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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:04 AM EDT

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Showing 1–24 of 35 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: 4-PBA Clear all
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, 5:04 AM EDT
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, 5:04 AM 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, 5:04 AM 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, 5:04 AM EDT
Completed Phase 1Phase 2 Interventional

Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)

NCT00107770
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
US Department of Veterans Affairs
Federal
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
10
States / cities
Iowa City, Iowa • Lexington, Kentucky • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2010 · Synced Oct 22, 2026, 5:04 AM EDT
Active, not recruiting Phase 2 Interventional

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034
View directory record Official record
Conditions
Wolfram Syndrome
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
17 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 22, 2026, 5:04 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)

NCT00590538
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Sodium 4-Phenylbutyrate, Genistein (Unconjugated Isoflavones 100), Placebo
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 30, 2011 · Synced Oct 22, 2026, 5:04 AM EDT
Completed Phase 4 Interventional Results available

Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

NCT03335488
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI, NaPBA
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 99 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Gainesville, Florida • New York, New York • Cleveland, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 22, 2026, 5:04 AM EDT
Completed Phase 1 Interventional

Azacitidine Plus Phenylbutyrate in Treating Patients With Advanced or Metastatic Solid Tumors That Have Not Responded to Previous Treatment

NCT00005639
View directory record Official record
Conditions
Lymphoma, Small Intestine Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
Azacitidine Injection, sodium phenylbutyrate
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years to 120 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 11, 2019 · Synced Oct 22, 2026, 5:04 AM 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, 5:04 AM 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, 5:04 AM EDT
Terminated Phase 1Phase 2 Interventional

Phase I-II Study of Fluorouracil in Combination With Phenylbutyrate in Advanced Colorectal Cancer

NCT00002796
View directory record Official record
Conditions
Mucinous Adenocarcinoma of the Colon, Mucinous Adenocarcinoma of the Rectum, Recurrent Colon Cancer, Recurrent Rectal Cancer, Signet Ring Adenocarcinoma of the Colon, Signet Ring Adenocarcinoma of the Rectum, Stage IVA Colon Cancer, Stage IVA Rectal Cancer, Stage IVB Colon Cancer, Stage IVB Rectal Cancer
Interventions
fluorouracil, sodium phenylbutyrate, indomethacin, recombinant interferon gamma
Drug · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 1, 2013 · Synced Oct 22, 2026, 5:04 AM EDT
Completed Phase 1 Interventional

Azacitidine Plus Phenylbutyrate in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

NCT00004871
View directory record Official record
Conditions
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
azacitidine, sodium phenylbutyrate
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 10, 2010 · Synced Oct 22, 2026, 5:04 AM 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, 5:04 AM 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, 5:04 AM 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, 5:04 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Months to 48 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
5
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2010 · Synced Oct 22, 2026, 5:04 AM 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, 5:04 AM EDT
Terminated Phase 4 Interventional Results available

Effectiveness, Safety, and Health-Related Outcomes of NUEDEXTA® in Nursing Home Patients With Pseudobulbar Affect (PBA)

NCT02496039
View directory record Official record
Conditions
Pseudobulbar Affect (Involuntary Laughing and/or Crying)
Interventions
NUEDEXTA®
Drug
Lead sponsor
Avanir Pharmaceuticals
Industry
Eligibility
18 Years to 90 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Miami, Florida • Centerville, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 3, 2017 · Synced Oct 22, 2026, 5:04 AM 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, 5:04 AM EDT
Completed Phase 2 Interventional

Safety and Tolerability Study of Phenylbutyrate in Huntington's Disease (PHEND-HD)

NCT00212316
View directory record Official record
Conditions
Huntington's Disease
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
8
States / cities
Birmingham, Alabama • San Diego, California • Iowa City, Iowa + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2012 · Synced Oct 22, 2026, 5:04 AM EDT
Completed Phase 2 Interventional Results available

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

NCT00551200
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100, BUPHENYL®
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
2
States / cities
Minneapolis, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Oct 22, 2026, 5:04 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of AVP-923 in PBA Patients With ALS or MS

NCT00573443
View directory record Official record
Conditions
Pseudobulbar Affect (PBA)
Interventions
dextromethorphan hydrobromide 20 mg and quinidine sulfate 10 mg, dextromethorphan hydrobromide 30 mg and quinidine sulfate 10 mg, Placebo
Drug
Lead sponsor
Avanir Pharmaceuticals
Industry
Eligibility
18 Years to 80 Years
Enrollment
326 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
45
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Anaheim, California + 39 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2017 · Synced Oct 22, 2026, 5:04 AM EDT
Completed Phase 2 Interventional Results available

Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

NCT00345605
View directory record Official record
Conditions
Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
Interventions
Sodium Phenylbutyrate, Arginine
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 31, 2018 · Synced Oct 22, 2026, 5:04 AM EDT