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A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

NCT04378075 · Status: TERMINATED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 68

Last updated 2026-03-31

Study results available
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Summary

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.

Conditions

  • Mitochondrial Diseases
  • Drug Resistant Epilepsy
  • Leigh Disease
  • Leigh Syndrome
  • Mitochondrial Encephalopathy (MELAS)
  • Pontocerebellar Hypoplasia Type 6 (PCH6)
  • Alpers Disease
  • Alpers Syndrome

Interventions

DRUG

Vatiquinone

Vatiquinone will be administered per the treatment arm description.

OTHER

Placebo

Vatiquinone-matching placebo will be administered per the treatment arm description

Sponsors & Collaborators

Principal Investigators

  • Vinay Penematsa, MD · PTC Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Max Age
20 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-28
Primary Completion
2023-03-18
Completion
2023-12-27
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France
  • Italy
  • Japan
  • Poland
  • Spain
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04378075 on ClinicalTrials.gov