Trial · NCT02352896
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT02352896, Phase 2, Completed, PTC Therapeutics
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome. Trials, record NCT02352896. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02352896
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
- NCT number
- NCT02352896
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Leigh Syndrome
- Interventions
- EPI-743
- First posted
- 2015-02-02
- Last updated
- 2024-10-23
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Disease Severity as Measured by Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 Score | Baseline up to Month 36 | NPMDS is a validated scale to assess the mitochondrial disease progression. |
| Primary | Number of Participants With Dose-Limiting Serious Adverse Events (SAEs) | Baseline up to Month 36 | |
| Secondary | Bayley Scales of Infant Development-III Score (Participants Age 0-3) | Baseline up to Month 24 | Effect of EPI-743 on neurodevelopment will be measured by Bayley Scales of Infant Development-III score. |
| Secondary | Wechsler Scale of Intelligence and Movement Assessment Battery Score for Children (Participants Age 4-18) | Baseline up to Month 24 | Effect of EPI-743 on neuromuscular function will be assessed by Wechsler Scale of Intelligence and Movement Assessment Battery score. |
| Secondary | Barry-Albright Dystonia Scale Score | Baseline up to Month 24 | Neuromuscular function will be assessed by Barry-Albright Dystonia Scale score. |
| Secondary | Gross Motor Function Measure | Baseline up to Month 24 | Neuromuscular function will be assessed by gross motor function measure. |
| Secondary | Awake Oxygen Saturation Levels | Baseline up to Month 24 | Effect of EPI-743 on respiratory function will be measured by awake oxygen saturation levels. |
| Secondary | Total Ventilator Days and Total Intensive Care Unit Days | Baseline up to Month 24 | |
| Secondary | Number of Participants With Pneumonia Episodes and Tracheostomy | Baseline up to Month 24 | |
| Secondary | Total Number of Mortalities, Medical Encounters, Hospitalizations, and Hospital Days | Baseline up to Month 24 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
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.
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/NCT02352896
GET https://conductscience.org/api/trials/v1/NCT02352896/registered