Trial · NCT05218655
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT05218655, Phase 3, Completed, PTC Therapeutics; results first posted 2025-12-22
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease. Trials, record NCT05218655. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05218655
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
- NCT number
- NCT05218655
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 101 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Inherited Mitochondrial Disease
- Interventions
- Vatiquinone
- First posted
- 2022-02-01
- Last updated
- 2025-12-22
- Other registries' IDs
- eudract:2022-000375-39
- Results first posted
- 2025-12-22
- Limitations and caveats
- The study was terminated early as PTC discontinued clinical development of vatiquinone for this indication based on prior clinical study results.
Source ClinicalTrials.gov
Registered outcomes1
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 30 days after last dose of study drug (956 days) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious adverse events (SAEs) and non-serious AEs. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug and within 30 days of the date of the last dose of treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. |
Source ClinicalTrials.gov
Outcomes1
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 30 days after last dose of study drug (956 days) | Safety population included all participants who received at least 1 dose of vatiquinone in the study. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group1
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) | Vatiquinone | 91 | Participants |
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 | Vatiquinone | 101 | |
| Overall Study | Received At Least 1 Dose of Study Drug | Vatiquinone | 101 | |
| Overall Study | COMPLETED | Vatiquinone | 0 | |
| Overall Study | NOT COMPLETED | Vatiquinone | 101 |
Source ClinicalTrials.gov
Adverse events73
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 | Vatiquinone | 6 | 101 | 7 |
| other | Vomiting | Gastrointestinal disorders | Vatiquinone | 11 | 101 | 15 |
| other | Pyrexia | General disorders | Vatiquinone | 12 | 101 | 24 |
| other | COVID-19 | Infections and infestations | Vatiquinone | 13 | 101 | 14 |
| other | Nasopharyngitis | Infections and infestations | Vatiquinone | 7 | 101 | 9 |
| other | Pneumonia | Infections and infestations | Vatiquinone | 6 | 101 | 9 |
| other | Respiratory tract infection | Infections and infestations | Vatiquinone | 6 | 101 | 10 |
| other | Upper respiratory tract infection | Infections and infestations | Vatiquinone | 14 | 101 | 19 |
| other | Urinary tract infection | Infections and infestations | Vatiquinone | 9 | 101 | 9 |
| other | Gamma-glutamyltransferase increased | Investigations | Vatiquinone | 6 | 101 | 7 |
1 to 10 of 73Source ClinicalTrials.gov
See all 73 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Vatiquinone | 7 | 101 |
| Total, other adverse events | Vatiquinone | 59 | 101 |
| Total, serious adverse events | Vatiquinone | 45 | 101 |
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.
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/NCT05218655
GET https://conductscience.org/api/trials/v1/NCT05218655/events?page=2