A Phase 2 interventional study of SRK-015 in Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 and Spinal Muscular Atrophy Type 2, sponsored by Scholar Rock, Inc.. Completed at 16 sites in 4 countries. Open to participants aged 2 Years to 21 Years. Per ClinicalTrials.gov, last updated 2024-12-02.
Sponsored by Scholar Rock, Inc. · Phase 2, Interventional, and Treatment
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.
494 studies on the registry are indexed under Muscular Atrophy; 94 are open to participants now.
This study's enrollment of 58 is above the median of 33 across 335 interventional studies indexed under Muscular Atrophy.
Browse Muscular Atrophy studies →Scholar Rock, Inc. is the lead sponsor of 9 studies on the registry; 3 are open to participants now.
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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.
Exclusion Criteria:
Ambulatory Type 3 SMA
Biological: SRK-015
Type 2 SMA / Non-Ambulatory Type 3 SMA
Biological: SRK-015
Type 2 SMA
Biological: 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.
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.
Time frame: 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.
Time frame: Baseline up to 12 months
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline to 12 months
| Milestone | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|---|---|
| Started | 11 | 12 | 15 | 10 | 10 |
| Completed | 6 | 10 | 13 | 10 | 10 |
| Not completed | 5 | 2 | 2 | 0 | 0 |
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.
| score on a scale | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy |
|---|---|---|
| Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] | -0.1 ± 5.01 | 0.0 ± 2.56 |
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.
| score on a scale | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Cohort 2 and Cohort 3: Change From Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score at Day 364 (Visit 15) [Month 12] | 0.6 ± 3.5 | 5.3 ± 8.93 | 7.1 ± 6.42 |
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.
| Participants | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy |
|---|---|---|
| ≥5pt at Day 56 (V4) | 1 | 1 |
| ≥5pt at Day 112 (V6) | 1 | 1 |
| ≥5pt at Day 168 (V8) | 2 | 1 |
| ≥5pt Month 12 Endpoint | 1 | 0 |
| ≥3pt at Day 56 (V4) | 3 | 3 |
| ≥3pt at Day 112 (V6) | 1 | 2 |
| ≥3pt at Day 168 (V8) | 4 | 1 |
| ≥3pt at Month 12 Endpoint | 3 | 3 |
| ≥1pt at Day 56 (V4) | 8 | 8 |
| ≥1pt at Day 112 (V6) | 3 | 5 |
| ≥1pt at Day 168 (V8) | 7 | 4 |
| ≥1pt at Month 12 Endpoint | 6 | 5 |
| No Change at Day 56 (V4) | 1 | 1 |
| No Change at Day 112 (V6) | 5 | 1 |
| No Change at Day 168 (V8) | 1 | 5 |
| No Change at Month 12 Endpoint | 0 | 2 |
| Decrease at Day 56 (V4) | 2 | 3 |
| Decrease at Day 112 (V6) | 2 | 5 |
| Decrease at Day 168 (V8) | 3 | 2 |
| Decrease at Month 12 Endpoint | 5 | 5 |
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.
| Participants | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy |
|---|---|---|
| ≥3pt increase | 3 | 3 |
| ≥1pt increase | 6 | 5 |
| No Change | 0 | 2 |
| Decrease | 5 | 5 |
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.
| Meters | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy |
|---|---|---|
| Cohort 1: Change From Baseline in 6-Minute Walk Test (6MWT) | -20.6 ± 44.96 | 11.0 ± 33.67 |
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.
| Stands | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy |
|---|---|---|
| Cohort 1: Change From Baseline in 30-Second Sit-to-Stand | -0.6 ± 1.63 | -0.2 ± 1.95 |
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.
| Seconds | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy |
|---|---|---|
| Cohort 1: Change From Baseline in 10-Meter Walk/Run (From RHS) | 0.1 ± 0.32 | 0.9 ± 3.32 |
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.
| Seconds | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy |
|---|---|---|
| Cohort 1: Change From Baseline in Timed Rise From Floor (From RHS) | 0.6 ± 1.75 | -0.6 ± 1.43 |
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.
| Participants | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|
| ≥5pt at Day 56 (V4) | 1 | 0 | 3 |
| ≥5pt at Day 112 (V6) | 1 | 2 | 5 |
| ≥5pt at Day 168 (V8) | 1 | 3 | 4 |
| ≥5pt at Month 12 Endpoint | 2 | 5 | 5 |
| ≥3pt at Day 56 (V4) | 1 | 4 | 5 |
| ≥3pt at Day 112 (V6) | 2 | 3 | 5 |
| ≥3pt at Day 168 (V8) | 2 | 4 | 5 |
| ≥3pt at Month 12 Endpoint | 4 | 5 | 5 |
| ≥1pt at Day 56 (V4) | 4 | 6 | 8 |
| ≥1pt at Day 112 (V6) | 6 | 5 | 9 |
| ≥1pt at Day 168 (V8) | 10 | 5 | 8 |
| ≥1pt at Month 12 Endpoint | 9 | 7 | 7 |
| No Change at Day 56 (V4) | 4 | 1 | 1 |
| No Change at Day 112 (V6) | 5 | 2 | 1 |
| No Change at Day 168 (V8) | 1 | 1 | 0 |
| No Change at Month 12 Endpoint | 1 | 0 | 0 |
| Decrease at Day 56 (V4) | 7 | 3 | 1 |
| Decrease at Day 112 (V6) | 3 | 3 | 0 |
| Decrease at Day 168 (V8) | 3 | 1 | 0 |
| Decrease at Month 12 Endpoint | 4 | 2 | 1 |
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.
| Participants | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|
| ≥3pt increase | 4 | 5 | 5 |
| ≥1pt increase | 9 | 7 | 7 |
| No Change | 1 | 0 | 0 |
| Decrease | 4 | 2 | 1 |
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.
| Score on scale | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Cohort 2 & 3: Change in Baseline in Revised Upper Limb Module (RULM) Total Score | 1.2 ± 2.99 | 0.9 ± 2.62 | 1 ± 2.94 |
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.
| Participants | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|
| ≥2pt increase | 1 | 0 | 1 |
| ≥1pt increase | 3 | 1 | 1 |
| No Change | 11 | 7 | 6 |
| Decrease | 0 | 1 | 1 |
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.
| Participants | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|
| ≥2pt increase | 5 | 3 | 2 |
| ≥1pt increase | 8 | 3 | 3 |
| No change | 1 | 3 | 2 |
| Decrease | 5 | 3 | 2 |
Collected over Day 0 (Visit 1) to 48 months (Visit EC14). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1 - Monotherapy | 0/11 (0%) | 4/11 (36.4%) | 11/11 (100%) |
| Cohort 1 - Dual Therapy | 0/12 (0%) | 4/12 (33.3%) | 11/12 (91.7%) |
| Cohort 2 | 0/15 (0%) | 9/15 (60%) | 15/15 (100%) |
| Cohort 3 - Low Dose | 0/10 (0%) | 6/10 (60%) | 10/10 (100%) |
| Cohort 3 - High Dose | 0/10 (0%) | 5/10 (50%) | 9/10 (90%) |
| Event | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|---|---|
| Scoliosis surgerySurgical and medical procedures | 0/11 | 1/12 | 5/15 | 0/10 | 1/10 |
| PneumoniaInfections and infestations | 0/11 | 0/12 | 0/15 | 1/10 | 2/10 |
| ScoliosisMusculoskeletal and connective tissue disorders | 1/11 | 0/12 | 3/15 | 0/10 | 1/10 |
| Gait InabilityGeneral disorders | 2/11 | 0/12 | 0/15 | 0/10 | 0/10 |
| Upper Respiratory Tract InfectionInfections and infestations | 1/11 | 0/12 | 0/15 | 0/10 | 1/10 |
| Acute respiratory failureRespiratory, thoracic and mediastinal disorders | 0/11 | 0/12 | 0/15 | 0/10 | 1/10 |
| Tonsillar HypertrophyRespiratory, thoracic and mediastinal disorders | 0/11 | 0/12 | 0/15 | 1/10 | 0/10 |
| VomitingGastrointestinal disorders | 1/11 | 0/12 | 0/15 | 1/10 | 0/10 |
| Respiratory syncytial virus infectionInfections and infestations | 0/11 | 0/12 | 1/15 | 0/10 | 1/10 |
| Rhinovirus infectionInfections and infestations | 0/11 | 0/12 | 0/15 | 1/10 | 0/10 |
| Event | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose |
|---|---|---|---|---|---|
| PyrexiaGeneral disorders | 2/11 | 5/12 | 6/15 | 4/10 | 9/10 |
| VomitingGastrointestinal disorders | 1/11 | 1/12 | 2/15 | 6/10 | 8/10 |
| CoughRespiratory, thoracic and mediastinal disorders | 2/11 | 5/12 | 4/15 | 5/10 | 7/10 |
| HeadacheNervous system disorders | 7/11 | 5/12 | 5/15 | 4/10 | 3/10 |
| NasopharyngitisInfections and infestations | 4/11 | 1/12 | 6/15 | 6/10 | 5/10 |
| COVID-19Infections and infestations | 5/11 | 3/12 | 6/15 | 6/10 | 6/10 |
| Upper respiratory tract infectionInfections and infestations | 5/11 | 5/12 | 5/15 | 4/10 | 5/10 |
| NauseaGastrointestinal disorders | 4/11 | 3/12 | 3/15 | 5/10 | 3/10 |
| FallInjury, poisoning and procedural complications | 5/11 | 6/12 | 1/15 | 2/10 | 1/10 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 2/11 | 1/12 | 2/15 | 2/10 | 4/10 |
Intent-to-Treat (ITT) Set: All participants who were enrolled/randomized and received at least 1 dose of study drug.
| Age, Continuous(Years) | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose | Total |
|---|---|---|---|---|---|---|
| Mean | 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(Participants) | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose | Total |
|---|---|---|---|---|---|---|
| Female | 8 | 7 | 8 | 3 | 5 | 31 |
| Male | 3 | 5 | 7 | 7 | 5 | 27 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose | Total |
|---|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 2 | 2 | 0 | 1 | 5 |
| Not Hispanic or Latino | 11 | 10 | 13 | 10 | 9 | 53 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1 - Monotherapy | Cohort 1 - Dual Therapy | Cohort 2 | Cohort 3 - Low Dose | Cohort 3 - High Dose | Total |
|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 6 | 0 | 2 | 0 | 1 | 9 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 1 | 0 | 1 | 2 |
| White | 5 | 12 | 12 | 10 | 8 | 47 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 |
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Scholar Rock, Inc.