Trial · NCT01557400
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT01557400, Phase 3, Completed, PTC Therapeutics; results first posted 2020-11-25
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada. Trials, record NCT01557400. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01557400
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
- NCT number
- NCT01557400
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 94 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Interventions
- Ataluren
- First posted
- 2012-03-19
- Last updated
- 2020-11-25
- Results first posted
- 2020-11-25
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Baseline up to Week 246 | A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of the study drug or study treatment, whether or not considered related to the treatment by the Investigator. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), or persistent or significant disability/incapacity not related to nmDBMD. AEs included both SAEs and non-serious AEs. AEs were classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 (CTCAE) and coded using the Medical Dictionary for Regulatory Activities (MedDRA). A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. |
| Secondary | Change From Baseline in 6MWD as Measured by the 6MWT | Baseline, Weeks 48, 96, 144, 192, and 240 | The 6MWD was assessed in participants who were ambulatory using standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. |
| Secondary | Change From Baseline in Physical Function as Measured by the NSAA | Baseline, Weeks 48, 96, 144, 192, and 240 | The NSAA was used to evaluate physical function in participants who were ambulatory at study entry, using standardized procedures. The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score. If fewer than 13 of the 17 activities were performed, the total score was considered missing. If 13 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. The linear score was the linear transformation of the NSAA score to a scale of 0 (worst) to 100 (best). |
| Secondary | Change From Baseline in Time to Stand From Supine Position | Baseline, Weeks 48, 96, 144, 192, and 240 | Time to stand from the supine position to a standing position was assessed in ambulatory participants. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression, a value of 30 seconds was used. |
| Secondary | Change From Baseline in Time to Walk/Run 10 Meters | Baseline, Weeks 48, 96, 144, 192, and 240 | Time to walk/run 10 meters was measured in ambulatory participants. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression, a value of 30 seconds was used. |
| Secondary | Change From Baseline in Pulmonary Function as Measured by Spirometry | Baseline, Weeks 48, 96, 144, 192, and 240 | Pulmonary function parameters of %-predicated FVC, percent-predicted FEV1 (adjusted using ulna length and age), PEF, and PCF was assessed in non-ambulatory participants by using a spirometer. Due to the difficulty in obtaining an accurate standing height measurement in non-ambulatory participants, ulna length and arm span were used as a surrogate measure for height when calculating percent-predicted FVC. |
| Secondary | Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale | Baseline, Weeks 48, 96, 144, 192, and 240 | Activities of daily living after loss of ambulation were measured using the EK scale. The EK scale is an ordinal scale ranging from 0 to 30 points where 0 represents the highest level of independent function and 30 the lowest. The scale consists of 10 categories (each scored 0 to 3), involving different functional domains including 1) ability to use wheelchair, 2) ability to transfer from wheelchair, 3) ability to stand, 4) ability to balance in the wheelchair, 5) ability to move arms, 6) ability to use hands and arms when eating, 7) ability to turn in bed, 8) ability to cough, 9) ability to speak, and 10) physical well-being. The administration of the EK scale consisted of an interview of the participant to capture how he performs the tasks of daily life (as described by Categories 1 to 9) and how he perceives his wellbeing (as described by Category 10). The interviewer observed the participant and assigned the final score for the tasks that could be observed in the clinic. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Baseline up to Week 246 | All enrolled participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in 6MWD as Measured by the 6MWT | Baseline, Weeks 48, 96, 144, 192, and 240 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable 6MWT data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry. | change in meters | MEAN |
| SECONDARY | Change From Baseline in Physical Function as Measured by the NSAA | Baseline, Weeks 48, 96, 144, 192, and 240 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable NSAA data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Time to Stand From Supine Position | Baseline, Weeks 48, 96, 144, 192, and 240 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable time to stand from supine data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry. | seconds | MEAN |
| SECONDARY | Change From Baseline in Time to Walk/Run 10 Meters | Baseline, Weeks 48, 96, 144, 192, and 240 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable data for time to walk/run 10 meters. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry. | seconds | MEAN |
| SECONDARY | Change From Baseline in Pulmonary Function as Measured by Spirometry | Baseline, Weeks 48, 96, 144, 192, and 240 | All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable spirometry data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry. | liters | MEAN |
| SECONDARY | Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale | Baseline, Weeks 48, 96, 144, 192, and 240 | All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable EK scale data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group47
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | TEAEs | 91 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Treatment-Emergent SAEs | 31 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | TEAEs Related to Study Treatment | 26 | Participants | |
| Change From Baseline in 6MWD as Measured by the 6MWT | Ataluren | Week 48 | -41.71 | Standard Deviation 48.172 | change in meters |
| Change From Baseline in 6MWD as Measured by the 6MWT | Ataluren | Week 96 | -76.80 | Standard Deviation 70.781 | change in meters |
| Change From Baseline in 6MWD as Measured by the 6MWT | Ataluren | Week 144 | -97.57 | Standard Deviation 83.761 | change in meters |
| Change From Baseline in 6MWD as Measured by the 6MWT | Ataluren | Week 192 | -109.37 | Standard Deviation 96.238 | change in meters |
| Change From Baseline in 6MWD as Measured by the 6MWT | Ataluren | Week 240 | -134.16 | Standard Deviation 94.716 | change in meters |
| Change From Baseline in Physical Function as Measured by the NSAA | Ataluren | Week 48 | -2.9 | Standard Deviation 3.08 | units on a scale |
| Change From Baseline in Physical Function as Measured by the NSAA | Ataluren | Week 96 | -7.4 | Standard Deviation 5.61 | units on a scale |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 94 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Ataluren | 94 | |
| Overall Study | COMPLETED | Ataluren | 37 | |
| Overall Study | NOT COMPLETED | Ataluren | 57 |
Source ClinicalTrials.gov
Adverse events247
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 | Vomiting | Gastrointestinal disorders | Ataluren | 28 | 94 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Ataluren | 14 | 94 | |
| other | Diarrhoea | Gastrointestinal disorders | Ataluren | 13 | 94 | |
| other | Constipation | Gastrointestinal disorders | Ataluren | 9 | 94 | |
| other | Nausea | Gastrointestinal disorders | Ataluren | 7 | 94 | |
| other | Abdominal pain | Gastrointestinal disorders | Ataluren | 6 | 94 | |
| other | Nasopharyngitis | Infections and infestations | Ataluren | 40 | 94 | |
| other | Gastroenteritis | Infections and infestations | Ataluren | 19 | 94 | |
| other | Upper respiratory tract infection | Infections and infestations | Ataluren | 19 | 94 | |
| other | Influenza | Infections and infestations | Ataluren | 9 | 94 |
1 to 10 of 247Source ClinicalTrials.gov
See all 247 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | 2 | 94 |
| Total, other adverse events | Ataluren | 88 | 94 |
| Total, serious adverse events | Ataluren | 31 | 94 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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.
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GET https://conductscience.org/api/trials/v1/NCT01557400/results?page=2