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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05933200 | A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD | PHASE3 | ACTIVE NOT_RECRUITING | 69 | — | — | Feb 28, 2023 | Aug 1, 2027 | Apr 7, 2026 | 15 | Czechia, Germany +5 |
| Arm | Type | Description |
|---|---|---|
| Triheptanoin | EXPERIMENTAL | Participants will be given prescriptions for triheptanoin in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. Triheptanoin will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. |
| MCT | ACTIVE_COMPARATOR | Participants will be given prescriptions for MCT in mL/day, which will be divided into approximately 4 administrations per day, with precise instructions for administration. MCT will be titrated up to the first 6 weeks of the study. After titration, the dose is to remain stable for the remainder of the Double-blind Treatment Period. Following the Double-blind Treatment Period, open-label triheptanoin will be provided at participants' current dose during an Open Access Period, if no other options to receive trihepatnoin are available locally. |
| Name | Type | Description |
|---|---|---|
| Triheptanoin | DRUG | Liquid for oral (PO) or enteral feeding tube administration |
| MCT Oil | DIETARY_SUPPLEMENT | Liquid for oral (PO) or enteral feeding tube administration |
Inclusion Criteria for Main Study: * Males and females, from 0 (including newborns) to \< 18 years of age at time of randomization * Confirmed diagnosis of LC-FAOD * Have a caregiver(s) willing and able to assist in all applicable study requirements * Have a legally authorized representative willin...