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The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Ambulatory Type 3 SMA |
|
| Cohort 2 | Experimental | Type 2 SMA / Non-Ambulatory Type 3 SMA |
|
| Cohort 3 | Experimental | Type 2 SMA |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRK-015 | Biological | SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 will be administered every 4 weeks by intravenous infusion. |
| Measure | Description | Time Frame |
|---|---|---|
| Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] | The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA. For the ambulatory Type 3 patients 5-21 years of age in Cohort 1 (N=23), the primary endpoint was the change from baseline in RHS total score at month 12. The RHS has a minimum achievable score of 0 and a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the lowest level and 2 denotes the highest level of ability/function. The remaining 3 items are scored 0 or 1, where 0 denotes an inability and 1 denotes an ability to achieve. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. | Baseline up to 12 months |
| Cohort 2 and Cohort 3: Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score at Day 364 (Visit 15) [Month 12] | Cohort 2: For the nonambulatory Type 2 and Type 3 patients 5-21 years of age in Cohort 2 (N=15), the primary efficacy endpoint was the change from baseline in HFMSE total score at month 12. Cohort 3: For the nonambulatory Type 2 patients ≥2 years of age in Cohort 3 (N=20), the primary efficacy endpoint was the change from baseline in HFMSE total score at month 12. The Hammersmith Functional Motor Scale Expanded (HFMSE) assesses the physical abilities of patients with Type 2 and Type 3 SMA comprises 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. | Baseline up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA; it has a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the lowest level and 2 denotes the highest level of ability/function. The remaining 3 items are scored 0 or 1, where 0 denotes an inability and 1 denotes an ability to achieve. |
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Inclusion Criteria:
Age 5 through 21 years old at the time of screening for Cohorts 1 and 2; Age ≥2 years old at the time of screening for Cohort 3.
Documented diagnosis of 5q SMA.
Diagnosed as later-onset (e.g., Type 2 or Type 3) SMA prior to receiving any treatment with therapy approved for SMA.
Non-ambulatory patients must be able to sit independently (sits up straight with head erect for at least 10 seconds; does not use arms or hands to balance body or support position) per World Health Organization (WHO) motor milestones definition at screening.
Ambulatory patients must have the ability to independently ambulate without aids or orthotics over 10 meters in 30 seconds or less at screening.
Receiving the same background SMA therapy (e.g., on an approved survival motor neuron (SMN) upregulator therapy such as nusinersen, or not on any SMA therapy) for at least 6 months prior to screening and anticipated to remain on that therapy throughout the duration of the study.
Nutritional status stable over the past 6 months and anticipated to be stable throughout the duration of the study.
Have no physical limitations that would prevent the patient from undergoing motor function outcome measures throughout the duration of the study.
Able to receive study drug infusions and provide blood samples through the use of a peripheral intravenous (IV) or a long-term IV access device that the patient has placed for reasons independent from the study throughout the duration of the study.
Able to adhere to the requirements of the protocol, including travel to the study center and completing all study procedures and study visits.
For patients who are expected to have reached reproductive maturity by the end of the study, adhere to study specific contraception requirements.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Thomas O. Crawford, MD | Johns Hopkins University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | United States | ||
| Stanford University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38330285 | Derived | Crawford TO, Darras BT, Day JW, Dunaway Young S, Duong T, Nelson LL, Barrett D, Song G, Bilic S, Cote S, Sadanowicz M, Iarrobino R, Xu TJ, O'Neil J, Rossello J, Place A, Kertesz N, Nomikos G, Chyung Y. Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study. Neurology. 2024 Mar 12;102(5):e209151. doi: 10.1212/WNL.0000000000209151. Epub 2024 Feb 8. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 - Monotherapy | Ambulatory Type 3 SMA apitegromab (SRK-015) 20mg/kg SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Aug 3, 2021 |
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Active treatment, randomized, double-blind for Cohort 3
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| Baseline to 12 months |
| Cohort 1: Proportion of Patients Achieving Various Magnitudes of Change in RHS Score From Baseline | The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA; it has a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the lowest level and 2 denotes the highest level of ability/function. The remaining 3 items are scored 0 or 1, where 0 denotes an inability and 1 denotes an ability to achieve. | Baseline to 12 months |
| Cohort 1: Change From Baseline in 6-Minute Walk Test (6MWT) | 6-Minute Walk Test The 6-Minute Walk Test (6MWT) is an assessment of exercise capacity and fatigue used for ambulatory patients with later-onset SMA who are directed to walk along a 25 meter course as fast as possible over 6 minutes. | Baseline to 12 months |
| Cohort 1: Change From Baseline in 30-Second Sit-to-Stand | The 30-Second Sit-to-Stand Test is an assessment of functional lower-limb strength that measures the maximal number of times a patient can transition from sitting to standing in 30 seconds. | Baseline to 12 months |
| Cohort 1: Change From Baseline in 10-Meter Walk/Run (From RHS) | The 10 Meter Walk/Run test is an enhanced function of the RHS used for ambulatory patients with Type 3 SMA. It is a measure of the time taken to walk/run 10 meters. The time to perform is summarized only for patients able to complete the RHS 10-meter walk/run item. | Baseline to 12 months |
| Cohort 1: Change From Baseline in Timed Rise From Floor (From RHS) | The timed rise from floor test is an enhanced function of the RHS used for ambulatory patients with Type 3 SMA. It is a measure of the time taken to rise to standing from the floor. The time to rise from floor is summarized only for patients able to complete the RHS rise from floor item. | Baseline to 12 months |
| Cohort 2 &3: Change From Baseline in HFMSE Total Score at Other Prespecified Timepoints | The Hammersmith Functional Motor Scale Expanded (HFMSE) assesses the physical abilities of patients with Type 2 and Type 3 SMA comprises 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. | Baseline to 12 months |
| Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in HFMSE Score From Baseline | The Hammersmith Functional Motor Scale Expanded (HFMSE) assesses the physical abilities of patients with Type 2 and Type 3 SMA comprises 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. | Baseline to 12 months |
| Cohort 2 & 3: Change in Baseline in Revised Upper Limb Module (RULM) Total Score | The RULM is a 20 item assessment of upper limb function in nonambulatory patients with SMA that was performed for patients who were 30 months of age or older at baseline. The 19 scored items assess functions that relate to everyday life, such as pressing a button and picking up a token; these items are scored 0, 1, or 2, where 0 denotes unable, 1 denotes able with modification, and 2 denotes able with no modification. The maximum score achievable is 37. | Baseline to 12 months |
| Cohort 2 & 3: Proportion of Patients Achieving a New WHO Motor Development Milestones Relative to Baseline | The WHO Multicenter Growth Reference Study performance criteria is being utilized to assess the World Health Organization (WHO) motor development milestones of patients with Type 2 and nonambulatory Type 3 SMA enrolled in Cohort 2 and Cohort 3 relative to baseline. The WHO milestone assessment consists of six items which were selected because they have been considered to be universal, fundamental, and simple to test and evaluate, they include 1) sitting without support, 2) hands and knees crawling, 3) standing with assistance, 4) walking with assistance, 5) standing alone, and 6) walking without assistance. Each item is recorded as 1 (unable), 2 (refusal), 3 (Yes) or 9 (did not test). The number of 3s was counted as the final score. The minimum was 0, which means no motor milestones were achieved; the maximum was 6, which means all 6 milestones were achieved. | Baseline to 12 months |
| Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in RULM Score From Baseline | The RULM is a 20 item assessment of upper limb function in nonambulatory patients with SMA that was performed for patients who were 30 months of age or older at baseline. The 19 scored items assess functions that relate to everyday life, such as pressing a button and picking up a token; these items are scored 0, 1, or 2, where 0 denotes unable, 1 denotes able with modification, and 2 denotes able with no modification. The maximum score achievable is 37. | Baseline to 12 months |
| Palo Alto |
| California |
| 94304 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| The Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | United States |
| Columbia University | New York | New York | 10032 | United States |
| Wake Forest School of Medicine | Winston-Salem | North Carolina | 27157 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Childrens Medical Center Dallas | Dallas | Texas | 75235 | United States |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23507 | United States |
| ASST Grande Ospedale Metropolitano Niguarda | Milan | 20162 | Italy |
| Centro Clinico Nemo Pediatrico Policlinico A. Gemelli-Università Cattolica Sacro Cuore | Roma | Italy |
| Universitair Medisch Centrum Utrecht | Utrecht | 3584 | Netherlands |
| Hospital Sant Joan de Déu | Barcelona | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| Cohort 1 - Dual Therapy |
Ambulatory Type 3 SMA apitegromab (SRK-015) + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| FG002 | Cohort 2 | Type 2 SMA / Non-Ambulatory Type 3 SMA apitegromab (SRK-015) 20 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| FG003 | Cohort 3 - Low Dose | Type 2 SMA apitegromab (SRK-015) 2 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| FG004 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| COMPLETED |
|
| NOT COMPLETED |
|
Intent-to-Treat (ITT) Set: All participants who were enrolled/randomized and received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 - Monotherapy | Ambulatory Type 3 SMA apitegromab (SRK-015) 20mg/kg SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| BG001 | Cohort 1 - Dual Therapy | Ambulatory Type 3 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| BG002 | Cohort 2 | Type 2 SMA / Non-Ambulatory Type 3 SMA apitegromab (SRK-015) 20 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| BG003 | Cohort 3 - Low Dose | Type 2 SMA apitegromab (SRK-015) 2 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| BG004 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| BG005 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Full Range | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] | The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA. For the ambulatory Type 3 patients 5-21 years of age in Cohort 1 (N=23), the primary endpoint was the change from baseline in RHS total score at month 12. The RHS has a minimum achievable score of 0 and a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the lowest level and 2 denotes the highest level of ability/function. The remaining 3 items are scored 0 or 1, where 0 denotes an inability and 1 denotes an ability to achieve. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. | The ITT Population: all enrolled/randomized patients who receive at least 1 dose of study drug. The ITT Population was the main population for analysis of efficacy endpoints; the analysis includes all assessments from all patients with the exception of assessments after missing 3 consecutive doses due to site access restrictions caused by COVID-19. The last observation carried forward (LOCF) method was used to impute the 12-month primary endpoint for data missing for other reasons. | Posted | Mean | Standard Deviation | score on a scale | Baseline up to 12 months |
|
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| Primary | Cohort 2 and Cohort 3: Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score at Day 364 (Visit 15) [Month 12] | Cohort 2: For the nonambulatory Type 2 and Type 3 patients 5-21 years of age in Cohort 2 (N=15), the primary efficacy endpoint was the change from baseline in HFMSE total score at month 12. Cohort 3: For the nonambulatory Type 2 patients ≥2 years of age in Cohort 3 (N=20), the primary efficacy endpoint was the change from baseline in HFMSE total score at month 12. The Hammersmith Functional Motor Scale Expanded (HFMSE) assesses the physical abilities of patients with Type 2 and Type 3 SMA comprises 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. | The ITT Population: all enrolled/randomized patients who receive at least one dose of study drug. The ITT Population was the main population for analysis of efficacy endpoints; the analysis included all assessments from all patients with the exception of assessments after missing 3 consecutive doses due to site access restrictions caused by COVID-19. The last observation carried forward (LOCF) method was used to impute the 12-month primary endpoint for data missing for other reasons. | Posted | Mean | Standard Deviation | score on a scale | Baseline up to 12 months |
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| Secondary | Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA; it has a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the lowest level and 2 denotes the highest level of ability/function. The remaining 3 items are scored 0 or 1, where 0 denotes an inability and 1 denotes an ability to achieve. | 1 patient in Cohort 1 Monotherapy did not complete Physical Therapy Assessments at V6 due to site imposed COVID-19 restrictions. 1 patient in Cohort 1 Dual Therapy withdrew consent following discontinuation of apitegromab after 3 months of treatment due to a nonserious TEAE of unresolved fatigue assessed as not related to apitegromab. | Posted | Count of Participants | Participants | Baseline to 12 months |
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| Secondary | Cohort 1: Proportion of Patients Achieving Various Magnitudes of Change in RHS Score From Baseline | The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA; it has a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the lowest level and 2 denotes the highest level of ability/function. The remaining 3 items are scored 0 or 1, where 0 denotes an inability and 1 denotes an ability to achieve. | Posted | Count of Participants | Participants | Baseline to 12 months |
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| Secondary | Cohort 1: Change From Baseline in 6-Minute Walk Test (6MWT) | 6-Minute Walk Test The 6-Minute Walk Test (6MWT) is an assessment of exercise capacity and fatigue used for ambulatory patients with later-onset SMA who are directed to walk along a 25 meter course as fast as possible over 6 minutes. | Posted | Mean | Standard Deviation | Meters | Baseline to 12 months |
|
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| Secondary | Cohort 1: Change From Baseline in 30-Second Sit-to-Stand | The 30-Second Sit-to-Stand Test is an assessment of functional lower-limb strength that measures the maximal number of times a patient can transition from sitting to standing in 30 seconds. | Posted | Mean | Standard Deviation | Stands | Baseline to 12 months |
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| Secondary | Cohort 1: Change From Baseline in 10-Meter Walk/Run (From RHS) | The 10 Meter Walk/Run test is an enhanced function of the RHS used for ambulatory patients with Type 3 SMA. It is a measure of the time taken to walk/run 10 meters. The time to perform is summarized only for patients able to complete the RHS 10-meter walk/run item. | Posted | Mean | Standard Deviation | Seconds | Baseline to 12 months |
|
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| Secondary | Cohort 1: Change From Baseline in Timed Rise From Floor (From RHS) | The timed rise from floor test is an enhanced function of the RHS used for ambulatory patients with Type 3 SMA. It is a measure of the time taken to rise to standing from the floor. The time to rise from floor is summarized only for patients able to complete the RHS rise from floor item. | Posted | Mean | Standard Deviation | Seconds | Baseline to 12 months |
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| Secondary | Cohort 2 &3: Change From Baseline in HFMSE Total Score at Other Prespecified Timepoints | The Hammersmith Functional Motor Scale Expanded (HFMSE) assesses the physical abilities of patients with Type 2 and Type 3 SMA comprises 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. | 1 patient in Cohort 2 and 3 patients in Cohort 3 (1 low dose and 2 high dose) missed 3 consecutive doses of apitegromab during the 12-month treatment period due to COVID-19-related site access restrictions and were not included in the primary analysis. In addition, 2 cohort 3 low dose patients skipped motor assessments at Day 168 (V8) and 1 cohort low dose patient at Day 365 (V15) due to COVID restrictions. | Posted | Count of Participants | Participants | Baseline to 12 months |
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| Secondary | Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in HFMSE Score From Baseline | The Hammersmith Functional Motor Scale Expanded (HFMSE) assesses the physical abilities of patients with Type 2 and Type 3 SMA comprises 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation. | 1 patient in Cohort 2 and 3 patients in Cohort 3 missed 3 consecutive doses of apitegromab during the 12-month treatment period due to COVID-19-related site access restrictions and were not included in the primary analysis. | Posted | Count of Participants | Participants | Baseline to 12 months |
| |||||||||||||||||||||||||||||||
| Secondary | Cohort 2 & 3: Change in Baseline in Revised Upper Limb Module (RULM) Total Score | The RULM is a 20 item assessment of upper limb function in nonambulatory patients with SMA that was performed for patients who were 30 months of age or older at baseline. The 19 scored items assess functions that relate to everyday life, such as pressing a button and picking up a token; these items are scored 0, 1, or 2, where 0 denotes unable, 1 denotes able with modification, and 2 denotes able with no modification. The maximum score achievable is 37. | 1 patient in Cohort 2 and 3 patients in Cohort 3 missed 3 consecutive doses of apitegromab during the 12-month treatment period due to COVID-19-related site access restrictions and were not included in the primary analysis. | Posted | Mean | Standard Deviation | Score on scale | Baseline to 12 months |
| ||||||||||||||||||||||||||||||
| Secondary | Cohort 2 & 3: Proportion of Patients Achieving a New WHO Motor Development Milestones Relative to Baseline | The WHO Multicenter Growth Reference Study performance criteria is being utilized to assess the World Health Organization (WHO) motor development milestones of patients with Type 2 and nonambulatory Type 3 SMA enrolled in Cohort 2 and Cohort 3 relative to baseline. The WHO milestone assessment consists of six items which were selected because they have been considered to be universal, fundamental, and simple to test and evaluate, they include 1) sitting without support, 2) hands and knees crawling, 3) standing with assistance, 4) walking with assistance, 5) standing alone, and 6) walking without assistance. Each item is recorded as 1 (unable), 2 (refusal), 3 (Yes) or 9 (did not test). The number of 3s was counted as the final score. The minimum was 0, which means no motor milestones were achieved; the maximum was 6, which means all 6 milestones were achieved. | 1 patient in Cohort 2 and 3 patients in Cohort 3 missed 3 consecutive doses of apitegromab during the 12-month treatment period due to COVID-19-related site access restrictions and were not included in the primary analysis. | Posted | Count of Participants | Participants | Baseline to 12 months |
| |||||||||||||||||||||||||||||||
| Secondary | Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in RULM Score From Baseline | The RULM is a 20 item assessment of upper limb function in nonambulatory patients with SMA that was performed for patients who were 30 months of age or older at baseline. The 19 scored items assess functions that relate to everyday life, such as pressing a button and picking up a token; these items are scored 0, 1, or 2, where 0 denotes unable, 1 denotes able with modification, and 2 denotes able with no modification. The maximum score achievable is 37. | 1 patient in Cohort 2 and 3 patients in Cohort 3 missed 3 consecutive doses of apitegromab during the 12-month treatment period due to COVID-19-related site access restrictions and were not included in the primary analysis. | Posted | Count of Participants | Participants | Baseline to 12 months |
|
Day 0 (Visit 1) to 48 months (Visit EC14)
Classification based on the FDA regulatory definition of an AE.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 - Monotherapy | Ambulatory Type 3 SMA apitegromab (SRK-015) 20 mg/kg SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. | 0 | 11 | 4 | 11 | 11 | 11 |
| EG001 | Cohort 1 - Dual Therapy | Ambulatory Type 3 SMA apitegromab (SRK-015) 20 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. | 0 | 12 | 4 | 12 | 11 | 12 |
| EG002 | Cohort 2 | Type 2 SMA / Non-Ambulatory Type 3 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. | 0 | 15 | 9 | 15 | 15 | 15 |
| EG003 | Cohort 3 - Low Dose | Type 2 SMA apitegromab (SRK-015) 2 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. | 0 | 10 | 6 | 10 | 10 | 10 |
| EG004 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. | 0 | 10 | 5 | 10 | 9 | 10 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gait Inability | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Post Lumbar-Puncture Syndrome | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tonsillar Hypertrophy | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Developmental hip dysplasia | Congenital, familial and genetic disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Liver Injury | Hepatobiliary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Pneumothorax traumatic | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Joint contracture | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Major depression | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Adenoidectomy | Surgical and medical procedures | MEDRA 26.1 | Systematic Assessment |
| |
| Hospitalization | Surgical and medical procedures | MEDRA 26.1 | Systematic Assessment |
| |
| Joint dislocation reduction | Surgical and medical procedures | MEDRA 26.1 | Systematic Assessment |
| |
| Scoliosis surgery | Surgical and medical procedures | MEDRA 26.1 | Systematic Assessment |
| |
| Spinal fusion surgery | Surgical and medical procedures | MEDRA 26.1 | Systematic Assessment |
| |
| Spinal implantation | Surgical and medical procedures | MEDRA 26.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Joint contracture | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Heart rate increased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Gait Inability | General disorders | MEDRA 26.1 | Systematic Assessment | Administration site conditions |
|
| Liver Injury | Hepatobiliary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Developmental hip dysplasia | Congenital, familial and genetic disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Ear congestion | Ear and labyrinth disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Motion sickness | Ear and labyrinth disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Astigmatism | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Chalazion | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Erythema of eyelid | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Excessive eye blinking | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Eyelid disorder | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Swelling of eyelid | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Aphthous ulcer | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Eosinophilic oesophagitis | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Eructation | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Faecaloma | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Food poisoning | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Gingival ulceration | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Lip haematoma | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Lip pain | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Lip ulceration | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Loose tooth | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Odynophagia | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tongue ulceration | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Application site rash | General disorders | MEDRA 26.1 | Systematic Assessment | General disorders and administration site conditions |
|
| Asthenia | Gastrointestinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MEDRA 26.1 | Systematic Assessment | General disorders and administration site conditions |
|
| Chest pain | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Discomfort | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Gait inability | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hyperpyrexia | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Infusion site bruising | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Infusion site nodule | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Infusion site pain | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Injection site rash | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Medical device site irritation | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Nodule | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Pain | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tissue infiltration | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vessel puncture site bruise | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vessel puncture site rash | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Injection site injury | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Injection site nodule | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Injection site pain | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Injection site rash | General disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Abscess neck | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Adenovirus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Beta haemolytic streptococcal infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Dysentery | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Enterobiasis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Folliculitis genital | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Heliobacter infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Infected bite | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Metapneumovirus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Molluscum contagiosum | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Myringitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Nail infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Norovirus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Pharyngotonsillitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Streptococcal infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Viral pharyngitis | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Viral rash | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MEDRA 26.1 | Systematic Assessment |
| |
| Anaemia postoperative | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Animal scratch | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Arthopod bite | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Concussion | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Fibula fracture | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Head crushing injury | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Immunisation reaction | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Post procedural fever | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Post-traumatic pain | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Procedural headache | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Procedural nausea | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Spinal column injury | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Urinary retention postoperative | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Vaccination complication | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Vascular access complication | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Vascular access site bruising | Injury, poisoning and procedural complications | MEDRA 26.1 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Blood ketone body | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Bone density decreased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Electrocardiogram abnormal | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Hepatic enzyme abnormal | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Serum ferritin decreased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Transaminases increased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Urine analysis abnormal | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Weight increased | Investigations | MEDRA 26.1 | Systematic Assessment |
| |
| Calcium deficiency | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Feeding disorder | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Increased appetite | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Obesity | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hip deformity | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Joint laxity | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Kyphosis | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Neuromuscular scoliosis | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Osteoporosis | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Spinal pain | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tendon pain | Musculoskeletal and connective tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MEDRA 26.1 | Systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Brain fog | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Drooling | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Adjustment disorder | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Attention deficit hyperactivity disorder | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Autism spectrum disorder | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Autism spectrum disorder | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Fear of injection | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Obsessive-compulsive disorder | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Panic attack | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Bladder pain | Renal and urinary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Polyuria | Renal and urinary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Pelvic pain | Reproductive system and breast disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Asthmatic crisis | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Bronchial secretion retention | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Paranasal sinus discomfort | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Snoring | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tonsillar erythema | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Dry Skin | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Erythema nodosum | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypohidrosis | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Keloid scar | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Miliaria | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Pityriasis rosea | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Pruritis | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rash follicular | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Skin striae | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Bone operation | Surgical and medical procedures | MEDRA 26.1 | Systematic Assessment |
| |
| Muscle operation | Vascular disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Tooth extraction | Vascular disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MEDRA 26.1 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MEDRA 26.1 | Systematic Assessment |
|
Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 45 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trials Coordinator | Scholar Rock | 833-724-7625 | clinicaltrials@scholarrock.com |
| Jan 19, 2022 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| D014897 | Spinal Muscular Atrophies of Childhood |
| D009468 | Neuromuscular Diseases |
| D009133 | Muscular Atrophy |
| D001284 | Atrophy |
| D020879 | Neuromuscular Manifestations |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009461 | Neurologic Manifestations |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012816 | Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C000722231 | apitegromab |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| OG001 | Cohort 3 - Low Dose | Type 2 SMA apitegromab (SRK-015) 2 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| OG002 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
|
| OG002 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
|
|
| OG002 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
|
|
| OG002 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
|
|
| OG001 |
| Cohort 3 - Low Dose |
Type 2 SMA apitegromab (SRK-015) 2 mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
| OG002 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
|
|
| OG002 | Cohort 3 - High Dose | Type 2 SMA apitegromab (SRK-015) 20mg/kg + nusinersen SRK-015: SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 was administered every 4 weeks by intravenous infusion. |
|
|