The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy (TOPAZ)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03921528
Recruitment Status : Completed
First Posted : April 19, 2019
Results First Posted : November 17, 2022
Last Update Posted : April 16, 2024
Sponsor:
Information provided by (Responsible Party):
Scholar Rock, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Spinal Muscular Atrophy
Spinal Muscular Atrophy Type 3
Spinal Muscular Atrophy Type 2
SMA
Neuromuscular Diseases
Muscular Atrophy
Atrophy
Muscular Atrophy, Spinal
Neuromuscular Manifestations
Intervention Biological: SRK-015
Enrollment 58
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Period Title: Overall Study
Started 11 12 15 10 10
Completed 11 11 15 10 10
Not Completed 0 1 0 0 0
Reason Not Completed
Withdrawal by Subject             0             1             0             0             0
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose Total
Hide Arm/Group Description

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

 Total of all reporting groups
Overall Number of Baseline Participants 11 12 15 10 10 58
Hide Baseline Analysis Population Description
Intent-to-Treat (ITT) Set: All participants who were randomized and recieved at least 1 dose of study drug.
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 11 participants 12 participants 15 participants 10 participants 10 participants 58 participants
12.1
(7 to 19)
13.1
(7 to 21)
11.7
(8 to 19)
4.1
(2 to 6)
3.8
(2 to 6)
9.67
(2 to 21)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 12 participants 15 participants 10 participants 10 participants 58 participants
Female
8
  72.7%
7
  58.3%
8
  53.3%
3
  30.0%
5
  50.0%
31
  53.4%
Male
3
  27.3%
5
  41.7%
7
  46.7%
7
  70.0%
5
  50.0%
27
  46.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 12 participants 15 participants 10 participants 10 participants 58 participants
Hispanic or Latino
0
   0.0%
2
  16.7%
2
  13.3%
0
   0.0%
1
  10.0%
5
   8.6%
Not Hispanic or Latino
11
 100.0%
10
  83.3%
13
  86.7%
10
 100.0%
9
  90.0%
53
  91.4%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 12 participants 15 participants 10 participants 10 participants 58 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
6
  54.5%
0
   0.0%
2
  13.3%
0
   0.0%
1
  10.0%
9
  15.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
1
   6.7%
0
   0.0%
1
  10.0%
2
   3.4%
White
5
  45.5%
12
 100.0%
12
  80.0%
10
 100.0%
8
  80.0%
47
  81.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12]
Hide Description 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 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 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.
Time Frame Baseline up to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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 the missing assessments due to missing 3 doses due to COVID related site restrictions (assessments excluded post dose missed). LOCF used to impute 12-month primary endpoint for other types of missingness.
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 11 12
Mean (Standard Deviation)
Unit of Measure: score on a scale
-0.4  (5.20) -0.3  (2.67)
2.Primary Outcome
Title Cohort 2 and Cohort 3: Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score at Day 364 (Visit 15) [Month 12]
Hide Description

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.
Time Frame Baseline up to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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 will include all assessments from all patients with the exception of the missing assessments due to missing 3 doses due to COVID related site restrictions, whereas assessments after dose-missing were excluded. LOCF were used to impute 12-month primary endpoint for other types of missingness.
Arm/Group Title Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 14 9 8
Mean (Standard Deviation)
Unit of Measure: score on a scale
0.6  (3.5) 5.3  (8.93) 7.1  (6.42)
3.Secondary Outcome
Title Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description

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.
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 11 12
Measure Type: Count of Participants
Unit of Measure: Participants
≥5pt at Day 56 (V4) Number Analyzed 11 participants 12 participants
1
   9.1%
1
   8.3%
≥5pt at Day 112 (V6) Number Analyzed 10 participants 11 participants
1
  10.0%
1
   9.1%
≥5pt at Day 168 (V8) Number Analyzed 11 participants 11 participants
1
   9.1%
1
   9.1%
≥5pt Month 12 Endpoint Number Analyzed 11 participants 12 participants
1
   9.1%
0
   0.0%
≥3pt at Day 56 (V4) Number Analyzed 11 participants 12 participants
3
  27.3%
2
  16.7%
≥3pt at Day 112 (V6) Number Analyzed 10 participants 11 participants
1
  10.0%
2
  18.2%
≥3pt at Day 168 (V8) Number Analyzed 11 participants 11 participants
4
  36.4%
1
   9.1%
≥3pt at Month 12 Endpoint Number Analyzed 11 participants 12 participants
3
  27.3%
2
  16.7%
≥1pt at Day 56 (V4) Number Analyzed 11 participants 12 participants
8
  72.7%
8
  66.7%
≥1pt at Day 112 (V6) Number Analyzed 10 participants 11 participants
3
  30.0%
5
  45.5%
≥1pt at Day 168 (V8) Number Analyzed 11 participants 11 participants
7
  63.6%
4
  36.4%
≥1pt at Month 12 Endpoint Number Analyzed 11 participants 12 participants
4
  36.4%
5
  41.7%
No Change at Day 56 (V4) Number Analyzed 11 participants 12 participants
1
   9.1%
0
   0.0%
No Change at Day 112 (V6) Number Analyzed 10 participants 11 participants
3
  30.0%
1
   9.1%
No Change at Day 168 (V8) Number Analyzed 11 participants 11 participants
1
   9.1%
3
  27.3%
No Change at Month 12 Endpoint Number Analyzed 11 participants 12 participants
2
  18.2%
2
  16.7%
Decrease at Day 56 (V4) Number Analyzed 11 participants 12 participants
2
  18.2%
4
  33.3%
Decrease at Day 112 (V6) Number Analyzed 10 participants 11 participants
4
  40.0%
5
  45.5%
Decrease at Day 168 (V8) Number Analyzed 11 participants 11 participants
3
  27.3%
4
  36.4%
Decrease at Month 12 Endpoint Number Analyzed 11 participants 12 participants
5
  45.5%
5
  41.7%
4.Secondary Outcome
Title Cohort 1: Proportion of Patients Achieving Various Magnitudes of Change in RHS Score From Baseline
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 11 12
Measure Type: Count of Participants
Unit of Measure: Participants
≥3pt increase
3
  27.3%
2
  16.7%
≥1pt increase
4
  36.4%
5
  41.7%
No Change
2
  18.2%
2
  16.7%
Decrease
5
  45.5%
5
  41.7%
5.Secondary Outcome
Title Cohort 1: Change From Baseline in 6-Minute Walk Test (6MWT)
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 8 11
Mean (Standard Deviation)
Unit of Measure: Meters
-20.6  (44.96) 11.0  (33.67)
6.Secondary Outcome
Title Cohort 1: Change From Baseline in 30-Second Sit-to-Stand
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 11 12
Mean (Standard Deviation)
Unit of Measure: Stands
-0.6  (1.63) -0.2  (1.95)
7.Secondary Outcome
Title Cohort 1: Change From Baseline in 10-Meter Walk/Run (From RHS)
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 8 12
Mean (Standard Deviation)
Unit of Measure: Seconds
-0.2  (0.78) 1.5  (4.07)
8.Secondary Outcome
Title Cohort 1: Change From Baseline in Timed Rise From Floor (From RHS)
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 8 12
Mean (Standard Deviation)
Unit of Measure: Seconds
0.4  (1.36) 3.8  (12.48)
9.Secondary Outcome
Title Cohort 2 &3: Change From Baseline in HFMSE Total Score at Other Prespecified Timepoints
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 15 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
≥5pt at Day 56 (V4) Number Analyzed 15 participants 10 participants 10 participants
1
   6.7%
0
   0.0%
3
  30.0%
≥5pt at Day 112 (V6) Number Analyzed 14 participants 10 participants 10 participants
1
   7.1%
2
  20.0%
5
  50.0%
≥5pt at Day 168 (V8) Number Analyzed 14 participants 7 participants 8 participants
1
   7.1%
3
  42.9%
4
  50.0%
≥5pt at Month 12 Endpoint Number Analyzed 14 participants 9 participants 8 participants
2
  14.3%
5
  55.6%
5
  62.5%
≥3pt at Day 56 (V4) Number Analyzed 15 participants 10 participants 10 participants
1
   6.7%
4
  40.0%
5
  50.0%
≥3pt at Day 112 (V6) Number Analyzed 14 participants 10 participants 10 participants
2
  14.3%
3
  30.0%
5
  50.0%
≥3pt at Day 168 (V8) Number Analyzed 14 participants 7 participants 8 participants
2
  14.3%
4
  57.1%
5
  62.5%
≥3pt at Month 12 Endpoint Number Analyzed 14 participants 9 participants 8 participants
4
  28.6%
5
  55.6%
5
  62.5%
≥1pt at Day 56 (V4) Number Analyzed 15 participants 10 participants 10 participants
4
  26.7%
6
  60.0%
8
  80.0%
≥1pt at Day 112 (V6) Number Analyzed 14 participants 10 participants 10 participants
6
  42.9%
5
  50.0%
9
  90.0%
≥1pt at Day 168 (V8) Number Analyzed 14 participants 7 participants 8 participants
10
  71.4%
5
  71.4%
8
 100.0%
≥1pt at Month 12 Endpoint Number Analyzed 14 participants 9 participants 8 participants
9
  64.3%
7
  77.8%
7
  87.5%
No Change at Day 56 (V4) Number Analyzed 15 participants 10 participants 10 participants
4
  26.7%
1
  10.0%
1
  10.0%
No Change at Day 112 (V6) Number Analyzed 14 participants 10 participants 10 participants
5
  35.7%
2
  20.0%
1
  10.0%
No Change at Day 168 (V8) Number Analyzed 14 participants 7 participants 8 participants
1
   7.1%
1
  14.3%
0
   0.0%
No Change at Month 12 Endpoint Number Analyzed 14 participants 9 participants 8 participants
1
   7.1%
0
   0.0%
0
   0.0%
Decrease at Day 56 (V4) Number Analyzed 15 participants 10 participants 10 participants
7
  46.7%
3
  30.0%
1
  10.0%
Decrease at Day 112 (V6) Number Analyzed 14 participants 10 participants 10 participants
3
  21.4%
3
  30.0%
0
   0.0%
Decrease at Day 168 (V8) Number Analyzed 14 participants 7 participants 8 participants
3
  21.4%
1
  14.3%
0
   0.0%
Decrease at Month 12 Endpoint Number Analyzed 14 participants 9 participants 8 participants
4
  28.6%
2
  22.2%
1
  12.5%
10.Secondary Outcome
Title Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in HFMSE Score From Baseline
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 14 9 8
Measure Type: Count of Participants
Unit of Measure: Participants
≥3pt increase
4
  28.6%
5
  55.6%
5
  62.5%
≥1pt increase
9
  64.3%
7
  77.8%
7
  87.5%
No Change
1
   7.1%
0
   0.0%
0
   0.0%
Decrease
4
  28.6%
2
  22.2%
1
  12.5%
11.Secondary Outcome
Title Cohort 2 & 3: Change in Baseline in Revised Upper Limb Module (RULM) Total Score
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 14 9 8
Mean (Standard Deviation)
Unit of Measure: Score on scale
1.2  (2.99) 0.9  (2.62) 1  (2.94)
12.Secondary Outcome
Title Cohort 2 & 3: Proportion of Patients Achieving a New WHO Motor Development Milestones Relative to Baseline
Hide Description 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 will be counted as the final score. The minimum will be 0, which means no motor milestones were achieved; the maximum will be 6, which means all 6 milestones were achieved.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 14 9 8
Measure Type: Count of Participants
Unit of Measure: Participants
≥2pt increase
1
   7.1%
0
   0.0%
1
  12.5%
≥1pt increase
3
  21.4%
1
  11.1%
1
  12.5%
No Change
11
  78.6%
7
  77.8%
6
  75.0%
Decrease
0
   0.0%
1
  11.1%
1
  12.5%
13.Secondary Outcome
Title Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in RULM Score From Baseline
Hide Description 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.
Time Frame Baseline to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description:

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
Overall Number of Participants Analyzed 14 9 7
Measure Type: Count of Participants
Unit of Measure: Participants
≥2pt increase
5
  35.7%
3
  33.3%
2
  28.6%
≥1pt increase
8
  57.1%
3
  33.3%
3
  42.9%
No change
1
   7.1%
3
  33.3%
2
  28.6%
Decrease
5
  35.7%
3
  33.3%
2
  28.6%
Time Frame Day 0 (Visit 1) to 12 months (Visit 15)
Adverse Event Reporting Description Classification based on the FDA regulatory definition of an AE.
 
Arm/Group Title Cohort 1 - Monotherapy Cohort 1 - Dual Therapy Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Hide Arm/Group Description

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.

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 will be administered every 4 weeks by intravenous infusion.
All-Cause Mortality
Cohort 1 - Monotherapy Cohort 1 - Dual Therapy Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/11 (0.00%)      0/12 (0.00%)      0/15 (0.00%)      0/10 (0.00%)      0/10 (0.00%)    
Hide Serious Adverse Events
Cohort 1 - Monotherapy Cohort 1 - Dual Therapy Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/11 (27.27%)      1/12 (8.33%)      0/15 (0.00%)      1/10 (10.00%)      0/10 (0.00%)    
General disorders           
Gait Inability † 1  2/11 (18.18%)  0/12 (0.00%)  0/15 (0.00%)  0/10 (0.00%)  0/10 (0.00%) 
Infections and infestations           
Upper Respiratory Tract Infection † 1  1/11 (9.09%)  0/12 (0.00%)  0/15 (0.00%)  0/10 (0.00%)  0/10 (0.00%) 
Injury, poisoning and procedural complications           
Post Lumbar-Puncture Syndrome † 1  0/11 (0.00%)  1/12 (8.33%)  0/15 (0.00%)  0/10 (0.00%)  0/10 (0.00%) 
Respiratory, thoracic and mediastinal disorders           
Adenoidal Hypertrophy † 1  0/11 (0.00%)  0/12 (0.00%)  0/15 (0.00%)  1/10 (10.00%)  0/10 (0.00%) 
Tonsillar Hypertrophy † 1  0/11 (0.00%)  0/12 (0.00%)  0/15 (0.00%)  1/10 (10.00%)  0/10 (0.00%) 
1
Term from vocabulary, MEDRA 23.0
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1 - Monotherapy Cohort 1 - Dual Therapy Cohort 2 Cohort 3 - Low Dose Cohort 3 - High Dose
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   11/11 (100.00%)      10/12 (83.33%)      14/15 (93.33%)      9/10 (90.00%)      9/10 (90.00%)    
Gastrointestinal disorders           
Vomiting † 1  0/11 (0.00%)  0 1/12 (8.33%)  1 1/15 (6.67%)  1 3/10 (30.00%)  3 3/10 (30.00%)  3
Nausea † 1  1/11 (9.09%)  1 2/12 (16.67%)  2 1/15 (6.67%)  1 2/10 (20.00%)  2 1/10 (10.00%)  1
Abdominal pain upper † 1  1/11 (9.09%)  1 0/12 (0.00%)  0 1/15 (6.67%)  1 1/10 (10.00%)  1 1/10 (10.00%)  1
Diarrhoea † 1  0/11 (0.00%)  0 1/12 (8.33%)  1 0/15 (0.00%)  0 2/10 (20.00%)  2 1/10 (10.00%)  1
General disorders           
Pyrexia † 1  1/11 (9.09%)  1 1/12 (8.33%)  1 2/15 (13.33%)  2 3/10 (30.00%)  3 6/10 (60.00%)  6
Fatigue † 1  1/11 (9.09%)  1 2/12 (16.67%)  2 1/15 (6.67%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
Infections and infestations           
Upper respiratory tract infection † 1  2/11 (18.18%)  2 2/12 (16.67%)  2 2/15 (13.33%)  2 3/10 (30.00%)  3 3/10 (30.00%)  3
Nasopharyngitis † 1  1/11 (9.09%)  1 1/12 (8.33%)  1 4/15 (26.67%)  4 3/10 (30.00%)  3 3/10 (30.00%)  3
Influenza † 1  0/11 (0.00%)  0 0/12 (0.00%)  0 2/15 (13.33%)  2 2/10 (20.00%)  2 1/10 (10.00%)  1
Ear infection † 1  0/11 (0.00%)  0 1/12 (8.33%)  1 1/15 (6.67%)  1 2/10 (20.00%)  2 0/10 (0.00%)  0
Injury, poisoning and procedural complications           
Fall † 1  2/11 (18.18%)  2 2/12 (16.67%)  2 0/15 (0.00%)  0 1/10 (10.00%)  1 1/10 (10.00%)  1
Post lumbar puncture syndrome † 1  1/11 (9.09%)  1 1/12 (8.33%)  1 1/15 (6.67%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
Contusion † 1  0/11 (0.00%)  0 2/12 (16.67%)  2 1/15 (6.67%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
Skin laceration † 1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/15 (0.00%)  0 1/10 (10.00%)  1 1/10 (10.00%)  1
Investigations           
Heart rate increased † 1  1/11 (9.09%)  1 0/12 (0.00%)  0 2/15 (13.33%)  2 0/10 (0.00%)  0 0/10 (0.00%)  0
Musculoskeletal and connective tissue disorders           
Scoliosis † 1  0/11 (0.00%)  0 3/12 (25.00%)  3 2/15 (13.33%)  2 0/10 (0.00%)  0 1/10 (10.00%)  1
Musculoskeletal pain † 1  0/11 (0.00%)  0 0/12 (0.00%)  0 4/15 (26.67%)  4 0/10 (0.00%)  0 0/10 (0.00%)  0
Joint contracture † 1  0/11 (0.00%)  0 0/12 (0.00%)  0 1/15 (6.67%)  1 1/10 (10.00%)  1 1/10 (10.00%)  1
Muscle contracture † 1  0/11 (0.00%)  0 0/12 (0.00%)  0 2/15 (13.33%)  2 1/10 (10.00%)  1 0/10 (0.00%)  0
Muscle spasms † 1  0/11 (0.00%)  0 2/12 (16.67%)  2 0/15 (0.00%)  0 1/10 (10.00%)  1 0/10 (0.00%)  0
Pain in extremity † 1  1/11 (9.09%)  1 1/12 (8.33%)  1 1/15 (6.67%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
Nervous system disorders           
Headache † 1  5/11 (45.45%)  5 3/12 (25.00%)  3 3/15 (20.00%)  3 2/10 (20.00%)  2 1/10 (10.00%)  1
Dizziness † 1  3/11 (27.27%)  3 1/12 (8.33%)  1 2/15 (13.33%)  2 0/10 (0.00%)  0 0/10 (0.00%)  0
Respiratory, thoracic and mediastinal disorders           
Cough † 1  1/11 (9.09%)  1 3/12 (25.00%)  3 2/15 (13.33%)  2 3/10 (30.00%)  3 4/10 (40.00%)  4
Nasal congestion † 1  1/11 (9.09%)  1 0/12 (0.00%)  0 2/15 (13.33%)  2 0/10 (0.00%)  0 2/10 (20.00%)  2
Rhinorrhoea † 1  1/11 (9.09%)  1 0/12 (0.00%)  0 0/15 (0.00%)  0 2/10 (20.00%)  2 2/10 (20.00%)  2
Oropharyngeal pain † 1  0/11 (0.00%)  0 0/12 (0.00%)  0 3/15 (20.00%)  3 0/10 (0.00%)  0 0/10 (0.00%)  0
Skin and subcutaneous tissue disorders           
Rash † 1  2/11 (18.18%)  2 0/12 (0.00%)  0 1/15 (6.67%)  1 2/10 (20.00%)  2 2/10 (20.00%)  2
Erythema † 1  0/11 (0.00%)  0 2/12 (16.67%)  2 3/15 (20.00%)  3 0/10 (0.00%)  0 0/10 (0.00%)  0
1
Term from vocabulary, MEDRA 23.0
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Trials Coordinator
Organization: Scholar Rock
Phone: 833-724-7625
EMail: clinicaltrials@scholarrock.com
Layout table for additonal information
Responsible Party: Scholar Rock, Inc.
ClinicalTrials.gov Identifier: NCT03921528    
Other Study ID Numbers: SRK-015-002
First Submitted: April 16, 2019
First Posted: April 19, 2019
Results First Submitted: January 19, 2022
Results First Posted: November 17, 2022
Last Update Posted: April 16, 2024