEAE | 1 Participants |
Secondary
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.
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 73 | 0.41 score on a scale | Standard Error 1.83 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 157 | -1.31 score on a scale | Standard Error 1.85 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 37 | 2.53 score on a scale | Standard Error 1.83 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 49 | 1.91 score on a scale | Standard Error 1.83 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 109 | 0.91 score on a scale | Standard Error 1.83 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 37 | -0.02 score on a scale | Standard Error 1.83 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 49 | 0.14 score on a scale | Standard Error 1.85 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 73 | 1.23 score on a scale | Standard Error 1.83 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 157 | 0.35 score on a scale | Standard Error 1.85 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 109 | 1.36 score on a scale | Standard Error 1.83 |
| Total NS-065/NCNP-01 Group | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 73 | 0.83 score on a scale | Standard Error 1.29 |
| Total NS-065/NCNP-01 Group | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 37 | 1.27 score on a scale | Standard Error 1.29 |
| Total NS-065/NCNP-01 Group | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 109 | 1.14 score on a scale | Standard Error 1.29 |
| Total NS-065/NCNP-01 Group | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 157 | -0.47 score on a scale | Standard Error 1.3 |
| Total NS-065/NCNP-01 Group | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | Week 49 | 1.05 score on a scale | Standard Error 1.29 |
Secondary
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.
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 37 | 0.55 lb | Standard Error 0.73 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 157 | 1.56 lb | Standard Error 0.91 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 49 | 0.65 lb | Standard Error 0.63 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 73 | 1.18 lb | Standard Error 0.66 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 49 | 0.48 lb | Standard Error 0.73 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 109 | 1.84 lb | Standard Error 0.63 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 205 | -0.06 lb | Standard Error 1.01 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 157 | 2.27 lb | Standard Error 0.81 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 205 | 0.74 lb | Standard Error 0.91 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 37 | 0.68 lb | Standard Error 0.76 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 49 | 0.54 lb | Standard Error 0.76 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 73 | 0.27 lb | Standard Error 0.76 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 73 | -1.02 lb | Standard Error 0.79 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 109 | 1.04 lb | Standard Error 0.76 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 109 | 1.23 lb | Standard Error 0.73 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 157 | 0.78 lb | Standard Error 0.93 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 205 | -0.11 lb | Standard Error 1.02 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 37 | 0.48 lb | Standard Error 0.63 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 157 | 0.22 lb | Standard Error 0.89 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 37 | 0.78 lb | Standard Error 0.67 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 37 | 1.21 lb | Standard Error 0.82 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 37 | 0.88 lb | Standard Error 0.78 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 49 | 1.19 lb | Standard Error 0.67 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 157 | -0.01 lb | Standard Error 0.86 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 109 | -0.07 lb | Standard Error 0.82 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 73 | 0.74 lb | Standard Error 0.7 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 49 | 0.53 lb | Standard Error 0.82 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 109 | -0.05 lb | Standard Error 0.78 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 109 | -0.22 lb | Standard Error 0.67 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 205 | 0.54 lb | Standard Error 0.95 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 73 | 1.08 lb | Standard Error 0.85 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 157 | -0.01 lb | Standard Error 0.75 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 205 | 0.07 lb | Standard Error 0.92 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 73 | 0.96 lb | Standard Error 0.81 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 205 | -0.39 lb | Standard Error 0.81 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 49 | 0.93 lb | Standard Error 0.78 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 205 | 0.17 lb | Standard Error 0.61 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 37 | 0.70 lb | Standard Error 0.53 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 49 | 0.68 lb | Standard Error 0.53 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 109 | 0.63 lb | Standard Error 0.53 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 157 | 0.70 lb | Standard Error 0.62 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 205 | 0.06 lb | Standard Error 0.67 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 37 | 0.92 lb | Standard Error 0.55 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 49 | 0.53 lb | Standard Error 0.55 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 73 | -0.05 lb | Standard Error 0.57 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 109 | 0.52 lb | Standard Error 0.55 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 157 | 0.43 lb | Standard Error 0.64 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Dominant Side Elbow Extensors, Week 205 | 0.25 lb | Standard Error 0.69 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 37 | 0.61 lb | Standard Error 0.46 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 49 | 0.90 lb | Standard Error 0.46 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 73 | 0.99 lb | Standard Error 0.48 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 109 | 0.87 lb | Standard Error 0.46 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Non-Dominant Side Elbow Extensors, Week 157 | 1.05 lb | Standard Error 0.55 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | Elbow Extensors, Week 73 | 0.60 lb | Standard Error 0.55 |
Secondary
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.
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 49 | 0.44 lb | Standard Error 0.68 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 49 | 0.57 lb | Standard Error 0.72 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 73 | -0.70 lb | Standard Error 0.75 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 109 | 0.53 lb | Standard Error 0.72 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 157 | -0.24 lb | Standard Error 0.89 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 109 | 0.71 lb | Standard Error 0.77 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 157 | -0.15 lb | Standard Error 0.85 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 205 | 0.74 lb | Standard Error 0.98 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 37 | -0.01 lb | Standard Error 0.77 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 49 | 0.48 lb | Standard Error 0.77 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 73 | -0.53 lb | Standard Error 0.8 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 157 | 0.00 lb | Standard Error 0.94 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 205 | 0.77 lb | Standard Error 1.04 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 37 | 0.42 lb | Standard Error 0.68 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 37 | -0.11 lb | Standard Error 0.72 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 73 | -0.45 lb | Standard Error 0.71 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 109 | 0.65 lb | Standard Error 0.68 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 205 | 0.47 lb | Standard Error 0.95 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 49 | 1.02 lb | Standard Error 0.73 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 205 | 0.07 lb | Standard Error 0.91 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 73 | 0.60 lb | Standard Error 0.76 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 73 | 0.97 lb | Standard Error 0.86 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 109 | 0.14 lb | Standard Error 0.83 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 205 | -0.45 lb | Standard Error 0.87 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 157 | 0.31 lb | Standard Error 0.91 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 109 | -0.21 lb | Standard Error 0.73 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 37 | 0.57 lb | Standard Error 0.78 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 49 | 0.63 lb | Standard Error 0.78 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 73 | 0.82 lb | Standard Error 0.81 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 37 | 0.84 lb | Standard Error 0.73 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 109 | 0.30 lb | Standard Error 0.78 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 157 | 1.05 lb | Standard Error 0.85 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 37 | 0.39 lb | Standard Error 0.83 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 49 | -0.38 lb | Standard Error 0.83 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 205 | 0.20 lb | Standard Error 0.97 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 157 | 1.52 lb | Standard Error 0.81 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 157 | 0.80 lb | Standard Error 0.59 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 109 | 0.42 lb | Standard Error 0.53 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 157 | 0.49 lb | Standard Error 0.61 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 49 | 0.71 lb | Standard Error 0.5 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 73 | 0.00 lb | Standard Error 0.55 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 205 | 0.34 lb | Standard Error 0.66 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 205 | -0.07 lb | Standard Error 0.64 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 37 | 0.18 lb | Standard Error 0.56 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 37 | 0.21 lb | Standard Error 0.53 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 49 | 0.08 lb | Standard Error 0.56 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 73 | 0.02 lb | Standard Error 0.52 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 205 | 0.46 lb | Standard Error 0.7 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 73 | 0.16 lb | Standard Error 0.58 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 37 | 0.61 lb | Standard Error 0.5 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 109 | 0.44 lb | Standard Error 0.56 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Elbow Flexors, Week 49 | 0.60 lb | Standard Error 0.53 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Non-Dominant Side Elbow Flexors, Week 109 | 0.25 lb | Standard Error 0.5 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | Dominant Side Elbow Flexors, Week 157 | 0.20 lb | Standard Error 0.65 |
Secondary
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.
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 37 | 0.56 lb | Standard Error 1.28 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 73 | 1.85 lb | Standard Error 1.32 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 205 | 7.34 lb | Standard Error 2.61 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 73 | 3.38 lb | Standard Error 1.24 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 157 | 4.67 lb | Standard Error 1.47 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 205 | 3.92 lb | Standard Error 2.53 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 49 | 1.63 lb | Standard Error 1.28 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 109 | 2.22 lb | Standard Error 1.28 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 157 | 2.94 lb | Standard Error 1.55 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 37 | 0.22 lb | Standard Error 1.28 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 49 | 1.17 lb | Standard Error 1.28 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 73 | 0.99 lb | Standard Error 1.33 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 109 | 1.86 lb | Standard Error 1.28 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 157 | 2.41 lb | Standard Error 1.57 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 205 | 7.37 lb | Standard Error 2.68 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 37 | 1.45 lb | Standard Error 1.2 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 49 | 1.95 lb | Standard Error 1.2 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 109 | 3.02 lb | Standard Error 1.2 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 49 | 1.99 lb | Standard Error 1.27 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 73 | 0.58 lb | Standard Error 1.33 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 205 | 4.48 lb | Standard Error 1.65 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 37 | 1.86 lb | Standard Error 1.42 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 49 | 2.34 lb | Standard Error 1.37 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 157 | 3.10 lb | Standard Error 1.59 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 37 | 2.19 lb | Standard Error 1.32 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 37 | 2.17 lb | Standard Error 1.42 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 49 | 2.70 lb | Standard Error 1.37 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 73 | 0.83 lb | Standard Error 1.42 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 109 | 2.00 lb | Standard Error 1.42 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 109 | 2.93 lb | Standard Error 1.33 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 205 | 4.05 lb | Standard Error 1.74 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 73 | 1.26 lb | Standard Error 1.43 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 109 | 1.61 lb | Standard Error 1.43 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 157 | 2.66 lb | Standard Error 1.61 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 205 | 4.36 lb | Standard Error 1.76 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 157 | 3.01 lb | Standard Error 1.5 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 205 | 5.00 lb | Standard Error 1.39 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 157 | 3.29 lb | Standard Error 1.14 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 49 | 1.88 lb | Standard Error 0.93 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 73 | 1.13 lb | Standard Error 0.96 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 205 | 2.94 lb | Standard Error 1.38 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 205 | 5.17 lb | Standard Error 1.42 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 37 | 1.09 lb | Standard Error 0.94 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 157 | 2.56 lb | Standard Error 1.11 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 109 | 2.12 lb | Standard Error 0.94 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 49 | 1.96 lb | Standard Error 0.93 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 73 | 1.39 lb | Standard Error 0.96 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 73 | 2.05 lb | Standard Error 0.98 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 109 | 3.08 lb | Standard Error 0.97 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Handgrip, Week 157 | 3.06 lb | Standard Error 1.1 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 109 | 1.76 lb | Standard Error 0.95 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 49 | 1.97 lb | Standard Error 0.96 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Dominant Side Handgrip, Week 37 | 1.03 lb | Standard Error 0.95 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | Non-Dominant Side Handgrip, Week 37 | 1.79 lb | Standard Error 0.96 |
Secondary
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.
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 37 | 1.72 lb | Standard Error 1.73 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 37 | 1.25 lb | Standard Error 1.93 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 205 | 0.63 lb | Standard Error 2.59 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 49 | 0.33 lb | Standard Error 1.82 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 157 | 2.97 lb | Standard Error 2.1 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 157 | 1.00 lb | Standard Error 2.1 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 73 | -1.45 lb | Standard Error 1.85 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 73 | -1.00 lb | Standard Error 1.96 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 109 | 1.19 lb | Standard Error 1.82 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 205 | 0.82 lb | Standard Error 2.57 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 109 | 0.89 lb | Standard Error 1.93 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 37 | -0.81 lb | Standard Error 1.82 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 109 | -0.39 lb | Standard Error 1.73 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 73 | -0.51 lb | Standard Error 1.78 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 157 | 3.01 lb | Standard Error 2.21 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 49 | 0.46 lb | Standard Error 1.73 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 205 | 1.16 lb | Standard Error 2.71 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 49 | 0.04 lb | Standard Error 1.93 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 109 | 1.14 lb | Standard Error 1.92 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 157 | 0.88 lb | Standard Error 2.02 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 37 | 1.10 lb | Standard Error 1.97 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 49 | 1.11 lb | Standard Error 1.96 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 73 | 2.57 lb | Standard Error 1.95 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 205 | -0.59 lb | Standard Error 2.15 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 37 | 1.87 lb | Standard Error 1.86 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 49 | 2.14 lb | Standard Error 1.88 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 73 | 1.11 lb | Standard Error 1.85 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 109 | 0.73 lb | Standard Error 1.81 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 157 | 1.28 lb | Standard Error 1.91 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 205 | 1.35 lb | Standard Error 2.04 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 37 | -0.12 lb | Standard Error 1.86 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 49 | 0.20 lb | Standard Error 1.86 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 73 | 1.03 lb | Standard Error 1.91 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 109 | 1.99 lb | Standard Error 1.86 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 157 | 0.42 lb | Standard Error 2.01 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 205 | -1.67 lb | Standard Error 2.08 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 205 | -0.11 lb | Standard Error 1.62 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 49 | 0.52 lb | Standard Error 1.37 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 37 | 0.86 lb | Standard Error 1.23 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 157 | 1.76 lb | Standard Error 1.55 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 205 | -0.58 lb | Standard Error 1.53 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 49 | 0.34 lb | Standard Error 1.23 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 109 | 1.03 lb | Standard Error 1.36 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 157 | 1.23 lb | Standard Error 1.46 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 73 | 0.18 lb | Standard Error 1.27 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 73 | -0.07 lb | Standard Error 1.3 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 73 | 0.87 lb | Standard Error 1.38 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 109 | 0.99 lb | Standard Error 1.28 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 49 | 1.16 lb | Standard Error 1.3 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Knee Extensors, Week 37 | 1.15 lb | Standard Error 1.38 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 157 | 1.51 lb | Standard Error 1.47 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 37 | 0.44 lb | Standard Error 1.3 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Non-Dominant Side Knee Extensors, Week 109 | 0.72 lb | Standard Error 1.23 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | Dominant Side Knee Extensors, Week 205 | 0.45 lb | Standard Error 1.55 |
Secondary
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.
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 157 | 2.43 lb | Standard Error 1.7 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 49 | 0.77 lb | Standard Error 1.48 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 73 | 1.04 lb | Standard Error 1.5 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 109 | 2.04 lb | Standard Error 1.48 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 37 | -0.35 lb | Standard Error 1.48 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 205 | 2.21 lb | Standard Error 2.09 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 37 | -0.69 lb | Standard Error 1.46 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 49 | 0.06 lb | Standard Error 1.46 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 73 | -0.23 lb | Standard Error 1.51 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 109 | 1.87 lb | Standard Error 1.46 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 157 | 3.00 lb | Standard Error 1.76 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 205 | 2.34 lb | Standard Error 1.93 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 37 | -0.78 lb | Standard Error 1.25 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 49 | 1.04 lb | Standard Error 1.25 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 73 | 0.93 lb | Standard Error 1.28 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 109 | 1.71 lb | Standard Error 1.25 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 157 | 1.35 lb | Standard Error 1.5 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 205 | 2.40 lb | Standard Error 1.84 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 205 | 2.22 lb | Standard Error 1.49 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 37 | 2.04 lb | Standard Error 1.56 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 109 | 2.63 lb | Standard Error 1.54 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 37 | 2.40 lb | Standard Error 1.33 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 49 | 1.20 lb | Standard Error 1.56 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 73 | 1.81 lb | Standard Error 1.37 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 109 | 2.17 lb | Standard Error 1.33 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 73 | 2.33 lb | Standard Error 1.6 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 157 | 2.26 lb | Standard Error 1.68 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 157 | 2.29 lb | Standard Error 1.44 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 109 | 2.73 lb | Standard Error 1.56 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 73 | 1.99 lb | Standard Error 1.6 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 49 | 1.05 lb | Standard Error 1.33 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 157 | 2.68 lb | Standard Error 1.66 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 49 | 1.77 lb | Standard Error 1.6 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 205 | 2.78 lb | Standard Error 1.79 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 205 | 2.87 lb | Standard Error 1.7 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 37 | 2.06 lb | Standard Error 1.54 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 205 | 2.24 lb | Standard Error 1.3 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 37 | 0.60 lb | Standard Error 1.07 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 49 | 0.84 lb | Standard Error 1.09 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 73 | 1.26 lb | Standard Error 0.95 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 73 | 0.80 lb | Standard Error 1.11 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 205 | 1.86 lb | Standard Error 1.14 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 109 | 2.24 lb | Standard Error 1.07 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 157 | 2.51 lb | Standard Error 1.22 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 109 | 1.90 lb | Standard Error 0.92 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Dominant Side Knee Flexors, Week 205 | 2.47 lb | Standard Error 1.31 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 37 | 0.82 lb | Standard Error 1.1 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 49 | 1.03 lb | Standard Error 1.1 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 37 | 0.67 lb | Standard Error 0.92 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 73 | 1.64 lb | Standard Error 1.12 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 109 | 2.42 lb | Standard Error 1.1 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Knee Flexors, Week 157 | 3.02 lb | Standard Error 1.26 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 49 | 1.02 lb | Standard Error 0.92 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | Non-Dominant Side Knee Flexors, Week 157 | 2.20 lb | Standard Error 1.09 |
Secondary
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)
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 37 | 15.15 m | Standard Error 26.84 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 49 | 9.90 m | Standard Error 26.84 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 73 | -0.73 m | Standard Error 26.84 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 109 | -16.35 m | Standard Error 26.84 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 157 | -41.61 m | Standard Error 28.08 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 49 | 16.94 m | Standard Error 29.7 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 73 | 18.14 m | Standard Error 26.89 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 157 | -27.76 m | Standard Error 29.71 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 37 | 13.03 m | Standard Error 28.15 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 109 | 18.51 m | Standard Error 26.89 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 37 | 14.30 m | Standard Error 19.29 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 49 | 13.32 m | Standard Error 19.75 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 109 | 1.10 m | Standard Error 18.87 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 157 | -34.86 m | Standard Error 20.28 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | Week 73 | 8.72 m | Standard Error 18.87 |
Secondary
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).
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 37 | 0.04 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 49 | 0.05 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 73 | 0.06 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 109 | 0.03 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 157 | 0.04 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 205 | 0.05 m/s | Standard Error 0.05 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 205 | -0.07 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 37 | 0.01 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 109 | -0.02 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 157 | -0.07 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 49 | 0.01 m/s | Standard Error 0.04 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 73 | 0.03 m/s | Standard Error 0.04 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 49 | 0.03 m/s | Standard Error 0.03 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 73 | 0.05 m/s | Standard Error 0.03 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 205 | -0.01 m/s | Standard Error 0.03 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 109 | 0.00 m/s | Standard Error 0.03 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 37 | 0.02 m/s | Standard Error 0.03 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | Week 157 | -0.01 m/s | Standard Error 0.03 |
Secondary
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)
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 157 | 4.28 seconds | Standard Error 1.55 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 49 | -0.26 seconds | Standard Error 1.54 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 109 | 1.02 seconds | Standard Error 1.54 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 205 | 3.00 seconds | Standard Error 1.66 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 73 | -0.08 seconds | Standard Error 1.54 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 37 | -0.37 seconds | Standard Error 1.54 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 73 | 0.44 seconds | Standard Error 1.54 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 109 | 1.08 seconds | Standard Error 1.54 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 37 | 0.38 seconds | Standard Error 1.54 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 157 | 2.95 seconds | Standard Error 1.55 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 205 | 3.18 seconds | Standard Error 1.58 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 49 | 0.28 seconds | Standard Error 1.56 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 49 | 0.02 seconds | Standard Error 1.09 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 205 | 3.16 seconds | Standard Error 1.14 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 37 | 0.01 seconds | Standard Error 1.09 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 157 | 3.61 seconds | Standard Error 1.09 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 73 | 0.18 seconds | Standard Error 1.09 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | Week 109 | 1.05 seconds | Standard Error 1.09 |
Secondary
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).
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 37 | 0.15 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 49 | 0.35 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 73 | 0.44 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 109 | 0.33 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 157 | 0.24 m/s | Standard Error 0.18 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 205 | 0.14 m/s | Standard Error 0.18 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 205 | -0.23 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 37 | 0.53 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 109 | 0.12 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 157 | -0.02 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 49 | 0.28 m/s | Standard Error 0.17 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 73 | 0.28 m/s | Standard Error 0.17 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 49 | 0.32 m/s | Standard Error 0.12 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 73 | 0.36 m/s | Standard Error 0.12 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 205 | -0.06 m/s | Standard Error 0.13 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 109 | 0.23 m/s | Standard Error 0.12 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 37 | 0.34 m/s | Standard Error 0.12 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | Week 157 | 0.11 m/s | Standard Error 0.12 |
Secondary
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)
Time frame: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Population: 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.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 205 | 0.89 seconds | Standard Error 1.1 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 73 | -1.15 seconds | Standard Error 1.06 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 109 | -0.67 seconds | Standard Error 1.06 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 157 | 0.13 seconds | Standard Error 1.07 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 49 | -0.87 seconds | Standard Error 1.06 |
| NS-065/NCNP-01 40mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 37 | -0.41 seconds | Standard Error 1.06 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 49 | -0.74 seconds | Standard Error 1.06 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 73 | -0.68 seconds | Standard Error 1.05 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 109 | -0.25 seconds | Standard Error 1.05 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 205 | 2.84 seconds | Standard Error 1.05 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 37 | -1.35 seconds | Standard Error 1.05 |
| NS-065/NCNP-01 80mg/kg | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 157 | 0.56 seconds | Standard Error 1.06 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 205 | 2.00 seconds | Standard Error 0.76 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 109 | -0.46 seconds | Standard Error 0.75 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 157 | 0.35 seconds | Standard Error 0.75 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 37 | -0.88 seconds | Standard Error 0.75 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 49 | -0.82 seconds | Standard Error 0.75 |
| Total NS-065/NCNP-01 Group | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | Week 73 | -0.91 seconds | Standard Error 0.75 |