Trial · NCT03703882
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
NCT03703882, Phase 3, Completed, Catabasis Pharmaceuticals; results first posted 2022-06-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy. Trials, record NCT03703882. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03703882
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
- NCT number
- NCT03703882
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 131 participants, actual
- Lead sponsor
- Catabasis Pharmaceuticals
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Edasalonexent, Placebo
- First posted
- 2018-10-12
- Last updated
- 2022-06-21
- Results first posted
- 2022-06-21
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in North Star Ambulatory Assessment (NSAA) | Baseline (Day 1) to Week 52 | To assess change from baseline in North Star Ambulatory Assessment(NSAA) Total Score at Wk52. NSAA is clinician-reported outcome instrument designed to measure ambulatory function in males with Duchenne muscular dystrophy(DMD). Patients asked to perform 17 different functional activities,including 10MWT,rising from sit to stand,standing on one leg,climbing \& descending a step,stand from supine, lifting the head, standing on heels, \& jumping. Each function activity will be scored as0=(unable to achieve independently),scored as1=(modified method but achieves goal independent of physical assistance from another),or scored as2=(no obvious modification of activity)or "Not Scored". If NSAA test was performed \& any of the individual items are scored as "not scored"(i.e, for reasons unrelated to patients physical capabilities), corresponding total score will be set to missing. Sum of 17 scores will be used to form an ordinal total score(range 0-34).Higher scores imply better functional status |
| Secondary | Change From Baseline in 10-meter Walk/Run Test | Baseline (Day 1) to Week 52 | To assess the changes from baseline to Week 52 on the 10-meter walk/run test (10MWT). For timed function tests (TFTs), the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1=Unable to walk independently 2=Unable to walk independently but can walk with knee-ankle foot orthoses or support from a person 3=Highly adapted wide based lordotic gait. Cannot increase walking speed 4=Moderately adapted gait. Can pick up speed but cannot run 5=Able to pick up speed, but runs with a double stance phase, i.e. cannot achieve both feet off the ground 6=Runs and gets both feet off the ground (with no double stance phase) |
| Secondary | Change From Baseline in Time to Stand From Supine | Baseline (Day 1) to Week 52 | To assess the change from baseline in the stand from supine speed at Week 52. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1 or 2 (from a 6-point scale). 1 = Unable to stand from supine, even with use of a chair, 2 = Assisted Gowers - requires furniture for assist in arising from supine to full upright posture (no time to be recorded) 3=Rolls over, stands up with both hands "climbing up" the legs to achieve full upright posture 4=Rolls over, stands up with 1 hand support on leg 5=Rolls to the side and stands up with one or both hands on the floor to start to rise but does not touch legs 6=Stands up without rolling over or using hands on legs or floor |
| Secondary | Change From Baseline in 4-stair Climb | Baseline (Day 1) to Week 52 | To assess the change from baseline to Week 52 on the 4-Stair Climb. For timed function tests (TFTs) , the time will be set to 12 seconds and the speed to 0 if the TFT assessment meets the following TFT grading criteria. Grade of 1(from a 6-point scale)1=Unable to climb 4 standard stairs(no time recorded) 2=Climbs 4 standard stairs "marking time"(climbs one foot at a time, with both feet on a step before moving to next step), uses both arms on one or both handrails or uses 1 handrail and the other arm pushes on the leg 3=Climbs 4 standard stairs "marking time", using one arm on one handrail or one hand pushing on leg or body 4=Climbs 4 standard stairs "marking time", not needing handrail and not using hands to push on leg 5=Climbs 4 standard stairs alternating feet, needs handrail/s for support or uses arms to push on the leg or body 6=Climbs 4 standard stairs alternating feet, not needing handrail support or using arm to push on the leg |
| Secondary | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Up to Week 52 | Adverse events that occurred from the time of the administration of the first dose of investigational product (IP) through the end of the safety follow-up were considered treatment-emergent AEs (TEAEs). Serious adverse event (SAE). |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in North Star Ambulatory Assessment (NSAA) | Baseline (Day 1) to Week 52 | Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in 10-meter Walk/Run Test | Baseline (Day 1) to Week 52 | Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Time to Stand From Supine | Baseline (Day 1) to Week 52 | Full Analysis population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in 4-stair Climb | Baseline (Day 1) to Week 52 | Full Analysis Population: All patients in the Randomized Population who received at least 1 dose of study drug and provided at least 1 valid post Baseline NSAA efficacy assessment). | score on a scale | MEAN |
| SECONDARY | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Up to Week 52 | Safety population: All patients who received at least 1 dose of study drug, with patients analyzed based on the actual study treatment received. This included the set of patients who were assigned the same treatment as their randomized sibling. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in North Star Ambulatory Assessment (NSAA) | Dose 1 | -1.5 | Standard Deviation 4.41 | score on a scale | |
| Change From Baseline in North Star Ambulatory Assessment (NSAA) | Placebo | -1.8 | Standard Deviation 3.81 | score on a scale | |
| Change From Baseline in 10-meter Walk/Run Test | Dose 1 | -0.0058 | Standard Deviation 0.03010 | score on a scale | |
| Change From Baseline in 10-meter Walk/Run Test | Placebo | -0.0093 | Standard Deviation 0.02538 | score on a scale | |
| Change From Baseline in Time to Stand From Supine | Dose 1 | -0.0389 | Standard Deviation 0.06728 | score on a scale | |
| Change From Baseline in Time to Stand From Supine | Placebo | -0.0459 | Standard Deviation 0.06171 | score on a scale | |
| Change From Baseline in 4-stair Climb | Dose 1 | -0.0220 | Standard Deviation 0.08920 | score on a scale | |
| Change From Baseline in 4-stair Climb | Placebo | -0.0392 | Standard Deviation 0.07352 | score on a scale | |
| Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Dose 1 | Treatment Emergent Adverse Event (TEAEs) | 85 | participants | |
| Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Placebo | Treatment Emergent Adverse Event (TEAEs) | 41 | participants |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Dose 1 | 88 | |
| Overall Study | STARTED | Placebo | 43 | |
| Overall Study | COMPLETED | Dose 1 | 85 | |
| Overall Study | COMPLETED | Placebo | 37 | |
| Overall Study | NOT COMPLETED | Dose 1 | 3 | |
| Overall Study | NOT COMPLETED | Placebo | 6 |
Source ClinicalTrials.gov
Adverse events96
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 | Diarrhoea | Gastrointestinal disorders | Dose 1 | 54 | 88 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 12 | 43 | |
| other | Vomiting | Gastrointestinal disorders | Dose 1 | 29 | 88 | |
| other | Vomiting | Gastrointestinal disorders | Placebo | 11 | 43 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Dose 1 | 15 | 88 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 9 | 43 | |
| other | Nausea | Gastrointestinal disorders | Dose 1 | 7 | 88 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 5 | 43 | |
| other | Constipation | Gastrointestinal disorders | Dose 1 | 6 | 88 | |
| other | Constipation | Gastrointestinal disorders | Placebo | 3 | 43 |
1 to 10 of 96Source ClinicalTrials.gov
See all 96 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Dose 1 | 0 | 88 |
| Total, all-cause mortality | Placebo | 0 | 43 |
| Total, other adverse events | Dose 1 | 85 | 88 |
| Total, other adverse events | Placebo | 41 | 43 |
| Total, serious adverse events | Dose 1 | 1 | 88 |
| Total, serious adverse events | Placebo | 1 | 43 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34120912 | 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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Dose 1 vs Placebo: deaths from any cause | Trial comparisons |
| Dose 1 vs Placebo: serious adverse events | Trial comparisons |
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/NCT03703882
GET https://conductscience.org/api/trials/v1/NCT03703882/events?page=2