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| Name | Class |
|---|---|
| Nippon Shinyaku Co., Ltd. | INDUSTRY |
| Cooperative International Neuromuscular Research Group | NETWORK |
| Therapeutic Research in Neuromuscular Disorders Solutions | INDUSTRY |
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The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.
This is a Phase II, multiple center, 2-period, randomized, placebo-controlled, dose finding study of NS-065/NCNP-01 administered by infusion once weekly for 24 weeks to ambulant boys ages 4-<10 years with DMD. Two dose level cohorts will be enrolled. Period 1 of this study will be conducted in a double-blind fashion. Randomized patients will receive weekly IV infusions of NS-065/NCNP-01 or placebo for the first 4 weeks of their participation (Period 1) and NS-065/NCNP-01 by IV infusion for weeks 5-24 (20 weeks of active treatment - Period 2). Analysis of safety data from Period 1 of the 40mg/kg dose cohort will be completed prior to enrolling patients in the 80mg/kg dose cohort.
Patients completing the 24-week study will be eligible for an open-label extension study.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six-minute walk test (6MWT), time to stand (TTSTAND), time to run/walk 10 meters (TTRW), time to climb 4 stairs (TTCLIMB) and quantitative muscle testing (QMT). All patients will undergo a muscle biopsy of the bicep at baseline and a second muscle biopsy at Week 24.
Safety will be assessed through the collection of adverse events (AEs), blood and urine laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.
Serial blood samples will be taken at four of the study visits to assess the pharmacokinetics of the study drug.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Experimental | Six 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 dose once a week for 24 weeks |
|
| NS-065/NCNP-01 80mg/kg | Experimental | Six 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 |
|
| Placebo | Placebo Comparator | 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NS-065/NCNP-01 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events as Assessed by CTCAE v4.0. | 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. | 24 weeks of treatment |
| Dystrophin Production by Western Blot | Percentage 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. | Baseline and 24 weeks of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity | The 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Paula R. Clemens, MD | University of Pittsburgh | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UC Davis | Sacramento | California | 95817 | United States | ||
| University of Florida Health |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32453377 | Derived | Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. JAMA Neurol. 2020 Aug 1;77(8):982-991. doi: 10.1001/jamaneurol.2020.1264. |
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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.
Study enrollment occurred between December 16, 2016, and August 17, 2017, at 6 sites in the US and Canada.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo (Period 1) | 4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4 |
| FG001 | NS-065/NCNP-01 40mg/kg (Period 1) | 4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4 |
| FG002 | NS-065/NCNP-01 80mg/kg (Period 1) | 4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4 |
| FG003 | NS-065/NCNP-01 40mg/kg (Period 2) | 20-week Open-label treatment period (Period 2) Week 5 - Week 24 |
| FG004 | NS-065/NCNP-01 80mg/kg (Period 2) | 20-week Open-label treatment period (Period 2) Week 5 - Week 24 |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 4-week Double-blinded Placebo-controlled |
| |||||||||||||
| 20-week Open Label Treatment Period |
|
A total of 16 participants were enrolled and 8 participants participated in each of the 2 dose cohorts.
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| ID | Title | Description |
|---|---|---|
| BG000 | NS-065/NCNP-01 40mg/kg | 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 |
| BG001 | NS-065/NCNP-01 80mg/kg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Incidence of Adverse Events as Assessed by CTCAE v4.0. | 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. | Within each category, patients were counted only once if they had >1 event reported during the treatment period. | Posted | Count of Participants | Participants | 24 weeks of treatment |
|
24 weeks
Incidence of Adverse Events as assessed by CTCAE v4.0
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo (Period 1) | 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 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Affairs | NS Pharma, Inc. | (201) 986-3860 | trialinfo@nspharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Nov 28, 2017 | Apr 23, 2021 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| Drug |
|
| Baseline and 24 weeks of treatment |
| Dystrophin Production by Mass Spectrometry | The 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. | Baseline and 24 weeks of treatment |
| Dystrophin Production by Immunofluorescence | The 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. | Baseline and 24 weeks of treatment |
| Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: quantitative muscle testing (QMT). | Baseline and 24 weeks of treatment |
| Change From Baseline in Distance Traveled in the Six-Minute Walk Test (6MWT). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Six-Minute Walk Test (6MWT). | Baseline and 24 weeks of treatment |
| Change From Baseline in Time to Climb 4 Stairs (TTCLIMB). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Climb 4 Stairs (TTCLIMB). | Baseline and 24 weeks of treatment |
| Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). The results were converted into velocity (meter/time). | Baseline and 24 weeks of treatment |
| Change From Baseline in Time to Run/Walk 10 Meters (TTRW). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). | Baseline and 24 weeks of treatment |
| Change From Baseline in Time to Run/Walk 10 Meters (TTRW) Velocity. | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). The results were converted into velocity (meter/time). | Baseline and 24 weeks of treatment |
| Change From Baseline in Time to Stand (TTSTAND) | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Stand (TTSTAND) | Baseline and 24 weeks of treatment |
| Change From Baseline in Time to Stand (TTSTAND) Velocity | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Stand (TTSTAND).The results were converted into velocity (rise/time). | Baseline and 24 weeks of treatment |
| Change From Baseline in 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. | Baseline and 24 weeks of treatment |
| Gainesville |
| Florida |
| United States |
| Lurie Children's Hospital | Chicago | Illinois | United States |
| Washington University | St Louis | Missouri | United States |
| Duke University Medical Center | Durham | North Carolina | United States |
| Children's Hospital of Richmond at VCU | Richmond | Virginia | United States |
| Alberta Children's Hospital | Calgary | Alberta | Canada |
| COMPLETED |
|
| NOT COMPLETED |
|
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
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Weight | Mean | Standard Deviation | kg |
|
| Height | Mean | Standard Deviation | cm |
|
| Body Mass Index(BMI) | Mean | Standard Deviation | kg/m^2 |
|
| Time to run/walk 10m velocity | Mean | Standard Deviation | m/s |
|
| Time to run/walk 10m | Mean | Standard Deviation | second |
|
| Time to stand from supine velocity | Mean | Standard Deviation | rise/s |
|
| Time to stand from supine | Mean | Standard Deviation | second |
|
| 6-Minute walk test | Mean | Standard Deviation | m |
|
| Time to climb 4 stairs velocity | Mean | Standard Deviation | m/s |
|
| Time to climb 4 stairs | Mean | Standard Deviation | second |
|
| 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. | Mean | Standard Deviation | scores on a scale |
|
| Dystrophin Production by Western Blot | Mean | Standard Deviation | % of normal control levels |
|
| Dystrophin Production by Mass Spectrometry | Mean | Standard Deviation | % of normal control levels |
|
| Dystrophin Production by Immunofluorescence | Mean | Standard Deviation | % of normal control levels |
|
| Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity | Mean | Standard Deviation | % of normal control levels |
|
| OG001 | NS-065/NCNP-01 40mg/kg (Period 1) | 4-week Double-blinded placebo-controlled period (Period 1) |
| OG002 | NS-065/NCNP-01 80mg/kg (Period 1) | 4-week Double-blinded placebo-controlled period (Period 1) |
| OG003 | NS-065/NCNP-01 40mg/kg (Period 2) | 20-week Open-label treatment period (Period 2) |
| OG004 | NS-065/NCNP-01 80mg/kg (Period 2) | 20-week Open-label treatment period (Period 2) |
| OG005 | Total | Total of all reporting groups |
|
|
| Primary | Dystrophin Production by Western Blot | Percentage 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. | Posted | Mean | Standard Deviation | % of normal control levels | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity | The 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. | Posted | Mean | Standard Deviation | % of normal control levels | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Dystrophin Production by Mass Spectrometry | The 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. | Posted | Mean | Standard Deviation | % of normal control levels | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Dystrophin Production by Immunofluorescence | The 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. | Posted | Mean | Standard Deviation | % dystrophin-positive fibers | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: quantitative muscle testing (QMT). | Posted | Mean | Standard Deviation | lb | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Distance Traveled in the Six-Minute Walk Test (6MWT). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Six-Minute Walk Test (6MWT). | Posted | Mean | Standard Deviation | m | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Climb 4 Stairs (TTCLIMB). | Posted | Mean | Standard Deviation | second | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). The results were converted into velocity (meter/time). | Posted | Mean | Standard Deviation | m/s | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Time to Run/Walk 10 Meters (TTRW). | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). | Posted | Mean | Standard Deviation | second | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Time to Run/Walk 10 Meters (TTRW) Velocity. | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Run/Walk 10 meters (TTRW). The results were converted into velocity (meter/time). | Posted | Mean | Standard Deviation | m/s | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Time to Stand (TTSTAND) | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Stand (TTSTAND) | Posted | Mean | Standard Deviation | second | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Time to Stand (TTSTAND) Velocity | A secondary efficacy endpoint was compared to Pre-Infusion Visit: Time to Stand (TTSTAND).The results were converted into velocity (rise/time). | Posted | Mean | Standard Deviation | rise/time | Baseline and 24 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in 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. | Posted | Mean | Standard Deviation | score on a scale | Baseline and 24 weeks of treatment |
|
|
|
| 0 |
| 5 |
| 0 |
| 5 |
| 3 |
| 5 |
| EG001 | NS-065/NCNP-01 40mg/kg (Period 1) | 4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4 | 0 | 6 | 0 | 6 | 4 | 6 |
| EG002 | NS-065/NCNP-01 80mg/kg (Period 1) | 4-week Double-blinded placebo-controlled period (Period 1) Week 0 - Week 4 | 0 | 5 | 0 | 5 | 4 | 5 |
| EG003 | NS-065/NCNP-01 40mg/kg (Period 2) | 20-week Open-label treatment period (Period 2) Week 5 - Week 24 | 0 | 8 | 0 | 8 | 5 | 8 |
| EG004 | NS-065/NCNP-01 80mg/kg (Period 2) | 20-week Open-label treatment period (Period 2) Week 5 - Week 24 | 0 | 8 | 0 | 8 | 7 | 8 |
| EG005 | Total | Total of all reporting groups | 0 | 16 | 0 | 16 | 15 | 16 |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
|
| Molluscum contagiosum | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
|
| Pharyngitis streptococcal | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.1 | Non-systematic Assessment |
|
| Facial pain | General disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Infusion site discomfort | General disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Injection site bruising | General disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Injection site pain | General disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Injection site reaction | General disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Thirst | General disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
|
| Arthropod sting | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
|
| Joint injury | Injury, poisoning and procedural complications | MedDRA 20.1 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Abnormal behaviour | Psychiatric disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Attention deficit/hyperactivity disorder | Psychiatric disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Otorrhoea | Ear and labyrinth disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Drug hypersensitivity | Immune system disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Blood creatinine increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
|
| Blood potassium increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
|
| Blood urea increased | Investigations | MedDRA 20.1 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 20.1 | Non-systematic Assessment |
|
| Peripheral artery occlusion | Vascular disorders | MedDRA 20.1 | Non-systematic Assessment |
|
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.
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
|
|
| Non-Dominant Side Handgrip |
|
| Elbow Flexors (biceps) |
|
| Dominant Side Elbow Flexors (biceps) |
|
| Non-Dominant Side Elbow Flexors (biceps) |
|
| Elbow Extensors (triceps) |
|
| Dominant Side Elbow Extensors (triceps) |
|
| Non-Dominant Side Elbow Extensors (triceps) |
|
| Knee Flexors (hamstrings) |
|
| Dominant Side Knee Flexors (hamstrings) |
|
| Non-Dominant Side Knee Flexors (hamstrings) |
|
| Knee Extensors (quadriceps) |
|
| Dominant Side Knee Extensors (quadriceps) |
|
| Non-Dominant Side Knee Extensors (quadriceps) |
|