A Study to Evaluate Efficacy and Safety of Vatiquinone fo... | NCT04378075

ID	Type	Description	Link
2020-002100-39	EudraCT Number

Label	Type	Description	Intervention Names
Vatiquinone	Experimental	15 milligrams/kilogram (mg/kg) if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, 3 times per day (TID) or up to 72 weeks	Drug: Vatiquinone Other: Placebo
Placebo	Placebo Comparator	Vatiquinone-matching placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.	Other: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Vatiquinone	Drug	Vatiquinone will be administered per the treatment arm description.	Vatiquinone

Measure	Description	Time Frame
Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period	The 28-day motor seizure frequency in the double-blind period was calculated as the (number of motor seizures)/ (the number of valid days where motor seizure count information is present) * 28 within the double-blind period. The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.	Baseline to Week 24

Measure	Description	Time Frame
Change From Baseline to Week 24 in Number of Disease-Related Hospitalization Days Per 28 Days in Double-Blind Period	The disease-related hospitalization days per 28 days in the double-blind period was calculated as the (number of disease-related hospitalizations)/(the number of days within the double-blind period) * 28. The baseline hospitalization used the 28 days observations immediately prior to treatment start date for this calculation.

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Signed informed consent form.
Participant or parent/legal guardian is able and willing to complete seizure diaries for the duration of the study.
Genetic confirmation of inherited mitochondrial disease with associated epilepsy phenotype (Alpers/polymerase subunit gamma [POLG], Leigh syndrome, mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes [MELAS]), or other genetically confirmed mitochondrial disease secondary to mitochondrial mutations (Pontocerebellar Hypoplasia Type 6 [PCH6], nuclear DNA RARS2 mutation) or myoclonic epilepsy with ragged red fibers (MERRF, mitochondrial DNA [mtDNA] mitochondrially encoded tRNA lysine [MT-TK] mutation).
Despite ongoing treatment with at least 2 antiepileptic drugs:
- have ≥6 observed motor seizures occurring during the 28 days prior to the baseline visit (Day 0).
- have ≥2 observed motor seizures in the first 14 days and ≥2 in the second 14 days of the Run-in period (Day -14).
- do not have a consecutive 20-day seizure free period.
- have at least 80% of seizure diary data.
Documented medical history of epilepsy associated with mitochondrial disease for at least 6 months prior to screening except for participants who are <2 years of age at the time of screening (participants <2 years of age can be considered for enrollment if all other screening criteria are met due to the potential for rapid progression in these participants).
Consent to abstain from non-approved therapies for 30 days prior to the screening visit and for the duration of the study.
Stable dose regimen of antiepileptic therapies 30 days prior to the screening visit.
Stable regimen of dietary supplements 30 days prior and, if on a ketogenic diet, stable ketogenic diet 90 days prior to the screening visit and for duration of the study.
Electroencephalogram (EEG) at screening or historical EEG up to 6 months prior to screening for diagnostic confirmation of seizures.

Exclusion Criteria:

Allergy to vatiquinone or sesame oil.
Aspartate transaminase (AST) or alanine transaminase (ALT) ≥3 × upper level of normal (ULN) at time of screening.
International normalized ratio (INR) >ULN at time of screening.
Serum creatinine ≥1.5 × ULN at time of screening.
Participation in another interventional clinical trial 60 days prior to randomization or for the duration of this clinical trial
Previously received vatiquinone.
Concomitant treatment with drug(s) that have not received regulatory agency approval for the treatment of mitochondrial diseases and use of artisanal (non-Epidiolex cannabidiol) cannabidiol therapies.
Concomitant treatment with idebenone.
Ongoing treatment with strong cytochrome P450 (CYP) inhibitors such as itraconazole or strong CYP inducers such as rifampin. Treatment with these agents must be completed at least 4 weeks prior to enrollment.During the study, participants should not use grapefruit/grapefruit juice or St John's wort extract.
Pregnant or lactating participants or those male or female sexually active participants who are unwilling to comply with proper birth control methods from the time consent is signed until 30 days after treatment discontinuation. Females of childbearing potential must have a negative pregnancy test at screening and during the baseline visit (Day 0).
Comorbidities that may confound study results (for example, fat malabsorption syndrome, other mitochondrial disorders) in the opinion of the investigator.

Name	Affiliation	Role
Vinay Penematsa, MD	PTC Therapeutics	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
University of California		San Diego	California	92123	United States
Stanford University

ID	Title	Description
FG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 milligrams (mg)/kilogram (kg) if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, 3 times per day (TID) for up to 24 weeks during the double-blind period.
FG001	Double-blind Period: Placebo

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	May 27, 2022	Mar 4, 2026

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period

The 28-day motor seizure frequency in the double-blind period was calculated as the (number of motor seizures)/ (the number of valid days where motor seizure count information is present) * 28 within the double-blind period. The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Median

95% Confidence Interval

percent change

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts
Participants	OG00034 OG00134

Title

Denominators

Categories

Title	Measurements
	OG000-12.74± -28.67(-28.67 to 9.42) OG001-0.33± -28.97(-28.97 to 17.06)

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		ANCOVA		= 0.173		Median Difference (Final Values)	-8.31			2-Sided	95	-31.3	16.4					Other

Secondary

Change From Baseline to Week 24 in Number of Disease-Related Hospitalization Days Per 28 Days in Double-Blind Period

The disease-related hospitalization days per 28 days in the double-blind period was calculated as the (number of disease-related hospitalizations)/(the number of days within the double-blind period) * 28.

The baseline hospitalization used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Median

95% Confidence Interval

days

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts
Participants

Secondary

Change From Baseline to Week 72 in Number of Disease-Related Hospitalization Days Per 28 Days in Overall Period

The disease-related hospitalization days per 28 days in the overall period was calculated as the (number of disease-related hospitalizations)/(the number of days within the overall treatment period) * 28.

The baseline hospitalization used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Median

95% Confidence Interval

days

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Units	Counts
Participants

Secondary

Change From Baseline to Week 24 in Occurrence/Recurrence of Status Epilepticus Per 28 Days in Double-blind Period

The status epilepticus per 28 Days in the double-blind period was calculated as the (number of status epilepticus incidences)/(the number of days in the double-blind period) * 28.

The baseline status epilepticus incidences used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Mean

Standard Deviation

status epilepticus per 28 days

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts
Participants

Secondary

Change From Baseline to Week 72 in Occurrence/Recurrence of Status Epilepticus Per 28 Days in Overall Period

The status epilepticus per 28 Days in the overall period was calculated as the (number of status epilepticus incidences)/(the number of days in the overall period) * 28.

The baseline status epilepticus incidences used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Mean

Standard Deviation

status epilepticus per 28 days

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Units	Counts
Participants

Secondary

Number of Participants With Disease-Related In-Patient Hospitalizations in Double-Blind Period

In-patient hospitalization per 28 days in the double-blind period was calculated as the (number of in-patient hospitalization)/(the number of days in the double-blind period) * 28.

The baseline in-patient hospitalization used the 28 days observations immediately prior to treatment start date for this calculation. Number of participants with in-patient hospitalizations for either seizure or epilepticus per 28 days in double-blind period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts

Secondary

Number of Participants With Disease-Related In-Patient Hospitalizations in Overall Period

In-patient hospitalization per 28 days in the overall period was calculated as the (number of in-patient hospitalization)/(the number of days in the overall period) * 28.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Units

Secondary

Number of Participants With Disease-Related Emergency Room Visits in Double-Blind Period

Disease-related emergency room visits per 28 days in the double-blind period was calculated as the (number of disease-related emergency room visits)/(the number of days in the double-blind period) * 28. The baseline disease-related emergency room visits used the 28 days observations immediately prior to treatment start date for this calculation. Number of participants with disease-related emergency room visits for either seizure or epilepticus per 28 days in double-blind period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts

Secondary

Number of Participants With Disease-Related Emergency Room Visits in Overall Period

Disease-related emergency room visits per 28 days in the overall period was calculated as the (number of disease-related emergency room visits)/(the number of days in the overall period) * 28.

The baseline disease-related emergency room visits used the 28 days observations immediately prior to treatment start date for this calculation. Number of participants with disease-related emergency room visits for either seizure or epilepticus per 28 days in overall period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Secondary

Number of Disease-Related In-Patient Hospitalizations in Double-Blind Period

In-patient hospitalization per 28 days in the double-blind period was calculated as the (number of in-patient hospitalization)/(the number of days in the double-blind period) * 28.

The baseline in-patient hospitalization used the 28 days observations immediately prior to treatment start date for this calculation. Number of participants with number of in-patient hospitalizations for either seizure or epilepticus per 28 days in double-blind period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts

Secondary

Number of Disease-Related In-Patient Hospitalizations in Overall Period

In-patient hospitalization per 28 days in the overall period was calculated as the (number of in-patient hospitalization)/(the number of days in the overall period) * 28.

The baseline in-patient hospitalization used the 28 days observations immediately prior to treatment start date for this calculation. Number of participants with number in-patient hospitalizations for either seizure or epilepticus per 28 days in overall period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Units	Counts

Secondary

Number of Disease-Related Emergency Room Visits in Double-Blind Period

The baseline disease-related emergency room visits used the 28 days observations immediately prior to treatment start date for this calculation. Number of participants with number of emergency room visits for either seizure or epilepticus per 28 days in double-blind period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts

Secondary

Number of Disease-Related Emergency Room Visits in Overall Period

Disease-related emergency room visits per 28 days in the overall period was calculated as the (number of disease-related emergency room visits)/(the number of days in the overall period) * 28.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Units

Secondary

Percent Change From Baseline to Week 24 in Total Seizure Frequency Per 28 Days in Double-Blind Period

The total seizure frequency per 28 days in the double-blind period was calculated as the (number of total seizures)/(the number of valid days where total seizure count information is present) * 28 within the double-blind period.

The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Median

95% Confidence Interval

percent change

Baseline, Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts
Participants

Secondary

Percent Change From Baseline to Week 72 in Total Seizure Frequency Per 28 Days in Overall Period

Overall period was defined as the period from the first dosing date of investigational product (IP) during double-blind period to the end of study (double-blind + open-label period).

The 28 day total seizure frequency in the overall period was calculated as the (number of total seizures)/(the number of valid days where total seizure count information is present) * 28 within the overall period.

The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Median

95% Confidence Interval

percent change

Baseline, Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Secondary

Number of Participants Taking Rescue Medications in the Double-blind Period

Number of participants taking rescue medications for epilepsy in double-blind period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts
Participants	OG000

Secondary

Number of Participants Taking Rescue Medications in the Overall Period

Number of participants taking rescue medications for epilepsy in overall period are reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Units	Counts
Participants	OG000

Secondary

Change From Baseline to Week 24 in Health-Related Quality of Life as Measured by the Care-Related Quality of Life of Informal Caregivers (CarerQoL-7D) Questionnaire Score in Double-blind Period

The CarerQol-7D consists of 5 negative and 2 positive dimensions of providing informal care. The negative dimensions are relational problems, mental health problems, problems combining daily activities with care, financial problems and physical health problems because of providing informal care. The 2 positive dimensions are fulfilment from caregiving and support with lending care. For each dimension, there are 3 possible responses: no, some and a lot. Utility tariffs for CarerQol have been developed to calculate a weighted sum score of CarerQol-7D from the responses on the 7 dimensions, ranging from 0 (worst imaginable caregiving situation) to 100 (best imaginable caregiving situation), for which discrete choice experiments were used. Higher sum scores reflect better care-related quality of life.

ITT population included all randomized participants who received at least 1 dose of treatment. 'Overall number of participants analyzed' = participants evaluable for this endpoint.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo

Secondary

Change From Baseline to Week 72 in Health-Related Quality of Life as Measured by the CarerQoL-7D Questionnaire Score in Overall Period

ITT population included all randomized participants who received at least 1 dose of treatment. 'Overall number of participants analyzed' = participants evaluable for this endpoint.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone

Secondary

Number of Participants With Motor Seizure Clusters in Double-Blind Period

Seizure clusters were defined by "too many to count" entries in the seizure diaries. The motor seizure clusters per 28 days in the double-blind period was calculated as the (number of motor seizure clusters)/(the number of valid days where motor seizure clusters count information was present) * 28 within the double-blind period.

The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts

Secondary

Number of Participants With Motor Seizure Clusters in Overall Period

Seizure clusters were defined by "too many to count" entries in the seizure diaries. The motor seizure clusters per 28 days in the overall period was calculated as the (number of motor seizure clusters)/(the number of valid days where motor seizure clusters count information was present) * 28 within the overall period.

The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 72

ID	Title	Description
OG000	Overall Period: Vatiquinone/Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 72 weeks.
OG001	Overall Period: Placebo/Vatiquinone	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks followed by vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 48 weeks.

Units

Secondary

Number of Participants With >30%, 30% to -30%, < -30% to -60%, < -60% to -100% Reduction in Motor Seizures Per 28 Days During the Double-blind Period

Number of participants whose motor seizure frequency reduction per 28 days was more than the specified percentage compared to baseline were reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts
Participants	OG000

Secondary

Number of Participants With >30%, 30% to -30%, < -30% to -60%, < -60% to -100% Reduction in Total Seizures Per 28 Days During the Double-blind Period

Number of participants whose total seizure frequency reduction per 28 days was more than the specified percentage compared to baseline were reported.

ITT population included all randomized participants who received at least 1 dose of treatment.

Posted

Count of Participants

Participants

Baseline to Week 24

ID	Title	Description
OG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
OG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.

Units	Counts
Participants	OG000

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Bradycardia	Cardiac disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG0030 events0 affected29 at risk
Vomiting	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0011 events1 affected34 at risk EG0024 events3 affected34 at risk EG003
Death	General disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Hypothermia	General disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0011 events1 affected34 at risk EG0020 events0 affected34 at risk EG003
COVID-19	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected34 at risk EG0010 events0 affected34 at risk EG0025 events5 affected34 at risk EG003
Gastroenteritis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0011 events1 affected34 at risk EG0021 events1 affected34 at risk EG003
Gastrointestinal infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Influenza	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Lower respiratory tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0011 events1 affected34 at risk EG0021 events1 affected34 at risk EG003
Metapneumovirus infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0011 events1 affected34 at risk EG0020 events0 affected34 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Pneumonia	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected34 at risk EG0012 events2 affected34 at risk EG0022 events2 affected34 at risk EG003
Pneumonia viral	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Respiratory syncytial virus infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0011 events1 affected34 at risk EG0020 events0 affected34 at risk EG003
Rhinovirus infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0002 events1 affected34 at risk EG0012 events1 affected34 at risk EG0024 events3 affected34 at risk EG003
Sepsis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Urinary tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Urine output decreased	Investigations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Seizure	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0008 events4 affected34 at risk EG0010 events0 affected34 at risk EG00215 events7 affected34 at risk EG003
Status epilepticus	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0002 events1 affected34 at risk EG0012 events2 affected34 at risk EG0023 events2 affected34 at risk EG003
Urinary retention	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0011 events1 affected34 at risk EG0020 events0 affected34 at risk EG003
Acute pulmonary oedema	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Hypoxia	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Pneumonitis aspiration	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Respiratory arrest	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Respiratory distress	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0011 events1 affected34 at risk EG0021 events1 affected34 at risk EG003
Respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0002 events1 affected34 at risk EG0010 events0 affected34 at risk EG0024 events3 affected34 at risk EG003
Respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Excessive granulation tissue	Skin and subcutaneous tissue disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0011 events1 affected34 at risk EG0020 events0 affected34 at risk EG003
Cardiac failure	Cardiac disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Constipation	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Dyspepsia	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Disease progression	General disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Bronchitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Bronchitis viral	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Ear infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Enterovirus infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Norovirus infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Pneumonia aspiration	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Pneumonia pseudomonal	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Respiratory tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Septic shock	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Upper respiratory tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Vascular device infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Joint dislocation	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Metabolic acidosis	Metabolism and nutrition disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Mitochondrial cytopathy	Metabolism and nutrition disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Change in seizure presentation	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Febrile convulsion	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Agitation	Psychiatric disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Inappropriate affect	Psychiatric disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Acute respiratory distress syndrome	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Atelectasis	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Increased upper airway secretion	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003
Pneumothorax	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Tracheal stenosis	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Hypotension	Vascular disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0021 events1 affected34 at risk EG003

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Vomiting	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0007 events7 affected34 at risk EG0010 events0 affected34 at risk EG00214 events10 affected34 at risk EG0035 events1 affected29 at risk
Pyrexia	General disorders	MedDRA 26.0	Systematic Assessment	EG0006 events4 affected34 at risk EG0011 events1 affected34 at risk EG00212 events9 affected34 at risk EG003
Bronchitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0013 events2 affected34 at risk EG0022 events2 affected34 at risk EG003
COVID-19	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0023 events3 affected34 at risk EG003
Conjunctivitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Ear infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0024 events4 affected34 at risk EG003
Gastroenteritis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0011 events1 affected34 at risk EG0020 events0 affected34 at risk EG003
Hordeolum	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0013 events2 affected34 at risk EG0020 events0 affected34 at risk EG003
Influenza	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0011 events1 affected34 at risk EG0022 events2 affected34 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected34 at risk EG0010 events0 affected34 at risk EG0024 events3 affected34 at risk EG003
Pharyngitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0013 events2 affected34 at risk EG0020 events0 affected34 at risk EG003
Pneumonia	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0002 events1 affected34 at risk EG0011 events1 affected34 at risk EG0022 events1 affected34 at risk EG003
Pneumonia aspiration	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0012 events2 affected34 at risk EG0020 events0 affected34 at risk EG003
Respiratory tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0015 events3 affected34 at risk EG0022 events2 affected34 at risk EG003
Respiratory tract infection viral	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Rhinitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0024 events2 affected34 at risk EG003
Sinusitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0024 events4 affected34 at risk EG003
Upper respiratory tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0003 events2 affected34 at risk EG0014 events3 affected34 at risk EG00210 events5 affected34 at risk EG003
Urinary tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0004 events3 affected34 at risk EG0012 events2 affected34 at risk EG00211 events4 affected34 at risk EG003
Viral upper respiratory tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected34 at risk EG0010 events0 affected34 at risk EG0024 events4 affected34 at risk EG003
Fall	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Tooth fracture	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0023 events2 affected34 at risk EG003
Hypokalaemia	Metabolism and nutrition disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0020 events0 affected34 at risk EG003
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Change in seizure presentation	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0013 events2 affected34 at risk EG0020 events0 affected34 at risk EG003
Headache	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Seizure	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0004 events4 affected34 at risk EG0011 events1 affected34 at risk EG00210 events8 affected34 at risk EG003
Somnolence	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0011 events1 affected34 at risk EG0022 events2 affected34 at risk EG003
Agitation	Psychiatric disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0023 events3 affected34 at risk EG003
Insomnia	Psychiatric disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0013 events3 affected34 at risk EG0020 events0 affected34 at risk EG003
Intermenstrual bleeding	Reproductive system and breast disorders	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0022 events2 affected34 at risk EG003
Respiratory disorder	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0010 events0 affected34 at risk EG0023 events3 affected34 at risk EG003
Blister	Skin and subcutaneous tissue disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected34 at risk EG0012 events2 affected34 at risk EG0020 events0 affected34 at risk EG003
Eczema	Skin and subcutaneous tissue disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected34 at risk EG0010 events0 affected34 at risk EG0025 events3 affected34 at risk EG003

ID	Term
D028361	Mitochondrial Diseases
D000069279	Drug Resistant Epilepsy
D007888	Leigh Disease
C538525	Mitochondrial encephalopathy
D017241	MELAS Syndrome
C548074	Pontocerebellar Hypoplasia Type 6
D002549	Diffuse Cerebral Sclerosis of Schilder
D017243	MERRF Syndrome
D012640	Seizures
D013226	Status Epilepticus
D009410	Nerve Degeneration

ID	Term
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases
D004827	Epilepsy
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D015323	Pyruvate Metabolism, Inborn Errors
D002239	Carbohydrate Metabolism, Inborn Errors
D017237	Mitochondrial Encephalomyopathies
D017240	Mitochondrial Myopathies
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases
D059345	Cerebral Small Vessel Diseases
D002561	Cerebrovascular Disorders
D009468	Neuromuscular Diseases
D014652	Vascular Diseases
D002318	Cardiovascular Diseases
D020278	Demyelinating Autoimmune Diseases, CNS
D020274	Autoimmune Diseases of the Nervous System
D056784	Leukoencephalopathies
D003711	Demyelinating Diseases
D001327	Autoimmune Diseases
D007154	Immune System Diseases
D020191	Myoclonic Epilepsies, Progressive
D004831	Epilepsies, Myoclonic
D004829	Epilepsy, Generalized
D000073376	Epileptic Syndromes
D009461	Neurologic Manifestations
D012816	Signs and Symptoms
D013568	Pathological Conditions, Signs and Symptoms
D010335	Pathologic Processes

ID	Title	Description
BG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.
BG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.
BG002	Total	Total of all reporting groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Double-blind Period: Vatiquinone	Participants received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for up to 24 weeks during the double-blind period.	3	34	18	34	26	34
EG001	Double-blind Period: Placebo	Participants received vatiquinone-matched placebo, administered orally, TID for up to 24 weeks during the double-blind period.	0	34	9	34	20	34
EG002	On-Vatiquinone Period: Vatiquinone/Vatiquinone	Participants who received vatiquinone for 24 weeks in the double-blind period continued to receive vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for 48 weeks during the long-term extension period.	5	34	26	34	30	34
EG003	On-Vatiquinone Period: Placebo/Vatiquinone	Participants who received placebo for 24 weeks in the double-blind period, received vatiquinone 15 mg/kg if body weight <13 kg, and 200 mg if body weight ≥13 kg, administered orally, TID for 48 weeks during the long-term extension period.	2	29	16	29	19	29