Drug
Phenylbutyric acid clinical trials
Also written sodium phenylbutyrate, Olpruva and BUPHENYL®.
Keep track of what changes. Save the phenylbutyric acid studies recruiting or about to start, and get an email each week — studies that open, close, add sites or post results.
Watch these studiesHow studies write it
Studies that test it under each name
Other names
Studies by the year they started
31 studies that test it; most in 2000 (5); 1 more planned to start later
| Year | Studies |
|---|---|
| 1996 | 1 |
| 1997 | 1 |
| 2000 | 5 |
| 2001 | 1 |
| 2003 | 1 |
| 2005 | 2 |
| 2007 | 3 |
| 2008 | 4 |
| 2009 | 1 |
| 2010 | 1 |
| 2013 | 1 |
| 2014 | 1 |
| 2018 | 1 |
| 2020 | 1 |
| 2021 | 1 |
| 2022 | 1 |
| 2023 | 1 |
| 2024 | 1 |
| 2025 | 3 |
By phase
A phase 1/2 study counts in both.
What it is tested for
Each study’s main condition, where its own words name it.
Who runs them
Industry 3 · Other 30
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (3) · National Cancer Institute (NCI) (3) · Jerry Vockley, MD, PhD (3) · Amgen (2) · Brendan Lee (2) · Children's Hospital of Philadelphia (2) · Westat (2) · University of Nottingham (1)
Often tested with
Tested in the same studies.
Azacitidine (3) · Glycerol phenylbutyrate (2) · Sodium Benzoate (2) · Ursodoxicoltaurine (1) · Arginine (1) · Tretinoin (1) · Phenylacetic acid (1) · Setogepram (1)
Studies
Open first, then newest update. How each study uses phenylbutyric acid is read from its arms, and is right about nine times in ten.
Testing it · 33
- Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)RecruitingPhase 2Jerry Vockley, MD, PhDNCT06773026Written as Sodium phenylbutyrate
- Na-Phenylbutyrate VAscular TrialNot yet recruitingEarly Phase 1University of PennsylvaniaNCT06645769Written as Sodium Phenylbutyrate
- An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)Enrolling by invitationPhase 1Jerry Vockley, MD, PhDNCT07125066Written as Sodium phenylbutyrate
- PBA Use for Treatment of ATF6-/- PatientsSuspendedEarly Phase 1Columbia UniversityNCT04041232Written as PBA
- Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)SuspendedPhase 2Jerry Vockley, MD, PhDNCT06069375Written as Sodium phenylbutyrate
- The Effects of Sodium Phenylbutyrate on Circulating Branched Chain Amino AcidsCompletedNot applicableUniversity of NottinghamNCT05028803Written as sodium phenylbutyrate
- Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular AtrophyCompletedPhase 1/2University of UtahNCT00528268Written as Sodium phenylbutyrate
- Safety and Tolerability of Phenylbutyrate in Inclusion Body MyositisCompletedPhase 1University of Kansas Medical CenterNCT04421677Written as Phenylbutyrate Oral Tablet
- Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle DisordersCompletedPhase 2AmgenNCT00551200Written as BUPHENYL®
- Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle DisordersCompletedPhase 3AmgenNCT00992459Written as Buphenyl (NaPBA)
- BSEP Function Rescue During Childhood Inhereditary Cholestatic DiseasesWithdrawnPhase 2/3Children's Hospital of Fudan UniversityNCT04531878Written as 4-Phenylbutyrate
- A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium PhenylbutyrateTerminatedPhase 1Liminal BioSciences Ltd.NCT05349435Written as Sodium phenylbutyrate
- Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.CompletedPhase 2Fondazione TelethonNCT03734263Written as sodium phenylbutyrate
- Phenylbutyrate Therapy for Maple Syrup Urine DiseaseCompletedPhase 2/3Brendan LeeNCT01529060Written as Phenylbutyrate
- Azacitidine Plus Phenylbutyrate in Treating Patients With Advanced or Metastatic Solid Tumors That Have Not Responded to Previous TreatmentCompletedPhase 1Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsNCT00005639Written as sodium phenylbutyrate
- Effect of Endoplasmic Reticulum Stress on Metabolic FunctionCompletedNot applicableWashington University School of MedicineNCT00771901Written as sodium phenylbutyrate
- Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle DisorderCompletedPhase 2Brendan LeeNCT00345605Written as Sodium Phenylbutyrate
- Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle DisordersCompletedNot applicableBaylor College of MedicineNCT02111200Written as Sodium Phenylbutyrate
- Phenylbutyrate and Tretinoin in Treating Patients With Hematologic CancerCompletedPhase 1Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsNCT00006239Written as sodium phenylbutyrate
- Phenylbutyrate Plus Azacitidine in Treating Patients With Acute Myeloid Leukemia, Myelodysplasia, Non-Hodgkin's Lymphoma, Multiple Myeloma, Non-small Cell Lung Cancer, or Prostate CancerCompletedPhase 2Memorial Sloan Kettering Cancer CenterNCT00006019Written as sodium phenylbutyrate
See all 33 studies that test phenylbutyric acid →
As the comparator · 2 — what is tested is compared with phenylbutyric acid
Codes
What other systems call phenylbutyric acid · ClinicalTrials.gg INT003646
- MeSH (NLM) C0757734-phenylbutyric acidConfirmed ClinicalTrials.gov, NLM, Wikidata
- UNII (FDA) 7WY7YBI87EPHENYLBUTYRIC ACIDConfirmed FDA, NLM, Wikidata
- RxNorm 1546447Confirmed FDA, Wikidata
- Wikidata Q27088364phenylbutyric acidConfirmed FDA, NLM, Wikidata
- NCI Thesaurus C636994-Phenylbutyric AcidOne source FDA
- DrugBank DB06819One source Wikidata
| System | Code | Name there | Checked by |
|---|---|---|---|
| MeSH (NLM) | C075773 | 4-phenylbutyric acid | Confirmed ClinicalTrials.gov, NLM, Wikidata |
| UNII (FDA) | 7WY7YBI87E | PHENYLBUTYRIC ACID | Confirmed FDA, NLM, Wikidata |
| RxNorm | 1546447 | — | Confirmed FDA, Wikidata |
| Wikidata | Q27088364 | phenylbutyric acid | Confirmed FDA, NLM, Wikidata |
| NCI Thesaurus | C63699 | 4-Phenylbutyric Acid | One source FDA |
| DrugBank | DB06819 | — | One source Wikidata |
Also registered as sodium phenylbutyrate (UNII NT6K61736T · RxNorm 214837).
“Confirmed” means two independent publishers link the code to this drug; “one source”, one does and its name agrees.