Trial · NCT00759876
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT00759876, Phase 2, Terminated, PTC Therapeutics; results first posted 2020-10-29
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD). Trials, record NCT00759876. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00759876
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
- NCT number
- NCT00759876
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 36 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- First posted
- 2008-09-25
- Last updated
- 2020-10-29
- Results first posted
- 2020-10-29
Source ClinicalTrials.gov
Registered outcomes19
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 89 | TEAE: any untoward medical occurrence or undesirable event(s) that begins or worsens following administration of the study drug, whether or not considered related to the treatment by the Investigator. Severity of an adverse event (AE) was classified as: mild (does not interfere with usual function), moderate (interferes with usual function; may require medical intervention), severe (interferes significantly with usual function; likely require medical intervention), life-threatening, and fatal. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
| Secondary | Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT) | Baseline, Week 48 and Week 60 | The 6MWD was assessed in participants who were ambulatory using standardized procedures. Participants were not permitted to use assistive devices during the 6MWD test. Only the results of the participant's best valid test at each visit were included in the analysis. The mean change from baseline in the distance the participant walked is reported. |
| Secondary | Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests | Baseline, Week 48 and Week 60 | Timed function tests included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. Timed function tests were assessed in ambulatory participants. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used. Test results were set to missing in the analysis for participants who could not perform the tests for reasons other than PD (for example, bone fracture). |
| Secondary | Change From Baseline in Standing From Supine Position as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used for standing from supine position: 1) Unable to stand from supine, even with use of a chair. 2) Assisted Gowers, requires furniture to rise from supine to full upright posture. 3) Full Gowers, rolls over, stands with both hands "climbing up" legs to above knees to achieve full upright posture. 4) Half Gowers, rolls over, stands up with 1 hand support on lower legs. 5) Rolls to side and/or stands with 1 or both hands on floor to start to rise but does not touch legs. 6) Stands without rolling over or using hands. Increases from baseline are indicative of improving ability to perform functional task. If a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture). |
| Secondary | Change From Baseline in Run/Walk 10-Meters as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to run/walk 10-meters: 1) Unable to walk independently. 2) Unable to walk independently but can walk with knee-ankle-foot orthoses (KAFOs) or with 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 (that is, cannot achieve both feet off the ground). 6) Runs and gets both feet off the ground (with no double-stance phase). At the visit, if a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture). |
| Secondary | Change From Baseline in Ascending 4 Stairs as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to ascend 4 stairs: 1) Unable to climb 4 standard stairs. 2) Climbs 4 standard stairs "marking time" (climbs 1 foot at a time, with both feet on a step before moving to next step), using both arms on 1 or both handrails. 3) Climbs 4 standard stairs "marking time" using 1 arm on 1 handrail. 4) Climbs 4 standard stairs "marking time" not needing handrail. 5) Climbs 4 standard stairs alternating feet, needs handrail for support. 6) Climbs 4 standard stairs alternating feet, not needing handrail support. At the visit, if a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture). |
| Secondary | Change From Baseline in Descending 4 Stairs as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to descend 4 stairs: 1) Unable to descend 4 standard stairs. 2) Descends 4 standard stairs "marking time" (climbs 1 foot at a time, with both feet on a step before moving to next step), using both arms on 1 or both handrails. 3) Descends 4 standard stairs "marking time", using 1 arm on 1 handrail. 4) Descends 4 standard stairs "marking time", not needing handrail. 5) Descends 4 standard stairs alternating feet in both directions, needs handrail for support. 6) Descends 4 standard stairs alternating feet, not needing handrail support. At the visit, if a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture). |
| Secondary | Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry | Baseline, Week (Wk) 48 and Wk 60 | Upper extremity myometry was performed using a hand-held dynamometer following standardized procedures. With this system, evaluators judged the strength of each muscle using an 11-point descriptive scoring system. From the individual muscle-group scores, a total composite score was derived. Bilateral assessments were done, and 3 measurements were recorded from each muscle group on each side, when possible. The best of the 3 replicates was used in the analysis. An increase from Baseline is reflective of increased muscle strength, whereas a decrease from Baseline is reflective of decreased muscle strength. Participants who became unable to perform a myometry test because of disease progression were assigned a value of 0 for each visit at which the participant was no longer able to perform the test. Test results were set to missing in the analysis for participants who could not perform the tests for reasons other than disease progression (for example, bone fracture). |
| Secondary | Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 | Baseline, Week 48 and Week 60 | Heart rate was measured with a Polar RS400 heart rate monitor, which consists of a transmitter strap worn around the chest and a wristwatch receiver. The monitor produces a digital text file with 1 value per minute that represents the mean heart rate for that minute. Mean heart rate values were collected before, during, and after the 6MWT. The participant rested for 5 minutes in a sitting position before the 6MWT, and the mean heart rate for the last minute of this rest period was obtained and documented as the resting heart rate. During the 6MWT, the mean heart rate was collected and documented as the active heart rate. After completing the 6MWT and resting for 3 minutes, the mean heart rate for 1 minute was obtained and documented as the recovery heart rate. |
| Secondary | Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task | Baseline, Week 48 and Week 60 | The digit span task is a 2-part (forward and backward) test in which a series of digits (3 to 9) were presented to participant in an auditory format only. For forward condition, the participant was to repeat digits back in the order they were presented. For backward condition, the participant was to reverse the order of presentation. Maximum score for each part (digit forward and digit backward) of task is 14; participants received a score of 2 points if they passed both trials, score of 1 point if they passed only 1 trial, and score of 0 points if they failed both trials. A raw score of total number of correct forward and backward responses was age-normalized by subtracting corresponding mean and dividing by corresponding standard deviation of a reference population for that age. For each forward and backward result, the resulting Z score was transformed into percentile rank of normal distribution. Change from Baseline in number of digits recalled forward and backward is reported. |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
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 89 | All enrolled participants who received at least 1 dose of study drug (As-Treated Population). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT) | Baseline, Week 48 and Week 60 | 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 walk ≥75 meters unassisted at screening. | meters | MEAN |
| SECONDARY | Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests | Baseline, Week 48 and Week 60 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable data for the applicable timed function test. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening. | seconds | MEAN |
| SECONDARY | Change From Baseline in Standing From Supine Position as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for standing from supine. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening. | scores on a scale | MEAN |
| SECONDARY | Change From Baseline in Run/Walk 10-Meters as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for running/walking 10 meters. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening. | scores on a scale | MEAN |
| SECONDARY | Change From Baseline in Ascending 4 Stairs as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for ascending 4 stairs. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening. | scores on a scale | MEAN |
| SECONDARY | Change From Baseline in Descending 4 Stairs as Assessed by Method Scores During Timed Function Tests | Baseline, Week 48 and Week 60 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for descending 4 stairs. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening. | scores on a scale | MEAN |
| SECONDARY | Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry | Baseline, Week (Wk) 48 and Wk 60 | All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable myometry data. | pounds | MEAN |
| SECONDARY | Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 | Baseline, Week 48 and Week 60 | All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable heart rate data. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening. | beeps per minute | MEAN |
| SECONDARY | Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task | Baseline, Week 48 and Week 60 | All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable verbal memory and attention data. | digits recalled | MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group195
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 | At least 1 TEAE | 36 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Mild TEAE | 9 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Moderate TEAE | 16 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Severe TEAE | 10 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Life-Threatening TEAE | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Fatal TEAE | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Related TEAE | 28 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Serious TEAE | 6 | Participants | |
| Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT) | Ataluren | Baseline | 367.8 | Standard Deviation 107.3 | meters |
| Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT) | Ataluren | Change from Baseline at Week 48 | -80.4 | Standard Deviation 59.5 | meters |
1 to 10 of 195Source ClinicalTrials.gov
See all 195 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 36 | |
| Overall Study | As-Treated Population | Ataluren | 36 | |
| Overall Study | Evaluable Population | Ataluren | 35 | |
| Overall Study | Ambulatory Evaluable Population | Ataluren | 24 | |
| Overall Study | COMPLETED | Ataluren | 0 | |
| Overall Study | NOT COMPLETED | Ataluren | 36 |
Source ClinicalTrials.gov
Adverse events76
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 | Myocardial fibrosis | Cardiac disorders | Ataluren | 2 | 36 | |
| other | Tachycardia | Cardiac disorders | Ataluren | 2 | 36 | |
| other | Abdominal pain | Gastrointestinal disorders | Ataluren | 6 | 36 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Ataluren | 11 | 36 | |
| other | Constipation | Gastrointestinal disorders | Ataluren | 3 | 36 | |
| other | Diarrhoea | Gastrointestinal disorders | Ataluren | 8 | 36 | |
| other | Flatulence | Gastrointestinal disorders | Ataluren | 12 | 36 | |
| other | Nausea | Gastrointestinal disorders | Ataluren | 3 | 36 | |
| other | Stomach discomfort | Gastrointestinal disorders | Ataluren | 3 | 36 | |
| other | Vomiting | Gastrointestinal disorders | Ataluren | 24 | 36 |
1 to 10 of 76Source ClinicalTrials.gov
See all 76 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | ||
| Total, other adverse events | Ataluren | 36 | 36 |
| Total, serious adverse events | Ataluren | 6 | 36 |
Source ClinicalTrials.gov
Papers2
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.
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Digit Span Test | Secondary outcome |
Source ConductCognition
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
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