Trial · NCT02090959
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT02090959, Phase 3, Terminated, PTC Therapeutics; results first posted 2020-08-11
3 sourcestrials-2026-11-12-0.4.0
Download JSON
Cite this record
ConductScience Library. An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy. Trials, record NCT02090959. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02090959
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
- NCT number
- NCT02090959
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 219 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren
- First posted
- 2014-03-19
- Last updated
- 2020-08-11
- Results first posted
- 2020-08-11
- Limitations and caveats
- The study was terminated early per Sponsor decision due to commercial availability of ataluren.
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 (Day 1) up to 6 weeks post-treatment (Week 150) | An adverse event (AE): any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of AEs: graded per Common Terminology Criteria for AEs (CTCAE), Version 3.0 as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). Drug-related AEs: AEs with possible, probable, unlikely relationship, or unrelated to study drug. Serious AEs (SAEs): 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: an 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 SAEs, regardless of causality is located in Reported AE section. |
| Primary | Number of Participants With Abnormalities in Clinical Laboratory Parameters | Baseline (Day 1) up to 6 weeks post-treatment (Week 150) | Abnormalities in laboratory variables as pre-defined in protocol for safety-monitoring were: Hepatic (Serum alanine aminotransferase \[ALT\]: increase of greater than \[\>\] 150 units/liter \[U/L\] with stable or decrease of creatinine kinese \[CK\]; Serum glutamyl amino transferase \[GGT\] \[U/L\]: Grade 2 \[\>2.5 - 5.0 \* upper limit of normal {ULN}\]), renal (Serum cystatin C miiligrams/liter \[mg/L\] \>1.33 - 2.00 mg/L; Serum blood urea nitrogen \[UREAN\] \[millimoles/liter {mmol/L}\] greater than or equal to \[≥\]1.5 - 3.0 \* ULN; Urine occult blood: 2+ \[Small\], 3+ \[Moderate\], 4+ \[Large\]), and electrolytes (Serum sodium: low \[mmol/L\], Grade 3-4 \[less than {\<}130 mmol/L\]; serum potassium: high \[mmol/L\], Grade 3-4 \[\>6.0 mmol/L\]; and Serum bicarbonate \[mmol/L\]: Grade 2 \[\<16 - 11 mmol/L\]). |
| Secondary | Change From Baseline in 6MWD at Week 144 | Baseline, Week 144 | 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 Time to Stand From Supine Position at Week 144 | Baseline, Week 144 | 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. |
| Secondary | Change From Baseline in Time to Walk/Run 10 Meters at Week 144 | Baseline, Week 144 | 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. |
| Secondary | Change From Baseline in Time to Climb 4 Stairs at Week 144 | Baseline, Week 144 | 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. |
| Secondary | Change From Baseline in Time to Descend 4 Stairs at Week 144 | Baseline, Week 144 | 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. |
| Secondary | Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144 | Baseline, Week 144 | Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for 'raise one's head' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0. |
| Secondary | Change From Baseline in PUL Total Score at Week 144 | Baseline, Week 144 | The PUL was used to assess motor performance of the upper limb. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into shoulder level (4 items), elbow level (9 items), and distal level (8 items) dimensions. Scoring options per item may not be uniform and may vary from 0-1 and 0-6, according to the performance, with higher values corresponding to better performance. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for elbow level, and 24 for distal level. Total score was calculated by adding the 3 level scores, with a maximum global score of 74 (total score range = 0-74). Higher score = better outcome. |
| Secondary | Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144 | Baseline, Week 144 | FVC is a standard pulmonary function test. FVC was defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Percent predicted FVC (in %) = \[(observed FVC)/(predicted FVC)\]\*100. |
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 (Day 1) up to 6 weeks post-treatment (Week 150) | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Abnormalities in Clinical Laboratory Parameters | Baseline (Day 1) up to 6 weeks post-treatment (Week 150) | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in 6MWD at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline 6MWD value at specified timepoint. | meters | MEAN |
| SECONDARY | Change From Baseline in Time to Stand From Supine Position at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint. | seconds | MEAN |
| SECONDARY | Change From Baseline in Time to Walk/Run 10 Meters at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint. | seconds | MEAN |
| SECONDARY | Change From Baseline in Time to Climb 4 Stairs at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint. | seconds | MEAN |
| SECONDARY | Change From Baseline in Time to Descend 4 Stairs at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint. | seconds | MEAN |
| SECONDARY | Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in PUL Total Score at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144 | Baseline, Week 144 | AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint. | percent predicted FVC | MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group82
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 | Any AEs | 202 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | SAEs | 24 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Drug-Related AEs | 44 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | AEs Leading to Withdrawal From Study | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Mild AEs | 87 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Moderate AEs | 71 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Severe AEs | 42 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Ataluren | Life-threatening AEs | 2 | Participants | |
| Number of Participants With Abnormalities in Clinical Laboratory Parameters | Ataluren | 29 | Participants | ||
| Change From Baseline in 6MWD at Week 144 | Ataluren | -98.18 | Standard Deviation 86.604 | meters |
1 to 10 of 82Source ClinicalTrials.gov
See all 82 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 | 219 | |
| Overall Study | As-treated Population | Ataluren | 218 | |
| Overall Study | COMPLETED | Ataluren | 68 | |
| Overall Study | NOT COMPLETED | Ataluren | 151 |
Source ClinicalTrials.gov
Adverse events52
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 | Ear infection | Infections and infestations | Ataluren | 11 | 218 | |
| other | Gastroenteritis | Infections and infestations | Ataluren | 18 | 218 | |
| other | Influenza | Infections and infestations | Ataluren | 23 | 218 | |
| other | Nasopharyngitis | Infections and infestations | Ataluren | 57 | 218 | |
| other | Upper respiratory tract infection | Infections and infestations | Ataluren | 28 | 218 | |
| other | Fall | Injury, poisoning and procedural complications | Ataluren | 48 | 218 | |
| other | Ligament sprain | Injury, poisoning and procedural complications | Ataluren | 19 | 218 | |
| other | Disease progression | General disorders | Ataluren | 56 | 218 | |
| other | Pyrexia | General disorders | Ataluren | 26 | 218 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Ataluren | 18 | 218 |
1 to 10 of 52Source ClinicalTrials.gov
See all 52 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | 0 | 218 |
| Total, other adverse events | Ataluren | 201 | 218 |
| Total, serious adverse events | Ataluren | 24 | 218 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 30135256 | 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/NCT02090959
GET https://conductscience.org/api/trials/v1/NCT02090959/results?page=2