Trial Outcomes & Findings for Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (NCT NCT02740972)
NCT ID: NCT02740972
Last Updated: 2021-12-07
Results Overview
Treatment emergent adverse events (TEAEs) were summarized for Period 1 by comparing low dose to high dose to placebo and for Period 2 between the low dose cohort and the high dose cohort. TEAEs were summarized both at the patient level for number of TEAEs, highest severity, relationship, action and outcome and at the TEAE level (summarizing events) by organ system and preferred term TEAE as well as severity, relationship, action and outcome. The Medical Dictionary for Regulatory Activities (MedDRA) version 20.1 was used and the Common Terminology Criteria for Adverse Events (CTCAE) grading.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
24 weeks of treatment
Results posted on
2021-12-07
Participant Flow
Study enrollment occurred between December 16, 2016, and August 17, 2017, at 6 sites in the US and Canada.
A total of 17 patients were screened at 6 sites in the United States and Canada. Of the 17 patients screened, 1 failed to meet an inclusion criterion.
Participant milestones
| Measure |
Placebo (Period 1)
4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4
|
NS-065/NCNP-01 40mg/kg (Period 1)
4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4
|
NS-065/NCNP-01 80mg/kg (Period 1)
4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2) Week 5 - Week 24
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2) Week 5 - Week 24
|
|---|---|---|---|---|---|
|
4-week Double-blinded Placebo-controlled
STARTED
|
5
|
6
|
5
|
0
|
0
|
|
4-week Double-blinded Placebo-controlled
COMPLETED
|
5
|
6
|
5
|
0
|
0
|
|
4-week Double-blinded Placebo-controlled
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
20-week Open Label Treatment Period
STARTED
|
0
|
0
|
0
|
8
|
8
|
|
20-week Open Label Treatment Period
COMPLETED
|
0
|
0
|
0
|
8
|
8
|
|
20-week Open Label Treatment Period
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Dose Finding 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
Patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 40mg/kg once a week for 24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
Patients with confirmed DMD with genetic deletions amenable to exon 53 skipping will be administered an intravenous infusion of NS-065/NCNP-01 80mg/kg once a week for 24 weeks
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
7.5 years
STANDARD_DEVIATION 1.8 • n=93 Participants
|
7.2 years
STANDARD_DEVIATION 2.0 • n=4 Participants
|
7.4 years
STANDARD_DEVIATION 1.8 • n=27 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
16 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
14 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=93 Participants
|
7 Participants
n=4 Participants
|
15 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Weight
|
23.7 kg
STANDARD_DEVIATION 4.7 • n=93 Participants
|
22.3 kg
STANDARD_DEVIATION 6.2 • n=4 Participants
|
23.0 kg
STANDARD_DEVIATION 5.3 • n=27 Participants
|
|
Height
|
114.6 cm
STANDARD_DEVIATION 6.5 • n=93 Participants
|
112.2 cm
STANDARD_DEVIATION 10.0 • n=4 Participants
|
113.4 cm
STANDARD_DEVIATION 8.2 • n=27 Participants
|
|
Body Mass Index(BMI)
|
17.9 kg/m^2
STANDARD_DEVIATION 2.3 • n=93 Participants
|
17.4 kg/m^2
STANDARD_DEVIATION 2.0 • n=4 Participants
|
17.7 kg/m^2
STANDARD_DEVIATION 2.1 • n=27 Participants
|
|
Time to run/walk 10m velocity
|
1.67 m/s
STANDARD_DEVIATION 0.39 • n=93 Participants
|
1.88 m/s
STANDARD_DEVIATION 0.36 • n=4 Participants
|
1.77 m/s
STANDARD_DEVIATION 0.37 • n=27 Participants
|
|
Time to run/walk 10m
|
6.30 second
STANDARD_DEVIATION 1.59 • n=93 Participants
|
5.55 second
STANDARD_DEVIATION 1.34 • n=4 Participants
|
5.93 second
STANDARD_DEVIATION 1.47 • n=27 Participants
|
|
Time to stand from supine velocity
|
0.26 rise/s
STANDARD_DEVIATION 0.06 • n=93 Participants
|
0.25 rise/s
STANDARD_DEVIATION 0.09 • n=4 Participants
|
0.25 rise/s
STANDARD_DEVIATION 0.07 • n=27 Participants
|
|
Time to stand from supine
|
4.17 second
STANDARD_DEVIATION 1.15 • n=93 Participants
|
4.76 second
STANDARD_DEVIATION 2.58 • n=4 Participants
|
4.44 second
STANDARD_DEVIATION 1.96 • n=27 Participants
|
|
6-Minute walk test
|
391.4 m
STANDARD_DEVIATION 33.3 • n=93 Participants
|
353.4 m
STANDARD_DEVIATION 106.3 • n=4 Participants
|
372.4 m
STANDARD_DEVIATION 78.6 • n=27 Participants
|
|
Time to climb 4 stairs velocity
|
0.27 m/s
STANDARD_DEVIATION 0.08 • n=93 Participants
|
0.32 m/s
STANDARD_DEVIATION 0.08 • n=4 Participants
|
0.30 m/s
STANDARD_DEVIATION 0.08 • n=27 Participants
|
|
Time to climb 4 stairs
|
3.90 second
STANDARD_DEVIATION 0.93 • n=93 Participants
|
3.33 second
STANDARD_DEVIATION 0.94 • n=4 Participants
|
3.61 second
STANDARD_DEVIATION 0.95 • n=27 Participants
|
|
NSAA score
|
24.8 scores on a scale
STANDARD_DEVIATION 5.9 • n=93 Participants
|
23.8 scores on a scale
STANDARD_DEVIATION 5.1 • n=4 Participants
|
24.3 scores on a scale
STANDARD_DEVIATION 5.4 • n=27 Participants
|
|
Dystrophin Production by Western Blot
Normalized to Myosin
|
0.3 % of normal control levels
STANDARD_DEVIATION 0.10 • n=93 Participants
|
0.6 % of normal control levels
STANDARD_DEVIATION 0.82 • n=4 Participants
|
0.4 % of normal control levels
STANDARD_DEVIATION 0.60 • n=27 Participants
|
|
Dystrophin Production by Western Blot
Normalized to Alpha-Actinin
|
0.2 % of normal control levels
STANDARD_DEVIATION 0.22 • n=93 Participants
|
0.4 % of normal control levels
STANDARD_DEVIATION 0.67 • n=4 Participants
|
0.3 % of normal control levels
STANDARD_DEVIATION 0.50 • n=27 Participants
|
|
Dystrophin Production by Mass Spectrometry
|
0.5 % of normal control levels
STANDARD_DEVIATION 0.15 • n=93 Participants
|
0.6 % of normal control levels
STANDARD_DEVIATION 0.19 • n=4 Participants
|
0.6 % of normal control levels
STANDARD_DEVIATION 0.17 • n=27 Participants
|
|
Dystrophin Production by Immunofluorescence
|
1.5 % of normal control levels
STANDARD_DEVIATION 0.98 • n=93 Participants
|
1.8 % of normal control levels
STANDARD_DEVIATION 2.36 • n=4 Participants
|
1.7 % of normal control levels
STANDARD_DEVIATION 1.75 • n=27 Participants
|
|
Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity
|
0.0 % of normal control levels
STANDARD_DEVIATION 0.00 • n=93 Participants
|
0.0 % of normal control levels
STANDARD_DEVIATION 0.00 • n=4 Participants
|
0.0 % of normal control levels
STANDARD_DEVIATION 0.00 • n=27 Participants
|
PRIMARY outcome
Timeframe: 24 weeks of treatmentPopulation: Within each category, patients were counted only once if they had \>1 event reported during the treatment period.
Treatment emergent adverse events (TEAEs) were summarized for Period 1 by comparing low dose to high dose to placebo and for Period 2 between the low dose cohort and the high dose cohort. TEAEs were summarized both at the patient level for number of TEAEs, highest severity, relationship, action and outcome and at the TEAE level (summarizing events) by organ system and preferred term TEAE as well as severity, relationship, action and outcome. The Medical Dictionary for Regulatory Activities (MedDRA) version 20.1 was used and the Common Terminology Criteria for Adverse Events (CTCAE) grading.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=5 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=6 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=5 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
n=8 Participants
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
n=8 Participants
20-week Open-label treatment period (Period 2)
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Incidence of Adverse Events as Assessed by CTCAE v4.0.
Participants with TEAE
|
3 Participants
|
4 Participants
|
4 Participants
|
5 Participants
|
7 Participants
|
15 Participants
|
|
Incidence of Adverse Events as Assessed by CTCAE v4.0.
Participants with drug-related TEAE
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Incidence of Adverse Events as Assessed by CTCAE v4.0.
Participants with CTCAE ≥ Grade 3
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Incidence of Adverse Events as Assessed by CTCAE v4.0.
Participants with TEAEs leading to discontinuation
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Incidence of Adverse Events as Assessed by CTCAE v4.0.
Participants with serious TEAE
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Incidence of Adverse Events as Assessed by CTCAE v4.0.
Death
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline and 24 weeks of treatmentPercentage normal dystrophin production in muscle biopsies from study participants at baseline and after 24 weeks treatment was measured by Western blot. To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity. Dystrophin protein levels were assessed using standard curves on each gel (range from 0-25% of normal levels) generated by mixing 5 normal control samples with one DMD sample.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Dystrophin Production by Western Blot
Normalized to Myosin
|
5.7 % of normal control levels
Standard Deviation 2.37
|
5.9 % of normal control levels
Standard Deviation 4.50
|
5.8 % of normal control levels
Standard Deviation 3.47
|
—
|
—
|
—
|
|
Dystrophin Production by Western Blot
Normalized to Alpha-Actinin
|
5.4 % of normal control levels
Standard Deviation 2.79
|
3.7 % of normal control levels
Standard Deviation 2.37
|
4.5 % of normal control levels
Standard Deviation 2.64
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentThe alteration of mRNA splicing was measured by RT-PCR of dystrophin mRNA transcripts from a patient muscle biopsy at baseline and after 24 weeks treatment. To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity. For RT-PCR, bands corresponding to specific versions of the spliced dystrophin mRNA were visualized by gel electrophoresis, and the amounts of different mRNA isoforms were compared.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity
|
17.4 % of normal control levels
Standard Deviation 7.17
|
43.9 % of normal control levels
Standard Deviation 16.68
|
30.6 % of normal control levels
Standard Deviation 18.45
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentThe production of dystrophin protein was measured by stable isotope mass spectrometry methods from a patient muscle biopsy at baseline and after 24 weeks treatment. To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity. Dystrophin peptides were identified and quantified using reversed-phase nanoflow high-performance liquid chromatography with high resolution Mass Spectrometry.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Dystrophin Production by Mass Spectrometry
|
2.1 % of normal control levels
Standard Deviation 1.09
|
4.2 % of normal control levels
Standard Deviation 3.73
|
3.1 % of normal control levels
Standard Deviation 2.88
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentThe production of dystrophin protein was measured by immunofluorescence staining methods from a patient muscle biopsy at baseline and after 24 weeks treatment. To analyze dystrophin induction, biopsies were taken from a biceps muscle at baseline and the other biceps muscle after 24 weeks of treatment. Muscle samples were snap frozen and delivered to a central laboratory. All laboratory researchers remained blinded to sample identity. Immunofluorescence staining for dystrophin was performed on serial muscle biopsy sections in duplicate.
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Dystrophin Production by Immunofluorescence
|
14.2 % dystrophin-positive fibers
Standard Deviation 7.77
|
34.8 % dystrophin-positive fibers
Standard Deviation 20.42
|
24.5 % dystrophin-positive fibers
Standard Deviation 18.32
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: quantitative muscle testing (QMT).
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Handgrip
|
-0.2029 lb
Standard Deviation 2.98334
|
-0.8513 lb
Standard Deviation 1.85797
|
-0.4808 lb
Standard Deviation 2.49621
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Dominant Side Handgrip
|
-0.4173 lb
Standard Deviation 2.46596
|
-1.0867 lb
Standard Deviation 1.75915
|
-0.7041 lb
Standard Deviation 2.14074
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Non-Dominant Side Handgrip
|
-0.2280 lb
Standard Deviation 3.39211
|
-0.1780 lb
Standard Deviation 2.27428
|
-0.2066 lb
Standard Deviation 2.86108
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Elbow Flexors (biceps)
|
0.3190 lb
Standard Deviation 1.99880
|
-0.7110 lb
Standard Deviation 1.61528
|
-0.1224 lb
Standard Deviation 1.85326
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Dominant Side Elbow Flexors (biceps)
|
0.5111 lb
Standard Deviation 2.57829
|
-0.4468 lb
Standard Deviation 1.49138
|
0.1006 lb
Standard Deviation 2.16263
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Non-Dominant Side Elbow Flexors (biceps)
|
0.2067 lb
Standard Deviation 2.71502
|
-0.3732 lb
Standard Deviation 2.50228
|
-0.0163 lb
Standard Deviation 2.54427
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Elbow Extensors (triceps)
|
0.6777 lb
Standard Deviation 2.65006
|
0.6895 lb
Standard Deviation 1.00990
|
0.6828 lb
Standard Deviation 2.04299
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Dominant Side Elbow Extensors (triceps)
|
0.7046 lb
Standard Deviation 2.78271
|
0.6502 lb
Standard Deviation 0.81291
|
0.6813 lb
Standard Deviation 2.10345
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Non-Dominant Side Elbow Extensors (triceps)
|
0.0001 lb
Standard Deviation 2.34657
|
0.5168 lb
Standard Deviation 1.44498
|
0.2216 lb
Standard Deviation 1.95920
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Knee Flexors (hamstrings)
|
-1.2209 lb
Standard Deviation 3.37401
|
-0.0283 lb
Standard Deviation 3.43240
|
-0.7098 lb
Standard Deviation 3.32207
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Dominant Side Knee Flexors (hamstrings)
|
-1.8985 lb
Standard Deviation 4.01798
|
-0.7175 lb
Standard Deviation 3.64572
|
-1.3924 lb
Standard Deviation 3.76469
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Non-Dominant Side Knee Flexors (hamstrings)
|
-1.7373 lb
Standard Deviation 3.10626
|
0.6377 lb
Standard Deviation 2.03566
|
-0.7194 lb
Standard Deviation 2.87696
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Knee Extensors (quadriceps)
|
-2.2261 lb
Standard Deviation 4.82146
|
1.5798 lb
Standard Deviation 3.33316
|
-0.5950 lb
Standard Deviation 4.53990
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Dominant Side Knee Extensors (quadriceps)
|
-2.1524 lb
Standard Deviation 4.58595
|
2.0867 lb
Standard Deviation 2.96796
|
-0.3356 lb
Standard Deviation 4.41039
|
—
|
—
|
—
|
|
Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT).
Non-Dominant Side Knee Extensors (quadriceps)
|
-0.5994 lb
Standard Deviation 4.38150
|
1.3308 lb
Standard Deviation 4.44474
|
0.2279 lb
Standard Deviation 4.34950
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: 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 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Distance Traveled in the Six-Minute Walk Test (6MWT).
|
15.6 m
Standard Deviation 26.40
|
44.0 m
Standard Deviation 41.98
|
28.9 m
Standard Deviation 36.31
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: 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 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB).
|
-0.34 second
Standard Deviation 1.140
|
0.00 second
Standard Deviation 0.600
|
-0.17 second
Standard Deviation 0.897
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). 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 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity
|
0.07 m/s
Standard Deviation 0.105
|
-0.00 m/s
Standard Deviation 0.054
|
0.32 m/s
Standard Deviation 0.088
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW).
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Time to Run/Walk 10 Meters (TTRW).
|
-0.65 second
Standard Deviation 1.225
|
-0.66 second
Standard Deviation 0.921
|
-0.66 second
Standard Deviation 1.047
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). 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 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Time to Run/Walk 10 Meters (TTRW) Velocity.
|
0.21 m/s
Standard Deviation 0.291
|
0.24 m/s
Standard Deviation 0.222
|
0.23 m/s
Standard Deviation 0.251
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: 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 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Time to Stand (TTSTAND)
|
0.05 second
Standard Deviation 1.446
|
-0.44 second
Standard Deviation 0.750
|
-0.19 second
Standard Deviation 1.141
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentA secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Stand (TTSTAND).The results were converted into velocity (rise/time).
Outcome measures
| Measure |
NS-065/NCNP-01 40mg/kg
n=8 Participants
NS-065/NCNP-01 40mg/kg dose once a week for 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Time to Stand (TTSTAND) Velocity
|
0.02 rise/time
Standard Deviation 0.093
|
0.02 rise/time
Standard Deviation 0.060
|
0.02 rise/time
Standard Deviation 0.075
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 weeks of treatmentThe 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 20-24 weeks
|
NS-065/NCNP-01 80mg/kg
n=8 Participants
NS-065/NCNP-01 80mg/kg dose once a week for 20-24 weeks
|
Total NS-065/NCNP-01 Group
n=16 Participants
Of the 16 patients randomized, all patients received treatment with NS-065/NCNP-01 and all patients completed treatment with NS-065/NCNP-01.
|
NS-065/NCNP-01 40mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
NS-065/NCNP-01 80mg/kg (Period 2)
20-week Open-label treatment period (Period 2)
|
Total
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Change From Baseline in North Star Ambulatory Assessment (NSAA) Score.
|
0.5 score on a scale
Standard Deviation 3.07
|
1.1 score on a scale
Standard Deviation 2.80
|
0.8 score on a scale
Standard Deviation 2.86
|
—
|
—
|
—
|
Adverse Events
Placebo (Period 1)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
NS-065/NCNP-01 40mg/kg (Period 1)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
NS-065/NCNP-01 80mg/kg (Period 1)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
NS-065/NCNP-01 40mg/kg (Period 2)
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
NS-065/NCNP-01 80mg/kg (Period 2)
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Total
Serious events: 0 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Placebo (Period 1)
n=5 participants at risk
4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4
Two patients in each of the dose groups will be administered placebo as an intravenous infusion once a week for 4 weeks followed by 20 weeks of open label treatment
|
NS-065/NCNP-01 40mg/kg (Period 1)
n=6 participants at risk
4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4
|
NS-065/NCNP-01 80mg/kg (Period 1)
n=5 participants at risk
4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4
|
NS-065/NCNP-01 40mg/kg (Period 2)
n=8 participants at risk
20-week Open-label treatment period (Period 2) Week 5 - Week 24
|
NS-065/NCNP-01 80mg/kg (Period 2)
n=8 participants at risk
20-week Open-label treatment period (Period 2) Week 5 - Week 24
|
Total
n=16 participants at risk
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
31.2%
5/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
16.7%
1/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Influenza
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Molluscum contagiosum
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Nasopharyngitis
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
50.0%
4/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
4/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Facial pain
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Infusion site discomfort
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Injection site bruising
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Injection site pain
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
16.7%
1/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Injection site reaction
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Pyrexia
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
General disorders
Thirst
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Arthropod sting
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Fall
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Injury, poisoning and procedural complications
Joint injury
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
25.0%
2/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
2/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Psychiatric disorders
Abnormal behaviour
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
16.7%
1/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Psychiatric disorders
Attention deficit/hyperactivity disorder
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
16.7%
1/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
16.7%
1/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Ear and labyrinth disorders
Otorrhoea
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Immune system disorders
Drug hypersensitivity
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
16.7%
1/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Investigations
Blood creatinine increased
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Investigations
Blood potassium increased
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Investigations
Blood urea increased
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Nervous system disorders
Headache
|
20.0%
1/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
|
Vascular disorders
Peripheral artery occlusion
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/6 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/5 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
0.00%
0/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
12.5%
1/8 • 24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
|
6.2%
1/16 • 24 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