Trial · NCT04336826
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT04336826, Phase 2, Completed, PTC Therapeutics; results first posted 2024-04-01
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD). Trials, record NCT04336826. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04336826
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- NCT number
- NCT04336826
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Nonsene Mutation Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- First posted
- 2020-04-07
- Last updated
- 2024-04-01
- Other registries' IDs
- eudract:2020-000980-21
- Results first posted
- 2024-04-01
Source ClinicalTrials.gov
Registered outcomes6
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 28 | An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. Serious adverse event (SAE): an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, important medical event. A TEAE was defined as an AE that occurred or worsened while on ataluren (on or after first dose of ataluren) up to 4 weeks after the last dose. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Secondary | Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren | Predose up to 12 hours postdose at Week 24 | |
| Secondary | Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-τ) of Ataluren | Predose up to 12 hours postdose at Week 24 | |
| Secondary | Maximum Concentration (Cmax) of Ataluren | Predose up to 12 hours postdose at Week 24 | |
| Secondary | Time to Maximum Plasma Concentration (Tmax) of Ataluren | Predose up to 12 hours postdose at Week 24 | |
| Secondary | Trough Concentration (Ctrough) of Ataluren | Predose up to 12 hours postdose at Week 24 |
Source ClinicalTrials.gov
Outcomes6
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 28 | Safety analysis set included all participants who received at least 1 dose of ataluren. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren | Predose up to 12 hours postdose at Week 24 | PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | hours*micrograms (μg)/milliliter (mL) | MEAN |
| SECONDARY | Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-τ) of Ataluren | Predose up to 12 hours postdose at Week 24 | PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum. | hours*μg/mL | MEAN |
| SECONDARY | Maximum Concentration (Cmax) of Ataluren | Predose up to 12 hours postdose at Week 24 | PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum. | μg/mL | MEAN |
| SECONDARY | Time to Maximum Plasma Concentration (Tmax) of Ataluren | Predose up to 12 hours postdose at Week 24 | PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum. | hours | MEDIAN |
| SECONDARY | Trough Concentration (Ctrough) of Ataluren | Predose up to 12 hours postdose at Week 24 | PK population included all participants who received at least 1 dose of ataluren and had 1 PK concentration datum. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | μg/mL | MEAN |
Source ClinicalTrials.gov
Results by group6
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 | 5 | Participants | ||
| Area Under the Concentration-Time Curve From Time 0 to 24 Hours (AUC0-24) of Ataluren | Ataluren | 167 | Standard Deviation 23.8 | hours*micrograms (μg)/milliliter (mL) | |
| Area Under the Concentration-Time Curve Between Dosing Interval (AUC0-τ) of Ataluren | Ataluren | NA | Standard Deviation NA | hours*μg/mL | |
| Maximum Concentration (Cmax) of Ataluren | Ataluren | 12.6 | Standard Deviation 3.64 | μg/mL | |
| Time to Maximum Plasma Concentration (Tmax) of Ataluren | Ataluren | 2.08 | Full Range 1.00 to 4.13 | hours | |
| Trough Concentration (Ctrough) of Ataluren | Ataluren | 3.48 | Standard Deviation 3.36 | μg/mL |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 6 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Ataluren | 6 | |
| Overall Study | COMPLETED | Ataluren | 6 | |
| Overall Study | NOT COMPLETED | Ataluren | 0 |
Source ClinicalTrials.gov
Adverse events16
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 | Ataluren | 2 | 6 | |
| other | Teething | Gastrointestinal disorders | Ataluren | 1 | 6 | |
| other | Vomiting | Gastrointestinal disorders | Ataluren | 1 | 6 | |
| other | Pyrexia | General disorders | Ataluren | 1 | 6 | |
| other | Body tinea | Infections and infestations | Ataluren | 1 | 6 | |
| other | Enterovirus infection | Infections and infestations | Ataluren | 1 | 6 | |
| other | Gastroenteritis | Infections and infestations | Ataluren | 2 | 6 | |
| other | Hand-foot-and-mouth disease | Infections and infestations | Ataluren | 1 | 6 | |
| other | Otitis media | Infections and infestations | Ataluren | 1 | 6 | |
| other | Upper respiratory tract infection | Infections and infestations | Ataluren | 1 | 6 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | 0 | 6 |
| Total, other adverse events | Ataluren | 5 | 6 |
| Total, serious adverse events | Ataluren | 0 | 6 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone 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/NCT04336826
GET https://conductscience.org/api/trials/v1/NCT04336826/registered