Trial Outcomes & Findings for A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease (NCT NCT05218655)
NCT ID: NCT05218655
Last Updated: 2025-12-22
Results Overview
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'.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
101 participants
Primary outcome timeframe
Baseline (Day 1) up to 30 days after last dose of study drug (956 days)
Results posted on
2025-12-22
Participant Flow
A total of 103 participants were screened, of which 101 participants were enrolled in the study and received at least 1 dose of vatiquinone.
Participant milestones
| Measure |
Vatiquinone
Participants received vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
|
|---|---|
|
Overall Study
STARTED
|
101
|
|
Overall Study
Received At Least 1 Dose of Study Drug
|
101
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
101
|
Reasons for withdrawal
| Measure |
Vatiquinone
Participants received vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
|
|---|---|
|
Overall Study
Adverse Event
|
6
|
|
Overall Study
Sponsor's decision
|
85
|
|
Overall Study
Death
|
1
|
|
Overall Study
Lost to Follow-up
|
3
|
|
Overall Study
Withdrawal by Subject
|
3
|
|
Overall Study
Other than specified
|
3
|
Baseline Characteristics
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Baseline characteristics by cohort
| Measure |
Vatiquinone
n=101 Participants
Participants received vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
|
|---|---|
|
Age, Continuous
|
15.5 years
STANDARD_DEVIATION 11.56 • n=18 Participants
|
|
Sex: Female, Male
Female
|
49 Participants
n=18 Participants
|
|
Sex: Female, Male
Male
|
52 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race · American Indian/Alaska Native
|
1 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
8 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race · Black/African American
|
2 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
83 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
1 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race · Unknown
|
1 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Race · Missing
|
5 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Hispanic or Latino
|
9 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Not Hispanic or Latino
|
86 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Not Reported
|
3 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Unknown
|
2 Participants
n=18 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Missing
|
1 Participants
n=18 Participants
|
PRIMARY outcome
Timeframe: Baseline (Day 1) up to 30 days after last dose of study drug (956 days)Population: Safety population included all participants who received at least 1 dose of vatiquinone in the study.
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'.
Outcome measures
| Measure |
Vatiquinone
n=101 Participants
Participants received vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
|
91 Participants
|
Adverse Events
Vatiquinone
Serious events: 45 serious events
Other events: 59 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Vatiquinone
n=101 participants at risk
Participants received vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube TID.
|
|---|---|
|
Hepatobiliary disorders
Hepatic failure
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Infections and infestations
Bronchitis
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Infections and infestations
COVID-19
|
3.0%
3/101 • Number of events 3 • 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.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Cardiac disorders
Atrial fibrillation
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Cardiac disorders
Cardiomyopathy
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Cardiac disorders
Tachycardia
|
0.99%
1/101 • Number of events 2 • 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.
|
|
Congenital, familial and genetic disorders
Fanconi syndrome
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Endocrine disorders
Adrenal insufficiency
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Constipation
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Erosive oesophagitis
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Oesophagitis
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Pancreatitis
|
0.99%
1/101 • Number of events 2 • 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.
|
|
Gastrointestinal disorders
Pancreatitis acute
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Pneumoperitoneum
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Vomiting
|
4.0%
4/101 • Number of events 4 • 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.
|
|
General disorders
Death
|
0.99%
1/101 • Number of events 1 • 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.
|
|
General disorders
Hypothermia
|
0.99%
1/101 • Number of events 1 • 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.
|
|
General disorders
Generalised oedema
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Infections and infestations
Influenza
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Infections and infestations
Lower respiratory tract infection
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Infections and infestations
Pneumonia
|
9.9%
10/101 • Number of events 11 • 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.
|
|
Infections and infestations
Pneumonia aspiration
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Infections and infestations
Pseudomonas infection
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Infections and infestations
Respiratory tract infection
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Infections and infestations
Rhinovirus infection
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Infections and infestations
Sepsis
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Infections and infestations
Streptococcal infection
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Infections and infestations
Tracheitis
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Infections and infestations
Urinary tract infection
|
3.0%
3/101 • Number of events 3 • 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.
|
|
Infections and infestations
Urosepsis
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Injury, poisoning and procedural complications
Fall
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Injury, poisoning and procedural complications
Postoperative ileus
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Investigations
SARS-CoV-2 test positive
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Metabolism and nutrition disorders
Mitochondrial cytopathy
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Nervous system disorders
Autonomic nervous system imbalance
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Nervous system disorders
Change in seizure presentation
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Nervous system disorders
Epilepsy
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Nervous system disorders
Myoclonus
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Nervous system disorders
Optic neuritis
|
0.99%
1/101 • Number of events 2 • 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.
|
|
Nervous system disorders
Seizure
|
2.0%
2/101 • Number of events 3 • 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.
|
|
Nervous system disorders
Status epilepticus
|
3.0%
3/101 • Number of events 4 • 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.
|
|
Renal and urinary disorders
Acute kidney injury
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
10.9%
11/101 • Number of events 16 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Hypercapnia
|
0.99%
1/101 • Number of events 3 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary amyloidosis
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
2.0%
2/101 • Number of events 2 • 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.
|
|
Surgical and medical procedures
Tracheostomy
|
0.99%
1/101 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Intestinal obstruction
|
0.99%
1/101 • Number of events 1 • 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.
|
Other adverse events
| Measure |
Vatiquinone
n=101 participants at risk
Participants received vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube TID.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
5.9%
6/101 • Number of events 7 • 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.
|
|
Gastrointestinal disorders
Vomiting
|
10.9%
11/101 • Number of events 15 • 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.
|
|
General disorders
Pyrexia
|
11.9%
12/101 • Number of events 24 • 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.
|
|
Infections and infestations
COVID-19
|
12.9%
13/101 • Number of events 14 • 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.
|
|
Infections and infestations
Nasopharyngitis
|
6.9%
7/101 • Number of events 9 • 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.
|
|
Infections and infestations
Pneumonia
|
5.9%
6/101 • Number of events 9 • 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.
|
|
Infections and infestations
Respiratory tract infection
|
5.9%
6/101 • Number of events 10 • 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.
|
|
Infections and infestations
Upper respiratory tract infection
|
13.9%
14/101 • Number of events 19 • 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.
|
|
Infections and infestations
Urinary tract infection
|
8.9%
9/101 • Number of events 9 • 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.
|
|
Investigations
Gamma-glutamyltransferase increased
|
5.9%
6/101 • Number of events 7 • 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.
|
|
Nervous system disorders
Seizure
|
8.9%
9/101 • Number of events 9 • 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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the Sponsor for review. The Sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER