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ClinicalTrials.gov public records Last synced Oct 21, 2026, 2:38 AM EDT

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Showing 1–24 of 61 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: PBA Clear all
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 21, 2026, 2:38 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 21, 2026, 2:38 AM 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 21, 2026, 2:38 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 21, 2026, 2:38 AM 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 21, 2026, 2:38 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Phenylbutyrate Therapy for Maple Syrup Urine Disease

NCT01529060
View directory record Official record
Conditions
Maple Syrup Urine Disease
Interventions
Phenylbutyrate, Placebo powder
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
3 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 19, 2019 · Synced Oct 21, 2026, 2:38 AM EDT
Completed Phase 1 Interventional Results available

Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation

NCT01881984
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Interventions
Ravicti
Drug
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 25, 2017 · Synced Oct 21, 2026, 2:38 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 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Oct 21, 2026, 2:38 AM EDT
Completed Phase 3 Interventional

Comparison of Efficacy and Safety of Azelaic Acid 15% Gel With Its Vehicle in Subjects With Mild to Moderate Acne

NCT00031096
View directory record Official record
Conditions
Acne Vulgaris
Interventions
Azelaic Acid Gel 15% (Finacea, BAY39-6251), Vehicle gel (SH H 655 PBA)
Drug
Lead sponsor
LEO Pharma
Industry
Eligibility
12 Years and older
Enrollment
879 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002
U.S. locations
16
States / cities
Mobile, Alabama • Boulder, Colorado • Newnan, Georgia + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2023 · Synced Oct 21, 2026, 2:38 AM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

NCT02246218
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Day to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 21, 2026, 2:38 AM 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 21, 2026, 2:38 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 21, 2026, 2:38 AM EDT
Active, not recruiting Early Phase 1 Interventional

Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy

NCT04937062
View directory record Official record
Conditions
STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder, Developmental and Epileptic Encephalopathy
Interventions
Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
0 Months to 17 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 21, 2026, 2:38 AM 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 21, 2026, 2:38 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 21, 2026, 2:38 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 21, 2026, 2:38 AM EDT
Enrolling by invitation Phase 1 Interventional

An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)

NCT07125066
View directory record Official record
Conditions
Combined D,L-2-hydroxyglutaric Aciduria
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Oct 21, 2026, 2:38 AM EDT
Completed Phase 4 Interventional Results available

Safety, Tolerability and Effectiveness of Nuedexta in the Treatment of Pseudobulbar Affect (PBA)

NCT01799941
View directory record Official record
Conditions
Pseudobulbar Affect (PBA), Stroke, Dementia, Traumatic Brain Injury (TBI)
Interventions
Nuedexta (DM 20 mg/Q 10 mg)
Drug
Lead sponsor
Avanir Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Pensacola, Florida
Source: ClinicalTrials.gov public record
Updated Mar 15, 2017 · Synced Oct 21, 2026, 2:38 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 21, 2026, 2:38 AM EDT
Completed Phase 1 Interventional

Phenylbutyrate and Tretinoin in Treating Patients With Hematologic Cancer

NCT00006239
View directory record Official record
Conditions
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
sodium phenylbutyrate, tretinoin
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 29, 2017 · Synced Oct 21, 2026, 2:38 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain

NCT02046434
View directory record Official record
Conditions
Parkinson's Disease
Interventions
Glycerol Phenylbutyrate
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
21 Years to 80 Years
Enrollment
38 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2019
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Oct 21, 2026, 2:38 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Process-Based Approach Case Series For Anxiety Problems

NCT07352592
View directory record Official record
Conditions
Anxiety
Interventions
Process-Based Approach
Behavioral
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2030
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 21, 2026, 2:38 AM 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 21, 2026, 2:38 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 21, 2026, 2:38 AM EDT