Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02352896 | Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome | PHASE2 | COMPLETED | 30 | — | — | Jan 31, 2014 | Oct 30, 2023 | Oct 23, 2024 | 4 | United States |
| NCT01721733 | Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome | PHASE2 | COMPLETED | 35 | — | — | Oct 31, 2012 | May 31, 2015 | Aug 31, 2020 | 4 | United States |
NPMDS is a validated scale to assess the mitochondrial disease progression.
Change from baseline to six months will be compared between subjects in active treatment group and placebo group
| Arm | Type | Description |
|---|---|---|
| EPI-743 | EXPERIMENTAL | 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. |
| Placebo | PLACEBO_COMPARATOR | Each patient will receive a volume of placebo based on weight |
| EPI-743 15 mg/kg | ACTIVE_COMPARATOR | Each subjects dose will be based on their weight. 15 mg/kg with a maximum dose of 200 mg per dose, t.i.d., will be administered in this treatment arm. |
| EPI-743 5 mg/kg | ACTIVE_COMPARATOR | Each subjects dose will be based on their weight. 5 mg/kg with a maximum dose of 100 mg per dose, t.i.d., will be administered in this treatment arm. |
| Name | Type | Description |
|---|---|---|
| EPI-743 | DRUG | EPI-743 100 milligrams/milliliter (mg/mL) oral solution will be administered per dose and schedule specified in the arm. |
| Placebo | DRUG | - |
| EPI-743 15 mg/kg | DRUG | - |
| EPI-743 5 mg/kg | DRUG | - |
Inclusion Criteria: 1. Diagnosis of Leigh syndrome with genetic confirmation 2. Completion of EPI743-12-002 protocol and initiation of treatment within 14 days of Month 12 visit in EPI743-12-002 study 3. Participant or participant's guardian able to consent and comply with protocol requirements 4. ...