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ClinicalTrials.gov public records Last synced Oct 22, 2026, 2:38 PM EDT

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Showing 1–24 of 52 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: phenylbutyrate Clear all
Completed Not applicable Interventional Accepts healthy volunteers Results available

Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders

NCT02111200
View directory record Official record
Conditions
Urea Cycle Disorders, Inborn
Interventions
Sodium Benzoate, Sodium Phenylbutyrate
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 64 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 9, 2018 · Synced Oct 22, 2026, 2:38 PM EDT
Recruiting Phase 2 Interventional

A Fully Remote Feasibility Trial of Phenylbutyrate for SLC6A1-Related Disorders

NCT07847918
View directory record Official record
Conditions
SLC6A1
Interventions
Glycerol Phenylbutyrate
Drug
Lead sponsor
Scripps Translational Science Institute
Other
Eligibility
2 Years to 60 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 2 Interventional Results available

Open Label Extension Study of AMX0035 in Patients With ALS

NCT03488524
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, ALS
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
Not listed
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
24
States / cities
Phoenix, Arizona • Orange, California • San Francisco, California + 20 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2025 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 2Phase 3 Interventional

AMX0035 and Progressive Supranuclear Palsy

NCT06122662
View directory record Official record
Conditions
Progressive Supranuclear Palsy, PSP, Neurodegenerative Diseases, Atypical Parkinsonism
Interventions
AMX0035, Placebo
Drug · Other
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
40 Years to 80 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
35
States / cities
Scottsdale, Arizona • Little Rock, Arkansas • Fountain Valley, California + 31 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 22, 2026, 2:38 PM EDT
Terminated No phase listed Observational Results available

Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)

NCT01948427
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Not listed
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
203 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Blue Bell, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 22, 2026, 2:38 PM EDT
Not listed Phase 1Phase 2 Interventional

Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS

NCT04987671
View directory record Official record
Conditions
ALS
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

NCT00528268
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
University of Utah
Other
Eligibility
1 Day to 6 Months
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 28, 2025 · Synced Oct 22, 2026, 2:38 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

NCT00439569
View directory record Official record
Conditions
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
5
States / cities
Stanford, California • St Louis, Missouri • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2010 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 2 Interventional Results available

Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease

NCT03533257
View directory record Official record
Conditions
Alzheimer Disease
Interventions
AMX0035, Placebo
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
55 Years to 89 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
11
States / cities
Jacksonville, Florida • Orlando, Florida • Palmetto Bay, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2025 · Synced Oct 22, 2026, 2:38 PM EDT
Recruiting Phase 2 Interventional

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

NCT06773026
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
4 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 2 Interventional Results available

AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT03127514
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Diseases, Neurodegenerative Diseases, Spinal Cord Diseases, TDP-43 Proteinopathies, Nervous System Diseases, Central Nervous System Diseases
Interventions
AMX0035, Placebo
Drug · Other
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
25
States / cities
Phoenix, Arizona • Orange, California • San Francisco, California + 21 more
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 2 Interventional Results available

A Study of Safety and Efficacy of HPN-100 in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy

NCT00999167
View directory record Official record
Conditions
Cirrhosis, Hepatic Encephalopathy
Interventions
HPN-100, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
189 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
28
States / cities
Palo Alto, California • Washington D.C., District of Columbia • Miami, Florida + 21 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Oct 22, 2026, 2:38 PM EDT
Suspended Phase 2 Interventional

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

NCT06069375
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
10 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Double-Blind Randomized Crossover Trial to Access Electrocardiogram Effects of HPN-100

NCT01135680
View directory record Official record
Conditions
Drug Toxicity
Interventions
HPN-100, HPN-100 or Placebo, Placebo, Moxifloxacin
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 45 Years
Enrollment
98 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 20, 2024 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 1 Interventional

Phase I and Pharmacokinetic Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Refractory Malignancy

NCT00001565
View directory record Official record
Conditions
Brain Neoplasms, Neuroblastoma
Interventions
phenylbutyrate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 2Phase 3 Interventional

A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT04516096
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
San Francisco, California • Boston, Massachusetts • Worcester, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2023 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 1 Interventional

Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis

NCT04421677
View directory record Official record
Conditions
Inclusion Body Myositis, Sporadic Inclusion Body Myositis
Interventions
Phenylbutyrate Oral Tablet
Drug
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 2 Interventional

5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

NCT00005934
View directory record Official record
Conditions
Beta Thalassemia
Interventions
5-Azacytidine
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 1 Interventional

Phenylbutyrate in Treating Patients With Refractory Solid Tumors or Lymphoma

NCT00002909
View directory record Official record
Conditions
Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
chemotherapy, oral sodium phenylbutyrate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 5, 2013 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Effect of Endoplasmic Reticulum Stress on Metabolic Function

NCT00771901
View directory record Official record
Conditions
Insulin Resistance, Diabetes, Obesity
Interventions
tauroursodeoxycholic acid, placebo, sodium phenylbutyrate
Drug · Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 65 Years
Enrollment
101 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2014
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 29, 2018 · Synced Oct 22, 2026, 2:38 PM EDT
Withdrawn Phase 2 Interventional

Phenylbutyrate and Valganciclovir in Treating Patients With Relapsed or Refractory Epstein-Barr Virus-Positive Cancer

NCT00387530
View directory record Official record
Conditions
Gastric Cancer, Head and Neck Cancer, Lymphoma, Lymphoproliferative Disorder
Interventions
oral sodium phenylbutyrate, valganciclovir, polymerase chain reaction, protein expression analysis, biopsy
Drug · Genetic · Procedure
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2019 · Synced Oct 22, 2026, 2:38 PM EDT
Completed Phase 4 Interventional Results available

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

NCT01257737
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Year and older
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
15
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 22, 2024 · Synced Oct 22, 2026, 2:38 PM EDT
Approved For Marketing No phase listed Expanded access

An Intermediate Size Expanded Access Protocol of AMX0035 for ALS

NCT05286372
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
U.S. locations
20
States / cities
Los Angeles, California • Oakland, California • Fort Lauderdale, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2022 · Synced Oct 22, 2026, 2:38 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)

NCT02323100
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ravicti low dose, Placebo
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 22, 2026, 2:38 PM EDT