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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 combined D,L-2-hydroxyglutaric aciduria and medium-chain acyl-CoA dehydrogenase deficiency. It is currently in clinical development and has not been approved by the FDA.
Phenylbutyrate targets histone deacetylases (HDACs). By inhibiting HDACs, it may influence gene expression and cellular processes relevant to the metabolic conditions it is being studied for. The drug is being evaluated for its potential therapeutic effects in these rare diseases.
Phenylbutyrate is being developed by Zevra Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ZVRA. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with specific metabolic disorders.
Phenylbutyrate is in early clinical development. One trial is in Phase 1 for combined D,L-2-hydroxyglutaric aciduria, and another is in Phase 2 for medium-chain acyl-CoA dehydrogenase deficiency. The drug is investigational and has not received regulatory approval.
Phenylbutyrate is being studied in two clinical trials. NCT07125066 is a Phase 1 open-label study in patients with combined D,L-2-hydroxyglutaric aciduria. NCT06773026 is a Phase 2 study in pediatric and adult patients with medium-chain acyl-CoA dehydrogenase deficiency. Both trials are enrolling in the United States.
Yes, phenylbutyrate is also known as sodium phenylbutyrate. The drug is being developed under the name ACER-001 in clinical trials. These names refer to the same investigational compound being studied for metabolic disorders.