Trial · NCT00847379
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00847379, Phase 2, Terminated, PTC Therapeutics; results first posted 2020-07-15
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD). Trials, record NCT00847379. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00847379
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
- NCT number
- NCT00847379
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 173 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Interventions
- Ataluren
- First posted
- 2009-02-19
- Last updated
- 2020-07-15
- Results first posted
- 2020-07-15
- Limitations and caveats
This study was prematurely terminated by Sponsor per Data Monitoring Committee (DMC) recommendation to discontinue ongoing studies of high-dose ataluren in nmDBMD due to lack of efficacy for the high-dose ataluren.
Source ClinicalTrials.gov
Registered outcomes23
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 (AEs) | Baseline (Week 48 of Study 007) up to Week 102 | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function) and severe (interferes significantly with usual function). 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. TEAE: AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
| Primary | Number of Participants With Clinically Significant Abnormal Laboratory Parameters | Baseline (Week 48 of Study 007) up to Week 102 | Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement. |
| Secondary | Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60 | Baseline (Week 48 of Study 007), Week 60 | The 6MWD test was performed in a 30 meters long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. 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 Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM) | Baseline (Week 48 of Study 007), Week 60 | The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean activity period/day/visit was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that greater than (\>) 2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was less than (\<) 50 percent (%) of the mean active period across all days for that participant's visit. |
| Secondary | Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAM | Baseline (Week 48 of Study 007), Week 60 | The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/day/visit during the active periods was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that \>2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was \<50% of the mean active period across all days for that participant's visit. |
| Secondary | Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAM | Baseline (Week 48 of Study 007), Week 60 | The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/hour during the active periods for the days in a visit was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that \>2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was \<50% of the mean active period across all days for that participant's visit. |
| Secondary | Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM | Baseline (Week 48 of Study 007), Week 60 | SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). The maximum continuous 10-minute, 20-minute, 30-minute, and 60-minute total step counts were computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that \>2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was \<50% of the mean active period across all days for that participant's visit. |
| Secondary | Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 | Baseline (Week 48 of Study 007), Week 60 | SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear SAM for at least 9 consecutive days. SAM was used to record number of strides/minute following each visit. A stride is leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again. Percentage of time during active periods spent at no activity (0 steps/min), low activity (≤15 steps/min), medium activity (16-30 steps/min), and high activity (\>30 steps/min) were computed for each participant. For each day, an active period was defined as first time after 3:00 AM that \>2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was \<50% of the mean active period across all days for that participant's visit. |
| Secondary | Change From Baseline in Time to Stand From Supine Position at Week 60 | Baseline (Week 48 of Study 007), Week 60 | If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported. |
| Secondary | Change From Baseline in Time to Walk/Run 10 Meters at Week 60 | Baseline (Week 48 of Study 007), Week 60 | If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
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 (AEs) | Baseline (Week 48 of Study 007) up to Week 102 | As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Abnormal Laboratory Parameters | Baseline (Week 48 of Study 007) up to Week 102 | As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60 | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints. | meters | MEAN |
| SECONDARY | Change From Baseline in Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM) | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints. | meters | MEAN |
| SECONDARY | Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAM | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints. | steps | MEAN |
| SECONDARY | Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAM | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints. | steps | MEAN |
| SECONDARY | Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories. | steps | MEAN |
| SECONDARY | Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories. | percentage of time | MEAN |
| SECONDARY | Change From Baseline in Time to Stand From Supine Position at Week 60 | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints. | seconds | MEAN |
| SECONDARY | Change From Baseline in Time to Walk/Run 10 Meters at Week 60 | Baseline (Week 48 of Study 007), Week 60 | Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints. | seconds | MEAN |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group351
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 (AEs) | High-Dose Ataluren/High-Dose Ataluren | Any AEs | 52 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Low-Dose Ataluren/High-Dose Ataluren | Any AEs | 49 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Placebo/High-Dose Ataluren | Any AEs | 48 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Overall Participants: High-Dose Ataluren | Any AEs | 149 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | High-Dose Ataluren/High-Dose Ataluren | Drug-related AEs | 10 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Low-Dose Ataluren/High-Dose Ataluren | Drug-related AEs | 10 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Placebo/High-Dose Ataluren | Drug-related AEs | 22 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Overall Participants: High-Dose Ataluren | Drug-related AEs | 42 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | High-Dose Ataluren/High-Dose Ataluren | Serious AEs | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) | Low-Dose Ataluren/High-Dose Ataluren | Serious AEs | 1 | Participants |
1 to 10 of 351Source ClinicalTrials.gov
See all 351 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | High-Dose Ataluren/High-Dose Ataluren | 59 | |
| Overall Study | STARTED | Low-Dose Ataluren/High-Dose Ataluren | 57 | |
| Overall Study | STARTED | Placebo/High-Dose Ataluren | 57 | |
| Overall Study | As-treated Population | High-Dose Ataluren/High-Dose Ataluren | 59 | |
| Overall Study | As-treated Population | Low-Dose Ataluren/High-Dose Ataluren | 57 | |
| Overall Study | As-treated Population | Placebo/High-Dose Ataluren | 57 | |
| Overall Study | COMPLETED | High-Dose Ataluren/High-Dose Ataluren | 0 | |
| Overall Study | COMPLETED | Low-Dose Ataluren/High-Dose Ataluren | 0 | |
| Overall Study | COMPLETED | Placebo/High-Dose Ataluren | 0 | |
| Overall Study | NOT COMPLETED | High-Dose Ataluren/High-Dose Ataluren | 59 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events848
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 | Lymphadenopathy | Blood and lymphatic system disorders | High-Dose Ataluren/High-Dose Ataluren | 0 | 59 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Low-Dose Ataluren/High-Dose Ataluren | 1 | 57 | 1 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Placebo/High-Dose Ataluren | 2 | 57 | 2 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Overall Participants: High-Dose Ataluren | 3 | 173 | 3 |
| other | Anaemia | Blood and lymphatic system disorders | High-Dose Ataluren/High-Dose Ataluren | 0 | 59 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Low-Dose Ataluren/High-Dose Ataluren | 0 | 57 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Placebo/High-Dose Ataluren | 1 | 57 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Overall Participants: High-Dose Ataluren | 1 | 173 | 1 |
| other | Microcytic anaemia | Blood and lymphatic system disorders | High-Dose Ataluren/High-Dose Ataluren | 1 | 59 | 1 |
| other | Microcytic anaemia | Blood and lymphatic system disorders | Low-Dose Ataluren/High-Dose Ataluren | 0 | 57 | 0 |
1 to 10 of 848Source ClinicalTrials.gov
See all 848 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | High-Dose Ataluren/High-Dose Ataluren | ||
| Total, all-cause mortality | Low-Dose Ataluren/High-Dose Ataluren | ||
| Total, all-cause mortality | Placebo/High-Dose Ataluren | ||
| Total, all-cause mortality | Overall Participants: High-Dose Ataluren | ||
| Total, other adverse events | High-Dose Ataluren/High-Dose Ataluren | 53 | 59 |
| Total, other adverse events | Low-Dose Ataluren/High-Dose Ataluren | 51 | 57 |
| Total, other adverse events | Placebo/High-Dose Ataluren | 51 | 57 |
| Total, other adverse events | Overall Participants: High-Dose Ataluren | 155 | 173 |
| Total, serious adverse events | High-Dose Ataluren/High-Dose Ataluren | 0 | 59 |
| Total, serious adverse events | Low-Dose Ataluren/High-Dose Ataluren | 1 | 57 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
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
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/NCT00847379
GET https://conductscience.org/api/trials/v1/NCT00847379/registered?page=2