Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02352896|
Recruitment Status : Active, not recruiting
First Posted : February 2, 2015
Last Update Posted : October 30, 2020
|Condition or disease||Intervention/treatment||Phase|
|Leigh Syndrome||Drug: EPI-743||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Long-Term Safety and Efficacy Evaluation of Vatiquinone (EPI-743) in Children With Leigh Syndrome|
|Actual Study Start Date :||January 31, 2014|
|Estimated Primary Completion Date :||December 31, 2021|
|Estimated Study Completion Date :||December 31, 2021|
Participants will receive EPI-743 at a dose of 15 milligrams/kilogram (mg/kg) up to a total 200 mg 3 times daily (TID) orally with meal or by using nasogastric (NG) or gastrostomy feeding tubes with liquid food for at least 36 months.
EPI-743 100 milligrams/milliliter (mg/mL) oral solution will be administered per dose and schedule specified in the arm.
Other Name: Vincerinone, Vatiquinone
- Disease Severity as Measured by Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 Score [ Time Frame: Baseline up to Month 36 ]NPMDS is a validated scale to assess the mitochondrial disease progression.
- Number of Participants With Dose-Limiting Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Month 36 ]
- Bayley Scales of Infant Development-III Score (Participants Age 0-3) [ Time Frame: Baseline up to Month 24 ]Effect of EPI-743 on neurodevelopment will be measured by Bayley Scales of Infant Development-III score.
- Wechsler Scale of Intelligence and Movement Assessment Battery Score for Children (Participants Age 4-18) [ Time Frame: Baseline up to Month 24 ]Effect of EPI-743 on neuromuscular function will be assessed by Wechsler Scale of Intelligence and Movement Assessment Battery score.
- Barry-Albright Dystonia Scale Score [ Time Frame: Baseline up to Month 24 ]Neuromuscular function will be assessed by Barry-Albright Dystonia Scale score.
- Gross Motor Function Measure [ Time Frame: Baseline up to Month 24 ]Neuromuscular function will be assessed by gross motor function measure.
- Awake Oxygen Saturation Levels [ Time Frame: Baseline up to Month 24 ]Effect of EPI-743 on respiratory function will be measured by awake oxygen saturation levels.
- Total Ventilator Days and Total Intensive Care Unit Days [ Time Frame: Baseline up to Month 24 ]
- Number of Participants With Pneumonia Episodes and Tracheostomy [ Time Frame: Baseline up to Month 24 ]
- Total Number of Mortalities, Medical Encounters, Hospitalizations, and Hospital Days [ Time Frame: Baseline up to Month 24 ]
- Health-Related Quality of Life as Measured by NPMDS Section 4 Score [ Time Frame: Baseline up to Month 24 ]
- Glutathione Cycle Biomarkers Level [ Time Frame: Baseline up to Month 24 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02352896
|United States, California|
|Stanford University, California, United States, 94305|
|United States, Ohio|
|Akron Children's Hospital|
|Akron, Ohio, United States, 44308|
|United States, Texas|
|Baylor College of Medicine|
|Houston, Texas, United States, 77030|
|United States, Washington|
|Seattle Children's Hospital|
|Seattle, Washington, United States, 98105|
|Principal Investigator:||Bruce Cohen, MD||Akron Children's Hospital|