A Phase 2/3 interventional study of Vatiquinone and Placebo in Mitochondrial Diseases, Drug Resistant Epilepsy and Leigh Disease, sponsored by PTC Therapeutics. Terminated at 27 sites in 9 countries. Open to participants aged Up to 20 Years. Per ClinicalTrials.gov, last updated 2026-03-31.
Sponsored by PTC Therapeutics · Phase 2/3, Interventional, and Treatment
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.
182 studies on the registry are indexed under Mitochondrial Diseases; 54 are open to participants now.
This study's enrollment of 68 is above the median of 30 across 100 interventional studies indexed under Mitochondrial Diseases.
Browse Mitochondrial Diseases studies →PTC Therapeutics is the lead sponsor of 65 studies on the registry; 3 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 14 (78%) have results posted.
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Despite ongoing treatment with at least 2 antiepileptic drugs:
Exclusion Criteria:
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
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
Vatiquinone will be administered per the treatment arm description.
Also known as: PTC743, EPI-743
Vatiquinone-matching placebo will be administered per the treatment arm description
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.
Time frame: Baseline to Week 24
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.
Time frame: Baseline to Week 24
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.
Time frame: Baseline to Week 72
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.
Time frame: Baseline to Week 24
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.
Time frame: Baseline to Week 72
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.
Time frame: Baseline to Week 24
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. 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 overall period are reported.
Time frame: Baseline to Week 72
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.
Time frame: Baseline to Week 24
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.
Time frame: Baseline to Week 72
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.
Time frame: Baseline to Week 24
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.
Time frame: Baseline to Week 72
Number of 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 number of emergency room visits for either seizure or epilepticus per 28 days in double-blind period are reported.
Time frame: Baseline to Week 24
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. 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 overall period are reported.
Time frame: Baseline to Week 72
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.
Time frame: Baseline, Week 24
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.
Time frame: Baseline, Week 72
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.
Time frame: Baseline to Week 24
Number of Participants Taking Rescue Medications in the Overall Period
Number of participants taking rescue medications for epilepsy in overall period are reported.
Time frame: Baseline to Week 72
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.
Time frame: Baseline, Week 24
Change From Baseline to Week 72 in Health-Related Quality of Life as Measured by the CarerQoL-7D Questionnaire Score in Overall 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.
Time frame: Baseline, Week 72
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.
Time frame: Baseline to Week 24
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.
Time frame: Baseline to Week 72
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.
Time frame: Baseline to Week 24
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.
Time frame: Baseline to Week 24
| Milestone | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | Long-term Extension: Vatiquinone/Vatiquinone | Long-term Extension: Placebo/Vatiquinone |
|---|---|---|---|---|
| Started | 34 | 34 | 0 | 0 |
| Received at least 1 dose of study drug | 34 | 34 | 0 | 0 |
| Completed | 28 | 29 | 0 | 0 |
| Not completed | 6 | 5 | 0 | 0 |
| Withdrew: Adverse event | 2 | 2 | 0 | 0 |
| Withdrew: Death | 3 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 0 | 1 | 0 | 0 |
| Withdrew: Protocol violation | 1 | 0 | 0 | 0 |
| Withdrew: Other than specified | 0 | 2 | 0 | 0 |
| Milestone | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | Long-term Extension: Vatiquinone/Vatiquinone | Long-term Extension: Placebo/Vatiquinone |
|---|---|---|---|---|
| Started | 0 | 0 | 28 | 29 |
| Completed | 0 | 0 | 16 | 18 |
| Not completed | 0 | 0 | 12 | 11 |
| Withdrew: Death | 0 | 0 | 2 | 2 |
| Withdrew: Other than specified | 0 | 0 | 9 | 7 |
| Withdrew: Sponsor's decision | 0 | 0 | 1 | 1 |
| Withdrew: Lack of efficacy | 0 | 0 | 0 | 1 |
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.
| percent change | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period | -12.74 ± -28.67 | -0.33 ± -28.97 |
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.
| days | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Change From Baseline to Week 24 in Number of Disease-Related Hospitalization Days Per 28 Days in Double-Blind Period | 0 ± 0 | 0 ± 0 |
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.
| days | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Change From Baseline to Week 72 in Number of Disease-Related Hospitalization Days Per 28 Days in Overall Period | 0.363 (0 to 1.304) | 0.166 (0 to 0.515) |
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.
| status epilepticus per 28 days | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Change From Baseline to Week 24 in Occurrence/Recurrence of Status Epilepticus Per 28 Days in Double-blind Period | 0.039 ± 0.9564 | -0.026 ± 1.1083 |
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.
| status epilepticus per 28 days | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Change From Baseline to Week 72 in Occurrence/Recurrence of Status Epilepticus Per 28 Days in Overall Period | 0.036 ± 0.6444 | -0.102 ± 1.0094 |
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.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Number of Participants With Disease-Related In-Patient Hospitalizations in Double-Blind Period | 6 | 2 |
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 in-patient hospitalizations for either seizure or epilepticus per 28 days in overall period are reported.
| Participants | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Number of Participants With Disease-Related In-Patient Hospitalizations in Overall Period | 11 | 3 |
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.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Number of Participants With Disease-Related Emergency Room Visits in Double-Blind Period | 9 | 4 |
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.
| Participants | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Number of Participants With Disease-Related Emergency Room Visits in Overall Period | 14 | 5 |
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.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| 0 Hospitalization | 28 | 32 |
| 1 Hospitalization | 4 | 1 |
| 2 Hospitalizations | 1 | 0 |
| 7 Hospitalizations | 0 | 1 |
| 10 Hospitalizations | 1 | 0 |
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.
| Participants | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| 0 Hospitalization | 23 | 31 |
| 1 Hospitalization | 7 | 1 |
| 2 Hospitalizations | 0 | 1 |
| 5 Hospitalizations | 1 | 0 |
| 6 Hospitalizations | 1 | 0 |
| 7 Hospitalizations | 0 | 1 |
| 8 Hospitalizations | 1 | 0 |
| 10 Hospitalizations | 1 | 0 |
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 number of emergency room visits for either seizure or epilepticus per 28 days in double-blind period are reported.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| 0 Visit | 25 | 30 |
| 1 Visit | 7 | 4 |
| 2 Visits | 1 | 0 |
| 10 Visits | 1 | 0 |
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 number of emergency room visits for either seizure or epilepticus per 28 days in overall period are reported.
| Participants | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| 0 Visit | 20 | 29 |
| 1 Visit | 8 | 4 |
| 2 Visits | 2 | 1 |
| 6 Visits | 2 | 0 |
| 8 Visits | 1 | 0 |
| 10 Visits | 1 | 0 |
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.
| percent change | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Percent Change From Baseline to Week 24 in Total Seizure Frequency Per 28 Days in Double-Blind Period | -14.27 ± -34.52 | -5.73 ± -31.59 |
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.
| percent change | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Percent Change From Baseline to Week 72 in Total Seizure Frequency Per 28 Days in Overall Period | -17.03 (-42.38 to 0) | -7.52 (-35.46 to 17.00) |
Number of participants taking rescue medications for epilepsy in double-blind period are reported.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Number of Participants Taking Rescue Medications in the Double-blind Period | 24 | 22 |
Number of participants taking rescue medications for epilepsy in overall period are reported.
| Participants | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Number of Participants Taking Rescue Medications in the Overall Period | 24 | 23 |
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.
| units on a scale | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| 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 | -10.38 ± 18.207 | -5.25 ± 10.112 |
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.
| units on a scale | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Change From Baseline to Week 72 in Health-Related Quality of Life as Measured by the CarerQoL-7D Questionnaire Score in Overall Period | -0.95 ± 14.235 | -3.14 ± 13.675 |
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.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| Number of Participants With Motor Seizure Clusters in Double-Blind Period | 19 | 25 |
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.
| Participants | Overall Period: Vatiquinone/Vatiquinone | Overall Period: Placebo/Vatiquinone |
|---|---|---|
| Number of Participants With Motor Seizure Clusters in Overall Period | 21 | 26 |
Number of participants whose motor seizure frequency reduction per 28 days was more than the specified percentage compared to baseline were reported.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| >30% | 7 | 9 |
| 30% to -30% | 17 | 14 |
| < -30% to -60% | 7 | 7 |
| < -60% to -100% | 3 | 4 |
Number of participants whose total seizure frequency reduction per 28 days was more than the specified percentage compared to baseline were reported.
| Participants | Double-blind Period: Vatiquinone | Double-blind Period: Placebo |
|---|---|---|
| >30% | 7 | 7 |
| 30% to -30% | 13 | 15 |
| < -30% to -60% | 10 | 9 |
| < -60% to -100% | 4 | 3 |
Collected over Baseline up to Week 77. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Double-blind Period: Vatiquinone | 3/34 (8.8%) | 18/34 (52.9%) | 26/34 (76.5%) |
| Double-blind Period: Placebo | 0/34 (0%) | 9/34 (26.5%) | 20/34 (58.8%) |
| On-Vatiquinone Period: Vatiquinone/Vatiquinone | 5/34 (14.7%) | 26/34 (76.5%) | 30/34 (88.2%) |
| On-Vatiquinone Period: Placebo/Vatiquinone | 2/29 (6.9%) | 16/29 (55.2%) | 19/29 (65.5%) |
| Event | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | On-Vatiquinone Period: Vatiquinone/Vatiquinone | On-Vatiquinone Period: Placebo/Vatiquinone |
|---|---|---|---|---|
| SeizureNervous system disorders | 4/34 | 0/34 | 7/34 | 1/29 |
| COVID-19Infections and infestations | 2/34 | 0/34 | 5/34 | 0/29 |
| PneumoniaInfections and infestations | 2/34 | 2/34 | 2/34 | 4/29 |
| VomitingGastrointestinal disorders | 1/34 | 1/34 | 3/34 | 1/29 |
| Rhinovirus infectionInfections and infestations | 1/34 | 1/34 | 3/34 | 0/29 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 1/34 | 0/34 | 3/34 | 2/29 |
| Pneumonia aspirationInfections and infestations | 0/34 | 0/34 | 2/34 | 2/29 |
| Status epilepticusNervous system disorders | 1/34 | 2/34 | 2/34 | 0/29 |
| DeathGeneral disorders | 1/34 | 0/34 | 1/34 | 1/29 |
| InfluenzaInfections and infestations | 1/34 | 0/34 | 1/34 | 1/29 |
| Event | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | On-Vatiquinone Period: Vatiquinone/Vatiquinone | On-Vatiquinone Period: Placebo/Vatiquinone |
|---|---|---|---|---|
| VomitingGastrointestinal disorders | 7/34 | 0/34 | 10/34 | 1/29 |
| PyrexiaGeneral disorders | 4/34 | 1/34 | 9/34 | 1/29 |
| Upper respiratory tract infectionInfections and infestations | 2/34 | 3/34 | 5/34 | 7/29 |
| SeizureNervous system disorders | 4/34 | 1/34 | 8/34 | 1/29 |
| Ear infectionInfections and infestations | 1/34 | 0/34 | 4/34 | 0/29 |
| SinusitisInfections and infestations | 1/34 | 0/34 | 4/34 | 0/29 |
| Urinary tract infectionInfections and infestations | 3/34 | 2/34 | 4/34 | 2/29 |
| ContusionInjury, poisoning and procedural complications | 2/34 | 0/34 | 4/34 | 0/29 |
| InfluenzaInfections and infestations | 1/34 | 1/34 | 2/34 | 3/29 |
| COVID-19Infections and infestations | 1/34 | 0/34 | 3/34 | 2/29 |
Intent-to-treat (ITT) population included all randomized participants who received at least 1 dose of treatment.
| Age, Continuous(years) | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | Total |
|---|---|---|---|
| Mean | 8.7 ± 5.34 | 6.6 ± 4.84 | 7.6 ± 5.17 |
| Sex: Female, Male(Participants) | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | Total |
|---|---|---|---|
| Female | 14 | 20 | 34 |
| Male | 20 | 14 | 34 |
| Ethnicity (NIH/OMB)(Participants) | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 2 | 1 | 3 |
| Not Hispanic or Latino | 26 | 30 | 56 |
| Unknown or Not Reported | 6 | 3 | 9 |
| Race/Ethnicity, Customized(Participants) | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | Total |
|---|---|---|---|
| Race — American Indian/Alaska Native | 0 | 1 | 1 |
| Race — Asian | 3 | 9 | 12 |
| Race — Black/African American | 1 | 3 | 4 |
| Race — White/Caucasian | 24 | 20 | 44 |
| Race — Other | 1 | 0 | 1 |
| Race — Not Reported | 5 | 1 | 6 |
| Number of Observable Motor Seizures per 28 Days(motor seizures/28 days) | Double-blind Period: Vatiquinone | Double-blind Period: Placebo | Total |
|---|---|---|---|
| Median | 112.5 (6 to 3139) | 237.0 (15 to 2918) | 164.0 (64.5 to 391.0) |
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PTC Therapeutics