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Also known as Sodium phenylbutyrate
phenylbutyrate · 2 trials · 2 indications
| 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 |
| sodium phenylbutyrate | EXPERIMENTAL | a single patient will take sodium phenylbutyrate (ACER-001) current at a dose of 12 g/day; dose will be modified if the patient gains more than 5 kg of weight |
| 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...
Phenylbutyrate, also known as sodium phenylbutyrate, is an investigational small molecule being studied for the treatment of Medium-chain Acyl-CoA Dehydrogenase Deficiency (MCADD) and Combined D,L-2-hydroxyglutaric Aciduria (C-2HGA). It is currently in clinical development and has not been approved for these indications.
Phenylbutyrate is being developed by Zevra Therapeutics, Inc. (NASDAQ: ZVRA). The company is conducting clinical trials to evaluate the drug for the treatment of Medium-chain Acyl-CoA Dehydrogenase Deficiency and Combined D,L-2-hydroxyglutaric Aciduria.
Phenylbutyrate is in Phase 1 and Phase 2 clinical trials. A Phase 2 study is recruiting patients with Medium-chain Acyl-CoA Dehydrogenase Deficiency, and a Phase 1 study is enrolling by invitation for Combined D,L-2-hydroxyglutaric Aciduria. The drug is investigational and not yet approved.
Phenylbutyrate is being studied in two clinical trials. NCT06773026 is a Phase 2 study of sodium phenylbutyrate (ACER-001) for pediatric and adult patients with Medium-chain Acyl-CoA Dehydrogenase Deficiency, with an enrollment of 24 participants. NCT07125066 is a Phase 1 open-label study for Combined D,L-2-hydroxyglutaric Aciduria with one participant.
Yes, phenylbutyrate is also known as sodium phenylbutyrate. In clinical trials, it is referred to as ACER-001. The drug is being investigated for the treatment of Medium-chain Acyl-CoA Dehydrogenase Deficiency and Combined D,L-2-hydroxyglutaric Aciduria.