Trial · NCT03921528
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
NCT03921528, Phase 2, Completed, Scholar Rock, Inc.; results first posted 2022-11-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy. Trials, record NCT03921528. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03921528
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
- NCT number
- NCT03921528
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 58 participants, actual
- Lead sponsor
- Scholar Rock, Inc.
- 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
- Interventions
- SRK-015
- First posted
- 2019-04-19
- Last updated
- 2024-12-02
- Results first posted
- 2022-11-17
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] | Baseline up to 12 months | 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. |
| 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] | Baseline up to 12 months | 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. |
| Secondary | Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | Baseline to 12 months | 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. |
| Secondary | Cohort 1: Proportion of Patients Achieving Various Magnitudes of Change in RHS Score From Baseline | Baseline to 12 months | 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. |
| Secondary | Cohort 1: Change From Baseline in 6-Minute Walk Test (6MWT) | Baseline to 12 months | 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. |
| Secondary | Cohort 1: Change From Baseline in 30-Second Sit-to-Stand | Baseline to 12 months | 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. |
| Secondary | Cohort 1: Change From Baseline in 10-Meter Walk/Run (From RHS) | Baseline to 12 months | 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. |
| Secondary | Cohort 1: Change From Baseline in Timed Rise From Floor (From RHS) | Baseline to 12 months | 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. |
| Secondary | Cohort 2 &3: Change From Baseline in HFMSE Total Score at Other Prespecified Timepoints | Baseline to 12 months | 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. |
| Secondary | Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in HFMSE Score From Baseline | Baseline to 12 months | 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 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] | Baseline up to 12 months | 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. | score on a scale | MEAN |
| 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] | Baseline up to 12 months | 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. | score on a scale | MEAN |
| SECONDARY | Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | Baseline to 12 months | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Cohort 1: Proportion of Patients Achieving Various Magnitudes of Change in RHS Score From Baseline | Baseline to 12 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Cohort 1: Change From Baseline in 6-Minute Walk Test (6MWT) | Baseline to 12 months | Meters | MEAN | |
| SECONDARY | Cohort 1: Change From Baseline in 30-Second Sit-to-Stand | Baseline to 12 months | Stands | MEAN | |
| SECONDARY | Cohort 1: Change From Baseline in 10-Meter Walk/Run (From RHS) | Baseline to 12 months | Seconds | MEAN | |
| SECONDARY | Cohort 1: Change From Baseline in Timed Rise From Floor (From RHS) | Baseline to 12 months | Seconds | MEAN | |
| SECONDARY | Cohort 2 &3: Change From Baseline in HFMSE Total Score at Other Prespecified Timepoints | Baseline to 12 months | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Cohort 2 & 3: Proportion of Patients Achieving Various Magnitudes of Change in HFMSE Score From Baseline | Baseline to 12 months | 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. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group160
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] | Cohort 1 - Monotherapy | -0.1 | Standard Deviation 5.01 | score on a scale | |
| Cohort 1: Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Day 364 (Visit 15) [Month 12] | Cohort 1 - Dual Therapy | 0.0 | Standard Deviation 2.56 | score on a scale | |
| 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 | 0.6 | Standard Deviation 3.5 | score on a scale | |
| 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 3 - Low Dose | 5.3 | Standard Deviation 8.93 | score on a scale | |
| 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 3 - High Dose | 7.1 | Standard Deviation 6.42 | score on a scale | |
| Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | Cohort 1 - Monotherapy | ≥5pt at Day 56 (V4) | 1 | Participants | |
| Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | Cohort 1 - Dual Therapy | ≥5pt at Day 56 (V4) | 1 | Participants | |
| Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | Cohort 1 - Monotherapy | ≥5pt at Day 112 (V6) | 1 | Participants | |
| Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | Cohort 1 - Dual Therapy | ≥5pt at Day 112 (V6) | 1 | Participants | |
| Cohort 1:Change From Baseline in the Revised Hammersmith Scale (RHS) Total Score at Other Prespecified Timepoints | Cohort 1 - Monotherapy | ≥5pt at Day 168 (V8) | 2 | Participants |
1 to 10 of 160Source ClinicalTrials.gov
See all 160 results by groupParticipant flow15
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 - Monotherapy | 11 | |
| Overall Study | STARTED | Cohort 1 - Dual Therapy | 12 | |
| Overall Study | STARTED | Cohort 2 | 15 | |
| Overall Study | STARTED | Cohort 3 - Low Dose | 10 | |
| Overall Study | STARTED | Cohort 3 - High Dose | 10 | |
| Overall Study | COMPLETED | Cohort 1 - Monotherapy | 6 | |
| Overall Study | COMPLETED | Cohort 1 - Dual Therapy | 10 | |
| Overall Study | COMPLETED | Cohort 2 | 13 | |
| Overall Study | COMPLETED | Cohort 3 - Low Dose | 10 | |
| Overall Study | COMPLETED | Cohort 3 - High Dose | 10 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 participant flowAdverse events1,625
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Upper respiratory tract infection | Infections and infestations | Cohort 1 - Monotherapy | 5 | 11 | 9 |
| other | Upper respiratory tract infection | Infections and infestations | Cohort 1 - Dual Therapy | 5 | 12 | 9 |
| other | Upper respiratory tract infection | Infections and infestations | Cohort 2 | 5 | 15 | 10 |
| other | Upper respiratory tract infection | Infections and infestations | Cohort 3 - Low Dose | 4 | 10 | 12 |
| other | Upper respiratory tract infection | Infections and infestations | Cohort 3 - High Dose | 5 | 10 | 10 |
| other | Nasopharyngitis | Infections and infestations | Cohort 1 - Monotherapy | 4 | 11 | 5 |
| other | Nasopharyngitis | Infections and infestations | Cohort 1 - Dual Therapy | 1 | 12 | 5 |
| other | Nasopharyngitis | Infections and infestations | Cohort 2 | 6 | 15 | 13 |
| other | Nasopharyngitis | Infections and infestations | Cohort 3 - Low Dose | 6 | 10 | 23 |
| other | Nasopharyngitis | Infections and infestations | Cohort 3 - High Dose | 5 | 10 | 19 |
1 to 10 of 1,625Source ClinicalTrials.gov
See all 1,625 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 - Monotherapy | 0 | 11 |
| Total, all-cause mortality | Cohort 1 - Dual Therapy | 0 | 12 |
| Total, all-cause mortality | Cohort 2 | 0 | 15 |
| Total, all-cause mortality | Cohort 3 - Low Dose | 0 | 10 |
| Total, all-cause mortality | Cohort 3 - High Dose | 0 | 10 |
| Total, other adverse events | Cohort 1 - Monotherapy | 11 | 11 |
| Total, other adverse events | Cohort 1 - Dual Therapy | 11 | 12 |
| Total, other adverse events | Cohort 2 | 15 | 15 |
| Total, other adverse events | Cohort 3 - Low Dose | 10 | 10 |
| Total, other adverse events | Cohort 3 - High Dose | 9 | 10 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38330285 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03921528
GET https://conductscience.org/api/trials/v1/NCT03921528/results?page=2