26 completed, 5 terminated, 3 withdrawn, 1 active not recruiting. 25 Phase 2, 14 Phase 1, 3 Not applicable, 2 Phase 3, 2 Phase 4.
Follow 4-phenylbutyric acid: new trials by email Literature Review of 4-phenylbutyric acid trials Research Report on 4-phenylbutyric acid
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Na-Phenylbutyrate VAscular Trial Sodium Phenylbutyrate, Placebo - Diabetes | Early Phase 1 | Not yet recruiting | University of Pennsylvania | 30 | 2027-06-01 | 2026-07-28 |
| An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA) Sodium phenylbutyrate - Combined D,L-2-hydroxyglutaric Aciduria | Phase 1 | Enrolling by invitation | Jerry Vockley, MD, PhD | 1 | 2025-07-30 | 2026-06-08 |
| Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Sodium phenylbutyrate - Medium-chain Acyl-CoA Dehydrogenase Deficiency | Phase 2 | Recruiting | Jerry Vockley, MD, PhD | 24 | 2025-06-30 | 2026-10-21 |
| Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) Sodium phenylbutyrate - Medium-chain Acyl-CoA Dehydrogenase Deficiency | Phase 2 | Suspended | Jerry Vockley, MD, PhD | 24 | 2024-04-01 | 2026-07-06 |
| AMX0035 in Adult Patients With Wolfram Syndrome AMX0035 - Wolfram Syndrome | Phase 2 | Active not recruiting | Amylyx Pharmaceuticals Inc. | 12 | 2023-03-03 | 2026-04-14 |
| BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases 4-Phenylbutyrate - Hereditary Diseases, Cholestasis, Intrahepatic | Phase 2 / Phase 3 | Withdrawn | Children's Hospital of Fudan University | 2023-02-08 | 2023-02-09 | |
| A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate Fezagepras, Sodium phenylbutyrate - Hyperammonemia | Phase 1 | Terminated | Liminal BioSciences Ltd. | 8 | 2022-05-13 | 2022-08-01 |
| The Effects of Sodium Phenylbutyrate on Circulating Branched Chain Amino Acids sodium phenylbutyrate - Obese Non Diabetic | Not applicable | Completed | University of Nottingham | 8 | 2021-09-01 | 2026-04-28 |
| Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. sodium phenylbutyrate - Pyruvate Dehydrogenase Complex Deficiency | Phase 2 | Completed | Fondazione Telethon | 1 | 2018-10-01 | 2021-10-13 |
| Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease AMX0035, Placebo - Alzheimer Disease | Phase 2 | Completed | Amylyx Pharmaceuticals Inc. | 95 | 2018-09-14 | 2025-03-07 |
| Open Label Extension Study of AMX0035 in Patients With ALS AMX0035 - Amyotrophic Lateral Sclerosis, ALS | Phase 2 | Completed | Amylyx Pharmaceuticals Inc. | 90 | 2018-03-29 | 2025-08-13 |
| Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) RAVICTI, NaPBA - Urea Cycle Disorder | Phase 4 | Completed | Amgen | 16 | 2018-02-20 | 2024-07-01 |
| AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) AMX0035, Placebo - Amyotrophic Lateral Sclerosis, Motor Neuron Disease | Phase 2 | Completed | Amylyx Pharmaceuticals Inc. | 137 | 2017-06-22 | 2024-05-16 |
| Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders Sodium Benzoate, Sodium Phenylbutyrate - Urea Cycle Disorders, Inborn | Not applicable | Completed | Baylor College of Medicine | 7 | 2014-09-01 | 2018-01-09 |
| Immune Reconstitution in Tuberculosis Disease vitamin D (cholecalciferol) and PBA (sodium phenylbutyrate), Placebo tablets - Pulmonary Tuberculosis (TB) | Phase 2 | Completed | Karolinska Institutet | 390 | 2012-09-01 | 2016-02-05 |
| Immune Reconstitution in HIV Disease (IREHIV) vitamin D (cholecalciferol) and PBA (sodium phenylbutyrate), Placebo tablets - HIV Infection | Phase 2 | Completed | Karolinska Institutet | 279 | 2012-09-01 | 2016-02-05 |
| Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3 Placebo, Sodium Phenylbutyrate - Spinocerebellar Ataxia Type 3 | Phase 2 | Withdrawn | Hospital de Clinicas de Porto Alegre | 2010-06-01 | 2012-08-17 | |
| Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders HPN-100, Buphenyl (NaPBA) - Urea Cycle Disorders | Phase 3 | Completed | Amgen | 46 | 2009-10-01 | 2024-07-09 |
| Effect of Endoplasmic Reticulum Stress on Metabolic Function tauroursodeoxycholic acid, placebo, sodium phenylbutyrate - Insulin Resistance, Diabetes | Not applicable | Completed | Washington University School of Medicine | 101 | 2008-02-01 | 2018-05-29 |
| Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder Sodium Phenylbutyrate, Arginine - Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors | Phase 2 | Completed | Brendan Lee | 12 | 2008-02-01 | 2018-01-31 |
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Urea Cycle Disorders, Inborn (6) · Leukemia (4) · Leukemia, Myeloid, Acute (4) · Lymphoma (4) · Amyotrophic Lateral Sclerosis (3) · Diabetes Mellitus (3) · Hodgkin Disease (3) · Leukemia, Lymphocytic, Chronic, B-Cell (3) · Lymphoma, B-Cell, Marginal Zone (3) · Lymphoma, Follicular (3) · Lymphoma, Large B-Cell, Diffuse (3) · Lymphoma, Non-Hodgkin (3) · Motor Neuron Disease (3) · Muscular Atrophy, Spinal (3) · Myelodysplastic Syndromes (3)
Amgen (4) · Amylyx Pharmaceuticals Inc. (4) · Jerry Vockley, MD, PhD (3) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (3) · Children's Hospital of Philadelphia (2) · Karolinska Institutet (2) · National Cancer Institute (NCI) (2) · Westat (2) · Baylor College of Medicine (1) · Brendan Lee (1) · Children's Hospital of Fudan University (1) · Fondazione Telethon (1) · Hospital de Clinicas de Porto Alegre (1) · Liminal BioSciences Ltd. (1) · Memorial Sloan Kettering Cancer Center (1)
United States (27) · Canada (2) · Ethiopia (2) · Italy (2) · United Kingdom (2) · Brazil (1) · Spain (1) · Switzerland (1) · Ukraine (1)
Gerard Vockley, MD, PhD (2) · Michael A. Carducci, MD (2) · Patrick Yeramian, MD (2) · Ronald Rubenstein, M.D., PhD. (2) · Steven D. Gore, MD (2) · Susanna Brighenti, PhD (2) · Brendan Lee, MD, PhD (1) · Camille Bedrosian, MD (1) · Gary F. Lewis, MD (1) · Gerard L Vockley, MD, PhD (1) · Jian-She Wang, Ph.D (1) · Johnson Liu, MD (1) · Juan C Marini, DVM., PhD (1)
PMID 24144944, PMID 33331662, PMID 35577511, PMID 37983082, PMID 19789320, PMID 20512995, PMID 20522594, PMID 22961727, PMID 29696707, PMID 30634590, PMID 31330899, PMID 32877582, PMID 33063909, PMID 35508892, PMID 37941072, PMID 38050066, PMID 39131742
P30CA006973 (3), N01NS42361 (2), R01CA067803 (2), U01CA070095 (2), 2015 (1), P50CA058236 (1), R01CA075525 (1), R01HD054599 (1), R01HL152446 (1), U54HD061221 (1)