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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06773026 | Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) | PHASE2 | RECRUITING | 24 | — | — | Jun 30, 2025 | Jul 1, 2027 | Jul 11, 2025 | 1 | United States |
| Arm | Type | Description |
|---|---|---|
| 4.0 g/m2/day BID sodium phenylbutyrate | EXPERIMENTAL | Up to 24 subjects (12: ages 4-9 years old; 12:10 years of age and older) will be randomized to take 4.0 g/m2/day divided into two daily doses |
| Name | Type | Description |
|---|---|---|
| Sodium phenylbutyrate | DRUG | Open-label design with doses of sodium phenylbutyrate at 4.0 g/m2/day |
Inclusion Criteria: 1. A diagnosis of MCADD and molecular confirmation of at least one copy of the common c.985A\>G mutation. 2. ≥4 years of age 3. Able to perform and comply with study activities placement of a continuous glucose monitor, IV catheter, and all blood draws. 4. Negative pregnancy tes...