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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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This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.
This is a Phase II, multicenter, open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 192 weeks to boys with DMD who complete Study NS-065/NCNP-01-201. This study will evaluate the safety, tolerability, and clinical efficacy of NS-065/NCNP-01 at dose levels of up to 80 mg/kg/week administered by weekly IV infusion over an additional treatment period of 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier.
Patients who complete the Phase II Dose-finding Study NS-065/NCNP-01-201 are eligible to enroll.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NS-065/NCNP-01 40mg/kg | Experimental | Patients receiving 40mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier. |
|
| NS-065/NCNP-01 80mg/kg | Experimental | Patients receiving 80mg/kg in the NS-065-NCNP-201 study will continue their current dose for an additional 192 weeks or until enrollment in a separate long-term follow up program of NS-065/NCNP-01, whichever is earlier. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NS-065/NCNP-01 | Drug | Received during weekly intravenous infusions |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | For adverse events (AEs) starting in study 201 (NCT02740972) which are not resolved at the time of enrollment into this study 202, any change in outcome or relatedness were reported in study 201. For AEs starting in study 201 which increase in severity or becomes serious after enrollment in this study 202, a new AE was reported in this study. Treatment-emergent AEs (TEAEs) were summarized by dose level. Coding was done by system organ class and preferred term (using the Medical Dictionary for Regulatory Activities (MedDRA)). Level of severity was assessed using the CTCAE grading system. | Up to 192 weeks of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW) | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls |
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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 | ||
| Lurie Children's Hospital |
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Participants who completed study: NS-065/NCNP-01-201 (NCT02740972) were eligible to enroll in this long-term extension study.
Participants were enrolled in the study at 6 sites in the United States and Canada from July 6, 2017 and August 25, 2018.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 |
| FG001 | 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 |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Full Analysis Set (FAS) population included all participants who received at least one dose of NS-065/NCNP-01 Injection 250 mg infusion in both the Study 201 and extension study (NS-065/NCNP-01-202)
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| ID | Title | Description |
|---|---|---|
| BG000 | NS-065/NCNP-01 40mg/kg | NS-065/NCNP-01 40mg/kg dose once a week for up to 192 weeks. Baseline of Study NS-065/NCNP-01-202: Defined as the measurement taken on Study NS-065/NCNP-01-201 Week 25. |
| 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 | Change From Baseline in Time to Stand (TTSTAND) Versus Matched Historical Controls | A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) | 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. | Posted | Least Squares Mean | Standard Error | seconds | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
Up to 192 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 | NS-065/NCNP-01 40mg/kg | NS-065/NCNP-01 40mg/kg dose once a week for up to 192 weeks. Baseline of Study NS-065/NCNP-01-202: Defined as the measurement taken on Study NS-065/NCNP-01-201 Week 25. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Affairs | NS Pharma, Inc. | +1(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 | Jul 21, 2020 | Sep 23, 2022 | 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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Low Dose cohort of 40 mg/kg and High Dose cohort of 80 mg/kg
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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). |
| Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB) | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB) Velocity. The results were converted into velocity (meter/time). | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): North Star Ambulatory Assessment (NSAA) score The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function. | Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202 |
| Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Six-Minute Walk Test (6MWT) | Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202 |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Handgrip For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Flexors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Extensors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee Flexors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee Extensors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Children's Hospital of Richmond at VCU | Richmond | Virginia | 23298 | United States |
| Alberta Children's Hospital | Calgary | Alberta | Canada |
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.
| 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 |
|
| NS-065/NCNP-01 80mg/kg |
NS-065/NCNP-01 80mg/kg dose once a week for up to 192 weeks. Baseline of Study NS-065/NCNP-01-202: Defined as the measurement taken on Study NS-065/NCNP-01-201 Week 25 Visit. |
| OG002 | Total NS-065/NCNP-01 Group | All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population. |
|
|
| Primary | Change From Baseline in Time to Stand (TTSTAND) Velocity Versus Matched Historical Controls | A primary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Stand (TTSTAND) Velocity | 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. | Posted | Least Squares Mean | Standard Error | rise/s | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Primary | Number of Participants With Treatment Related Adverse Events as Assessed by CTCAE v4.0. | For adverse events (AEs) starting in study 201 (NCT02740972) which are not resolved at the time of enrollment into this study 202, any change in outcome or relatedness were reported in study 201. For AEs starting in study 201 which increase in severity or becomes serious after enrollment in this study 202, a new AE was reported in this study. Treatment-emergent AEs (TEAEs) were summarized by dose level. Coding was done by system organ class and preferred term (using the Medical Dictionary for Regulatory Activities (MedDRA)). Level of severity was assessed using the CTCAE grading system. | 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. | Posted | Count of Participants | Participants | Up to 192 weeks of treatment |
|
|
|
| Secondary | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW) | 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. | Posted | Least Squares Mean | Standard Error | seconds | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Time to Run/Walk 10 Meters Test (TTRW) Velocity Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Run/Walk 10 meters test (TTRW) Velocity. The results were converted into velocity (meter/time). | 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. | Posted | Least Squares Mean | Standard Error | m/s | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB) | 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. | Posted | Least Squares Mean | Standard Error | seconds | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Time to Climb 4 stairs (TTCLIMB) Velocity. The results were converted into velocity (meter/time). | 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. | Posted | Least Squares Mean | Standard Error | m/s | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in North Star Ambulatory Assessment (NSAA) Score Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): North Star Ambulatory Assessment (NSAA) score The NSAA is a functional scale devised for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). It assesses abilities necessary to remain ambulant that have been found to progressively deteriorate in untreated DMD patients, as well as in other muscular dystrophies such as Becker Muscular Dystrophy. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function. | 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. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Six-Minute Walk Test (6MWT) Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Six-Minute Walk Test (6MWT) | 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. | Posted | Least Squares Mean | Standard Error | m | Baseline 201, Weeks 37, 49, 73, 109, 157 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Quantitative Muscle Testing (QMT) for Handgrip Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Handgrip For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | 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. | Posted | Least Squares Mean | Standard Error | lb | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Flexors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Flexors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | 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. | Posted | Least Squares Mean | Standard Error | lb | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Quantitative Muscle Testing (QMT) for Elbow Extensors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Elbow Extensors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | 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. | Posted | Least Squares Mean | Standard Error | lb | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Flexors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee Flexors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | 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. | Posted | Least Squares Mean | Standard Error | lb | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| Secondary | Change From Baseline in Quantitative Muscle Testing (QMT) for Knee Extensors Versus Matched Historical Controls | A secondary efficacy endpoint was compared to Baseline of Study 201 (NCT02740972): Quantitative Muscle Testing (QMT) for Knee Extensors For QMT tests, the higher of each of the bilateral scores recorded for each muscle group at each visit were analyzed. QMT tests were analyzed by dominant/non-dominant side. QMT is a well-established method for measuring muscle weakness in neuromuscular disease. Patients will be placed on an examination table with a back-support system to eliminate the need for manual back stabilization. Following a single practice administration, each patient will complete a scored QMT evaluation (perform 2 tests; with the higher of the 2 values used for data analysis). QMT will be performed by recording force in pounds through a direct computer interface with a strain gauge. | 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. | Posted | Least Squares Mean | Standard Error | lb | Baseline 201, Weeks 37, 49, 73, 109, 157, 205 in Study 202 |
|
|
|
| 0 |
| 8 |
| 1 |
| 8 |
| 8 |
| 8 |
| EG001 | NS-065/NCNP-01 80mg/kg | NS-065/NCNP-01 80mg/kg dose once a week for up to 192 weeks. Baseline of Study NS-065/NCNP-01-202: Defined as the measurement taken on Study NS-065/NCNP-01-201 Week 25. | 0 | 8 | 2 | 8 | 8 | 8 |
| EG002 | Total NS-065/NCNP-01 Group | All 16 patients who received treatment with NS-065/NCNP-01 were included in the Full Analysis Set population. | 0 | 16 | 3 | 16 | 16 | 16 |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Body tinea | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Pharyngitis streptococcal | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Respiratory tract infection viral | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Viral infection | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Bacterial infection | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Coxsackie viral infection | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Hordeolum | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Onychomycosis | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Otitis externa | Infections and infestations | MedDRA Version 21.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Laceration | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Limb injury | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Thermal burn | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Animal bite | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Foot fracture | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Foreign body in respiratory tract | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Incision site swelling | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
|
| Incision site vesicles | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Ligament sprain | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Limb fracture | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Skin injury | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Spinal column injury | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Sunburn | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Traumatic tooth displacement | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Vascular access complication | Injury, poisoning and procedural complications | MedDRA Version 21.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Allergic sinusitis | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Respiratory symptom | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Haematochezia | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Tooth development disorder | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Sinus headache | Nervous system disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Catheter site erythema | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Catheter site swelling | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Infusion site pain | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Injection site extravasation | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Pain | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Peripheral swelling | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Tenderness | General disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Joint hyperextension | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Limb discomfort | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Spinal deformity | Musculoskeletal and connective tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Alopecia | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Onycholysis | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Rash macular | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Attention deficit/hyperactivity disorder | Psychiatric disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Flat affect | Psychiatric disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Body height decreased | Investigations | MedDRA Version 21.0 | Systematic Assessment |
|
| Vitamin D decreased | Investigations | MedDRA Version 21.0 | Systematic Assessment |
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| Weight increased | Investigations | MedDRA Version 21.0 | Systematic Assessment |
|
| Hypercalciuria | Renal and urinary disorders | MedDRA Version 21.0 | Systematic Assessment |
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| Pyelocaliectasis | Renal and urinary disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Sinus arrhythmia | Cardiac disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Ankyloglossia congenital | Congenital, familial and genetic disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Cushingoid | Endocrine disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Cataract | Eye disorders | MedDRA Version 21.0 | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA Version 21.0 | 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 |
| Week 49 |
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| Week 73 |
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| Week 109 |
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| Week 157 |
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| Week 205 |
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| Participants with CTCAE ≥ Grade 3 |
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| Participants with TEAEs leading to discontinuation |
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| Participants with serious TEAE |
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| Death |
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| Week 49 |
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| Week 73 |
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| Week 109 |
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| Week 157 |
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| Week 205 |
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| Week 49 |
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| Week 73 |
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| Week 109 |
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| Week 157 |
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| Week 205 |
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| Week 49 |
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| Week 73 |
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| Week 109 |
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| Week 157 |
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| Week 205 |
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| Week 49 |
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| Week 73 |
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| Week 109 |
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| Week 157 |
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| Week 205 |
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| Week 49 |
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| Week 73 |
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| Week 109 |
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| Week 157 |
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| Week 49 |
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| Week 73 |
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| Week 109 |
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| Week 157 |
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| Handgrip, Week 49 |
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| Handgrip, Week 73 |
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| Handgrip, Week 109 |
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| Handgrip, Week 157 |
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| Handgrip, Week 205 |
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| Dominant Side Handgrip, Week 37 |
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| Dominant Side Handgrip, Week 49 |
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| Dominant Side Handgrip, Week 73 |
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| Dominant Side Handgrip, Week 109 |
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| Dominant Side Handgrip, Week 157 |
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| Dominant Side Handgrip, Week 205 |
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| Non-Dominant Side Handgrip, Week 37 |
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| Non-Dominant Side Handgrip, Week 49 |
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| Non-Dominant Side Handgrip, Week 73 |
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| Non-Dominant Side Handgrip, Week 109 |
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| Non-Dominant Side Handgrip, Week 157 |
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| Non-Dominant Side Handgrip, Week 205 |
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| Elbow Flexors, Week 49 |
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| Elbow Flexors, Week 73 |
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| Elbow Flexors, Week 109 |
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| Elbow Flexors, Week 157 |
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| Elbow Flexors, Week 205 |
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| Dominant Side Elbow Flexors, Week 37 |
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| Dominant Side Elbow Flexors, Week 49 |
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| Dominant Side Elbow Flexors, Week 73 |
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| Dominant Side Elbow Flexors, Week 109 |
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| Dominant Side Elbow Flexors, Week 157 |
|
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| Dominant Side Elbow Flexors, Week 205 |
|
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| Non-Dominant Side Elbow Flexors, Week 37 |
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| Non-Dominant Side Elbow Flexors, Week 49 |
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| Non-Dominant Side Elbow Flexors, Week 73 |
|
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| Non-Dominant Side Elbow Flexors, Week 109 |
|
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| Non-Dominant Side Elbow Flexors, Week 157 |
|
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| Non-Dominant Side Elbow Flexors, Week 205 |
|
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| Elbow Extensors, Week 49 |
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| Elbow Extensors, Week 73 |
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| Elbow Extensors, Week 109 |
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| Elbow Extensors, Week 157 |
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| Elbow Extensors, Week 205 |
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| Dominant Side Elbow Extensors, Week 37 |
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| Dominant Side Elbow Extensors, Week 49 |
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| Dominant Side Elbow Extensors, Week 73 |
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| Dominant Side Elbow Extensors, Week 109 |
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| Dominant Side Elbow Extensors, Week 157 |
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| Dominant Side Elbow Extensors, Week 205 |
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| Non-Dominant Side Elbow Extensors, Week 37 |
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| Non-Dominant Side Elbow Extensors, Week 49 |
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| Non-Dominant Side Elbow Extensors, Week 73 |
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| Non-Dominant Side Elbow Extensors, Week 109 |
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| Non-Dominant Side Elbow Extensors, Week 157 |
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| Non-Dominant Side Elbow Extensors, Week 205 |
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| Knee Flexors, Week 49 |
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| Knee Flexors, Week 73 |
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| Knee Flexors, Week 109 |
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| Knee Flexors, Week 157 |
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| Knee Flexors, Week 205 |
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| Dominant Side Knee Flexors, Week 37 |
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| Dominant Side Knee Flexors, Week 49 |
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| Dominant Side Knee Flexors, Week 73 |
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| Dominant Side Knee Flexors, Week 109 |
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| Dominant Side Knee Flexors, Week 157 |
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| Dominant Side Knee Flexors, Week 205 |
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| Non-Dominant Side Knee Flexors, Week 37 |
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| Non-Dominant Side Knee Flexors, Week 49 |
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| Non-Dominant Side Knee Flexors, Week 73 |
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| Non-Dominant Side Knee Flexors, Week 109 |
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| Non-Dominant Side Knee Flexors, Week 157 |
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| Non-Dominant Side Knee Flexors, Week 205 |
|
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| Knee Extensors, Week 49 |
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| Knee Extensors, Week 73 |
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| Knee Extensors, Week 109 |
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| Knee Extensors, Week 157 |
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| Knee Extensors, Week 205 |
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| Dominant Side Knee Extensors, Week 37 |
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| Dominant Side Knee Extensors, Week 49 |
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| Dominant Side Knee Extensors, Week 73 |
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| Dominant Side Knee Extensors, Week 109 |
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| Dominant Side Knee Extensors, Week 157 |
|
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| Dominant Side Knee Extensors, Week 205 |
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| Non-Dominant Side Knee Extensors, Week 37 |
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| Non-Dominant Side Knee Extensors, Week 49 |
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| Non-Dominant Side Knee Extensors, Week 73 |
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| Non-Dominant Side Knee Extensors, Week 109 |
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| Non-Dominant Side Knee Extensors, Week 157 |
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| Non-Dominant Side Knee Extensors, Week 205 |
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