Trial Outcomes & Findings for Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (NCT NCT03167255)
NCT ID: NCT03167255
Last Updated: 2022-12-28
Results Overview
A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202
Results posted on
2022-12-28
Participant Flow
Participants were enrolled in the study at 6 sites in the United States and Canada from July 6, 2017 and August 25, 2018.
Participants who completed study: NS-065/NCNP-01-201 (NCT02740972) were eligible to enroll in this long-term extension study.
Participant milestones
| Measure |
NS-065/NCNP-01 40mg/kg
Patients who received 40mg/kg in the NS-065-NCNP-201 study, continued the same treatment with 40mg/kg once weekly for 192 weeks in this extension study.
NS-065/NCNP-01: Received during weekly intravenous infusions
|
NS-065/NCNP-01 80mg/kg
Patients who received 80mg/kg in the NS-065-NCNP-201 study, continued the same treatment with 80mg/kg once weekly for 192 weeks in this extension study.
NS-065/NCNP-01: Received during weekly intravenous infusions
|
|---|---|---|
|
Overall Study
STARTED
|
8
|
8
|
|
Overall Study
COMPLETED
|
8
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Baseline characteristics by cohort
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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.
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
8.0 years
STANDARD_DEVIATION 1.75 • n=5 Participants
|
7.8 years
STANDARD_DEVIATION 2.11 • n=7 Participants
|
7.9 years
STANDARD_DEVIATION 1.88 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Weight
|
25.0 kg
STANDARD_DEVIATION 4.95 • n=5 Participants
|
23.0 kg
STANDARD_DEVIATION 6.72 • n=7 Participants
|
24.0 kg
STANDARD_DEVIATION 5.79 • n=5 Participants
|
|
Height
|
115.9 cm
STANDARD_DEVIATION 7.54 • n=5 Participants
|
115.3 cm
STANDARD_DEVIATION 9.96 • n=7 Participants
|
115.6 cm
STANDARD_DEVIATION 8.54 • n=5 Participants
|
|
Body Mass Index(BMI)
|
18.4 kg/m^2
STANDARD_DEVIATION 2.17 • n=5 Participants
|
17.0 kg/m^2
STANDARD_DEVIATION 2.54 • n=7 Participants
|
17.7 kg/m^2
STANDARD_DEVIATION 2.39 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND)
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Week 37
|
0.17 seconds
Standard Error 1.60
|
-0.38 seconds
Standard Error 1.59
|
-0.11 seconds
Standard Error 1.12
|
|
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Week 49
|
0.69 seconds
Standard Error 1.60
|
-0.60 seconds
Standard Error 1.61
|
0.07 seconds
Standard Error 1.13
|
|
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Week 73
|
0.55 seconds
Standard Error 1.63
|
0.03 seconds
Standard Error 1.62
|
0.29 seconds
Standard Error 1.14
|
|
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Week 109
|
0.77 seconds
Standard Error 1.68
|
0.30 seconds
Standard Error 1.67
|
0.54 seconds
Standard Error 1.18
|
|
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Week 157
|
1.32 seconds
Standard Error 1.80
|
1.45 seconds
Standard Error 1.72
|
1.44 seconds
Standard Error 1.23
|
|
Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls
Week 205
|
1.62 seconds
Standard Error 1.82
|
3.58 seconds
Standard Error 1.70
|
2.71 seconds
Standard Error 1.24
|
PRIMARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Week 73
|
0.03 rise/s
Standard Error 0.03
|
0.50 rise/s
Standard Error 0.03
|
0.04 rise/s
Standard Error 0.02
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Week 109
|
0.04 rise/s
Standard Error 0.03
|
0.04 rise/s
Standard Error 0.03
|
0.04 rise/s
Standard Error 0.02
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Week 157
|
0.02 rise/s
Standard Error 0.04
|
-0.05 rise/s
Standard Error 0.03
|
-0.01 rise/s
Standard Error 0.02
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Week 205
|
-0.02 rise/s
Standard Error 0.03
|
-0.07 rise/s
Standard Error 0.03
|
-0.04 rise/s
Standard Error 0.02
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Week 37
|
0.03 rise/s
Standard Error 0.03
|
0.04 rise/s
Standard Error 0.03
|
0.04 rise/s
Standard Error 0.02
|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls
Week 49
|
0.03 rise/s
Standard Error 0.03
|
0.04 rise/s
Standard Error 0.03
|
0.04 rise/s
Standard Error 0.02
|
PRIMARY outcome
Timeframe: Up to 192 weeks of treatmentPopulation: 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.
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.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Participants with TEAE
|
8 Participants
|
8 Participants
|
16 Participants
|
|
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Participants with drug-related TEAE
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Participants with CTCAE ≥ Grade 3
|
0 Participants
|
2 Participants
|
2 Participants
|
|
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Participants with TEAEs leading to discontinuation
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Participants with serious TEAE
|
1 Participants
|
2 Participants
|
3 Participants
|
|
Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0.
Death
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW)
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
-1.35 seconds
Standard Error 1.05
|
-0.88 seconds
Standard Error 0.75
|
|
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
|
-0.74 seconds
Standard Error 1.06
|
-0.82 seconds
Standard Error 0.75
|
|
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
|
-0.68 seconds
Standard Error 1.05
|
-0.91 seconds
Standard Error 0.75
|
|
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
|
-0.25 seconds
Standard Error 1.05
|
-0.46 seconds
Standard Error 0.75
|
|
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
|
0.56 seconds
Standard Error 1.06
|
0.35 seconds
Standard Error 0.75
|
|
Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls
Week 205
|
0.89 seconds
Standard Error 1.10
|
2.84 seconds
Standard Error 1.05
|
2.00 seconds
Standard Error 0.76
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
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).
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
0.53 m/s
Standard Error 0.17
|
0.34 m/s
Standard Error 0.12
|
|
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
|
0.28 m/s
Standard Error 0.17
|
0.32 m/s
Standard Error 0.12
|
|
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
|
0.28 m/s
Standard Error 0.17
|
0.36 m/s
Standard Error 0.12
|
|
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
|
0.12 m/s
Standard Error 0.17
|
0.23 m/s
Standard Error 0.12
|
|
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
|
-0.02 m/s
Standard Error 0.17
|
0.11 m/s
Standard Error 0.12
|
|
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
|
-0.23 m/s
Standard Error 0.17
|
-0.06 m/s
Standard Error 0.13
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB)
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
Week 37
|
-0.37 seconds
Standard Error 1.54
|
0.38 seconds
Standard Error 1.54
|
0.01 seconds
Standard Error 1.09
|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
Week 49
|
-0.26 seconds
Standard Error 1.54
|
0.28 seconds
Standard Error 1.56
|
0.02 seconds
Standard Error 1.09
|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
Week 73
|
-0.08 seconds
Standard Error 1.54
|
0.44 seconds
Standard Error 1.54
|
0.18 seconds
Standard Error 1.09
|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
Week 109
|
1.02 seconds
Standard Error 1.54
|
1.08 seconds
Standard Error 1.54
|
1.05 seconds
Standard Error 1.09
|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
Week 157
|
4.28 seconds
Standard Error 1.55
|
2.95 seconds
Standard Error 1.55
|
3.61 seconds
Standard Error 1.09
|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls
Week 205
|
3.00 seconds
Standard Error 1.66
|
3.18 seconds
Standard Error 1.58
|
3.16 seconds
Standard Error 1.14
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
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).
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
0.03 m/s
Standard Error 0.04
|
0.05 m/s
Standard Error 0.03
|
|
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
|
-0.07 m/s
Standard Error 0.04
|
-0.01 m/s
Standard Error 0.03
|
|
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
|
0.01 m/s
Standard Error 0.04
|
0.02 m/s
Standard Error 0.03
|
|
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
|
0.01 m/s
Standard Error 0.04
|
0.03 m/s
Standard Error 0.03
|
|
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
|
-0.02 m/s
Standard Error 0.04
|
0.00 m/s
Standard Error 0.03
|
|
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
|
-0.07 m/s
Standard Error 0.04
|
-0.01 m/s
Standard Error 0.03
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202Population: 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.
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.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
-0.02 score on a scale
Standard Error 1.83
|
1.27 score on a scale
Standard Error 1.29
|
|
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
|
0.14 score on a scale
Standard Error 1.85
|
1.05 score on a scale
Standard Error 1.29
|
|
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
|
1.23 score on a scale
Standard Error 1.83
|
0.83 score on a scale
Standard Error 1.29
|
|
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
|
1.36 score on a scale
Standard Error 1.83
|
1.14 score on a scale
Standard Error 1.29
|
|
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
|
0.35 score on a scale
Standard Error 1.85
|
-0.47 score on a scale
Standard Error 1.30
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202Population: 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.
A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Six-Minute Walk Test (6MWT)
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
Week 109
|
-16.35 m
Standard Error 26.84
|
18.51 m
Standard Error 26.89
|
1.10 m
Standard Error 18.87
|
|
Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
Week 37
|
15.15 m
Standard Error 26.84
|
13.03 m
Standard Error 28.15
|
14.30 m
Standard Error 19.29
|
|
Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
Week 49
|
9.90 m
Standard Error 26.84
|
16.94 m
Standard Error 29.70
|
13.32 m
Standard Error 19.75
|
|
Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
Week 73
|
-0.73 m
Standard Error 26.84
|
18.14 m
Standard Error 26.89
|
8.72 m
Standard Error 18.87
|
|
Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls
Week 157
|
-41.61 m
Standard Error 28.08
|
-27.76 m
Standard Error 29.71
|
-34.86 m
Standard Error 20.28
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
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.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
2.66 lb
Standard Error 1.61
|
2.56 lb
Standard Error 1.11
|
|
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 1.20
|
1.99 lb
Standard Error 1.27
|
1.97 lb
Standard Error 0.96
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
Handgrip, Week 37
|
0.56 lb
Standard Error 1.28
|
1.86 lb
Standard Error 1.42
|
1.09 lb
Standard Error 0.94
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
Handgrip, Week 49
|
1.63 lb
Standard Error 1.28
|
2.34 lb
Standard Error 1.37
|
1.96 lb
Standard Error 0.93
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
Handgrip, Week 73
|
1.85 lb
Standard Error 1.32
|
0.83 lb
Standard Error 1.42
|
1.39 lb
Standard Error 0.96
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
Handgrip, Week 109
|
2.22 lb
Standard Error 1.28
|
2.00 lb
Standard Error 1.42
|
2.12 lb
Standard Error 0.94
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
Handgrip, Week 157
|
2.94 lb
Standard Error 1.55
|
3.10 lb
Standard Error 1.59
|
3.06 lb
Standard Error 1.10
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls
Handgrip, Week 205
|
7.34 lb
Standard Error 2.61
|
4.05 lb
Standard Error 1.74
|
5.00 lb
Standard Error 1.39
|
|
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
|
2.17 lb
Standard Error 1.42
|
1.03 lb
Standard Error 0.95
|
|
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
|
2.70 lb
Standard Error 1.37
|
1.88 lb
Standard Error 0.93
|
|
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
|
1.26 lb
Standard Error 1.43
|
1.13 lb
Standard Error 0.96
|
|
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
|
1.61 lb
Standard Error 1.43
|
1.76 lb
Standard Error 0.95
|
|
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
|
4.36 lb
Standard Error 1.76
|
5.17 lb
Standard Error 1.42
|
|
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 1.20
|
2.19 lb
Standard Error 1.32
|
1.79 lb
Standard Error 0.96
|
|
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
|
0.58 lb
Standard Error 1.33
|
2.05 lb
Standard Error 0.98
|
|
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 1.20
|
2.93 lb
Standard Error 1.33
|
3.08 lb
Standard Error 0.97
|
|
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
|
3.01 lb
Standard Error 1.50
|
3.29 lb
Standard Error 1.14
|
|
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
|
4.48 lb
Standard Error 1.65
|
2.94 lb
Standard Error 1.38
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
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.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
0.57 lb
Standard Error 0.78
|
0.21 lb
Standard Error 0.53
|
|
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
|
0.63 lb
Standard Error 0.78
|
0.60 lb
Standard Error 0.53
|
|
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
|
0.82 lb
Standard Error 0.81
|
0.00 lb
Standard Error 0.55
|
|
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
|
0.30 lb
Standard Error 0.78
|
0.42 lb
Standard Error 0.53
|
|
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
|
1.05 lb
Standard Error 0.85
|
0.49 lb
Standard Error 0.61
|
|
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
|
0.07 lb
Standard Error 0.91
|
0.34 lb
Standard Error 0.66
|
|
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
|
0.39 lb
Standard Error 0.83
|
0.18 lb
Standard Error 0.56
|
|
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
|
-0.38 lb
Standard Error 0.83
|
0.08 lb
Standard Error 0.56
|
|
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 0.80
|
0.97 lb
Standard Error 0.86
|
0.16 lb
Standard Error 0.58
|
|
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
|
0.14 lb
Standard Error 0.83
|
0.44 lb
Standard Error 0.56
|
|
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
|
0.31 lb
Standard Error 0.91
|
0.20 lb
Standard Error 0.65
|
|
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
|
0.20 lb
Standard Error 0.97
|
0.46 lb
Standard Error 0.70
|
|
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
|
0.84 lb
Standard Error 0.73
|
0.61 lb
Standard Error 0.50
|
|
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
|
1.02 lb
Standard Error 0.73
|
0.71 lb
Standard Error 0.50
|
|
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
|
0.60 lb
Standard Error 0.76
|
0.02 lb
Standard Error 0.52
|
|
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
|
-0.21 lb
Standard Error 0.73
|
0.25 lb
Standard Error 0.50
|
|
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
|
1.52 lb
Standard Error 0.81
|
0.80 lb
Standard Error 0.59
|
|
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
|
-0.45 lb
Standard Error 0.87
|
-0.07 lb
Standard Error 0.64
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
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.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
-0.39 lb
Standard Error 0.81
|
0.17 lb
Standard Error 0.61
|
|
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
|
1.19 lb
Standard Error 0.67
|
0.90 lb
Standard Error 0.46
|
|
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
|
0.74 lb
Standard Error 0.70
|
0.99 lb
Standard Error 0.48
|
|
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
|
-0.22 lb
Standard Error 0.67
|
0.87 lb
Standard Error 0.46
|
|
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
|
-0.01 lb
Standard Error 0.75
|
1.05 lb
Standard Error 0.55
|
|
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
|
0.88 lb
Standard Error 0.78
|
0.70 lb
Standard Error 0.53
|
|
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
|
0.93 lb
Standard Error 0.78
|
0.68 lb
Standard Error 0.53
|
|
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
|
0.96 lb
Standard Error 0.81
|
0.60 lb
Standard Error 0.55
|
|
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
|
-0.05 lb
Standard Error 0.78
|
0.63 lb
Standard Error 0.53
|
|
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
|
-0.01 lb
Standard Error 0.86
|
0.70 lb
Standard Error 0.62
|
|
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
|
0.07 lb
Standard Error 0.92
|
0.06 lb
Standard Error 0.67
|
|
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
|
1.21 lb
Standard Error 0.82
|
0.92 lb
Standard Error 0.55
|
|
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
|
0.53 lb
Standard Error 0.82
|
0.53 lb
Standard Error 0.55
|
|
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
|
1.08 lb
Standard Error 0.85
|
-0.05 lb
Standard Error 0.57
|
|
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
|
-0.07 lb
Standard Error 0.82
|
0.52 lb
Standard Error 0.55
|
|
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
|
0.22 lb
Standard Error 0.89
|
0.43 lb
Standard Error 0.64
|
|
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
|
0.54 lb
Standard Error 0.95
|
0.25 lb
Standard Error 0.69
|
|
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
|
0.78 lb
Standard Error 0.67
|
0.61 lb
Standard Error 0.46
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
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.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
2.04 lb
Standard Error 1.56
|
0.82 lb
Standard Error 1.10
|
|
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
|
1.20 lb
Standard Error 1.56
|
1.03 lb
Standard Error 1.10
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls
Knee Flexors, Week 73
|
1.04 lb
Standard Error 1.50
|
2.33 lb
Standard Error 1.60
|
1.64 lb
Standard Error 1.12
|
|
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
|
2.73 lb
Standard Error 1.56
|
2.42 lb
Standard Error 1.10
|
|
Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls
Knee Flexors, Week 157
|
2.43 lb
Standard Error 1.70
|
2.68 lb
Standard Error 1.66
|
3.02 lb
Standard Error 1.26
|
|
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
|
2.87 lb
Standard Error 1.70
|
2.24 lb
Standard Error 1.30
|
|
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
|
2.06 lb
Standard Error 1.54
|
0.60 lb
Standard Error 1.07
|
|
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
|
1.77 lb
Standard Error 1.60
|
0.84 lb
Standard Error 1.09
|
|
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
|
1.99 lb
Standard Error 1.60
|
0.80 lb
Standard Error 1.11
|
|
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
|
2.63 lb
Standard Error 1.54
|
2.24 lb
Standard Error 1.07
|
|
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
|
2.26 lb
Standard Error 1.68
|
2.51 lb
Standard Error 1.22
|
|
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
|
2.78 lb
Standard Error 1.79
|
2.47 lb
Standard Error 1.31
|
|
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
|
2.17 lb
Standard Error 1.33
|
1.90 lb
Standard Error 0.92
|
|
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
|
2.40 lb
Standard Error 1.33
|
0.67 lb
Standard Error 0.92
|
|
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
|
1.05 lb
Standard Error 1.33
|
1.02 lb
Standard Error 0.92
|
|
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
|
1.81 lb
Standard Error 1.37
|
1.26 lb
Standard Error 0.95
|
|
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 1.50
|
2.29 lb
Standard Error 1.44
|
2.20 lb
Standard Error 1.09
|
|
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
|
2.22 lb
Standard Error 1.49
|
1.86 lb
Standard Error 1.14
|
SECONDARY outcome
Timeframe: Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202Population: 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.
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.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
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 Visit.
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
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 Visit.
|
Total NS-065/NCNP-01 Group
n=16 Participants
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
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
|
1.10 lb
Standard Error 1.97
|
1.15 lb
Standard Error 1.38
|
|
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
|
1.11 lb
Standard Error 1.96
|
0.52 lb
Standard Error 1.37
|
|
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
|
2.57 lb
Standard Error 1.95
|
0.87 lb
Standard Error 1.38
|
|
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
|
1.14 lb
Standard Error 1.92
|
1.03 lb
Standard Error 1.36
|
|
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
|
0.88 lb
Standard Error 2.02
|
1.76 lb
Standard Error 1.55
|
|
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
|
-0.59 lb
Standard Error 2.15
|
-0.11 lb
Standard Error 1.62
|
|
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
|
1.87 lb
Standard Error 1.86
|
0.44 lb
Standard Error 1.30
|
|
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
|
2.14 lb
Standard Error 1.88
|
1.16 lb
Standard Error 1.30
|
|
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
|
1.11 lb
Standard Error 1.85
|
-0.07 lb
Standard Error 1.30
|
|
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
|
0.73 lb
Standard Error 1.81
|
0.99 lb
Standard Error 1.28
|
|
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 2.10
|
1.28 lb
Standard Error 1.91
|
1.51 lb
Standard Error 1.47
|
|
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
|
1.35 lb
Standard Error 2.04
|
0.45 lb
Standard Error 1.55
|
|
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
|
-0.12 lb
Standard Error 1.86
|
0.86 lb
Standard Error 1.23
|
|
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
|
0.20 lb
Standard Error 1.86
|
0.34 lb
Standard Error 1.23
|
|
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
|
1.03 lb
Standard Error 1.91
|
0.18 lb
Standard Error 1.27
|
|
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
|
1.99 lb
Standard Error 1.86
|
0.72 lb
Standard Error 1.23
|
|
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 2.10
|
0.42 lb
Standard Error 2.01
|
1.23 lb
Standard Error 1.46
|
|
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
|
-1.67 lb
Standard Error 2.08
|
-0.58 lb
Standard Error 1.53
|
Adverse Events
NS-065/NCNP-01 40mg/kg
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
NS-065/NCNP-01 80mg/kg
Serious events: 2 serious events
Other events: 8 other events
Deaths: 0 deaths
Total NS-065/NCNP-01 Group
Serious events: 3 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 participants at risk
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.
|
NS-065/NCNP-01 80mg/kg
n=8 participants at risk
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.
|
Total NS-065/NCNP-01 Group
n=16 participants at risk
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Femur fracture
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
Other adverse events
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 participants at risk
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.
|
NS-065/NCNP-01 80mg/kg
n=8 participants at risk
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.
|
Total NS-065/NCNP-01 Group
n=16 participants at risk
All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population.
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Sunburn
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Nasopharyngitis
|
50.0%
4/8 • Number of events 10 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
62.5%
5/8 • Number of events 16 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
56.2%
9/16 • Number of events 26 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Influenza
|
37.5%
3/8 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
4/16 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Body tinea
|
25.0%
2/8 • Number of events 3 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 3 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Ear infection
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Pharyngitis streptococcal
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Pneumonia
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Respiratory tract infection viral
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Sinusitis
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Viral infection
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Bacterial infection
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Bronchitis
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Conjunctivitis
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Coxsackie viral infection
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Hordeolum
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Onychomycosis
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Otitis externa
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Fall
|
50.0%
4/8 • Number of events 9 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
31.2%
5/16 • Number of events 10 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
50.0%
4/8 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
37.5%
6/16 • Number of events 7 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Contusion
|
37.5%
3/8 • Number of events 3 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
4/16 • Number of events 4 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Laceration
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Animal bite
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Femur fracture
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Foot fracture
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Foreign body in respiratory tract
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Incision site swelling
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Incision site vesicles
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Limb fracture
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Skin injury
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Spinal column injury
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Traumatic tooth displacement
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Vascular access complication
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
62.5%
5/8 • Number of events 7 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
62.5%
5/8 • Number of events 9 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
62.5%
10/16 • Number of events 16 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
37.5%
3/8 • Number of events 7 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
31.2%
5/16 • Number of events 9 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
18.8%
3/16 • Number of events 3 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
25.0%
2/8 • Number of events 3 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 3 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Allergic sinusitis
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory symptom
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Vomiting
|
37.5%
3/8 • Number of events 7 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
37.5%
3/8 • Number of events 7 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
37.5%
6/16 • Number of events 14 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Constipation
|
25.0%
2/8 • Number of events 4 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
18.8%
3/16 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 4 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
18.8%
3/16 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Abdominal pain upper
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Haematochezia
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Dyspepsia
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Nausea
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Tooth development disorder
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Toothache
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Nervous system disorders
Headache
|
37.5%
3/8 • Number of events 21 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 6 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
31.2%
5/16 • Number of events 27 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Nervous system disorders
Dizziness
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Nervous system disorders
Seizure
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Nervous system disorders
Sinus headache
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Pyrexia
|
25.0%
2/8 • Number of events 6 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
50.0%
4/8 • Number of events 6 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
37.5%
6/16 • Number of events 12 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Catheter site erythema
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Catheter site swelling
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Fatigue
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Infusion site pain
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Injection site extravasation
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Pain
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Peripheral swelling
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Tenderness
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
37.5%
3/8 • Number of events 4 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
4/16 • Number of events 4 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
18.8%
3/16 • Number of events 3 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Joint hyperextension
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Spinal deformity
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
50.0%
4/8 • Number of events 5 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
37.5%
6/16 • Number of events 7 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
25.0%
2/8 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Erythema
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Onycholysis
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Psychiatric disorders
Anxiety
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • Number of events 2 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Psychiatric disorders
Attention deficit/hyperactivity disorder
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Psychiatric disorders
Flat affect
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Investigations
Body height decreased
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Investigations
Vitamin D decreased
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Investigations
Weight increased
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Renal and urinary disorders
Hypercalciuria
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Renal and urinary disorders
Pyelocaliectasis
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Cardiac disorders
Sinus arrhythmia
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Congenital, familial and genetic disorders
Ankyloglossia congenital
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Endocrine disorders
Cushingoid
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Eye disorders
Cataract
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Immune system disorders
Hypersensitivity
|
0.00%
0/8 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • Number of events 1 • Up to 192 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The most restrictive relevant agreement on the PI provides that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is not less than 30 days from the time submitted to the sponsor for review.
- Publication restrictions are in place
Restriction type: OTHER