Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 3, 2026, 6:55 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 52 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: Phenylbutyrate Clear all
Completed Phase 2 Interventional

Phenylbutyrate to Treat Children With Progressive or Recurrent Brain Tumors

NCT00006450
View directory record Official record
Conditions
Brain Tumor
Interventions
Phenylbutyrate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 3, 2026, 6:55 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 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 3, 2026, 6:55 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 3, 2026, 6:55 PM EDT
Completed Phase 3 Interventional Results available

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT01347073
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
29 Days to 6 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
8
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Portland, Maine + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2024 · Synced Oct 3, 2026, 6:55 PM 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 3, 2026, 6:55 PM EDT
Not listed Phase 3 Interventional

Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

NCT05021536
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Placebo, AMX0035
Other · Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
664 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
28
States / cities
Phoenix, Arizona • Los Angeles, California • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2024 · Synced Oct 3, 2026, 6:55 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 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Oct 3, 2026, 6:55 PM 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 3, 2026, 6:55 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 3, 2026, 6:55 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 3, 2026, 6:55 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 PM EDT
Completed Phase 3 Interventional Results available

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

NCT00947297
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
21
States / cities
Long Beach, California • Los Angeles, California • Stanford, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Oct 3, 2026, 6:55 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 3, 2026, 6:55 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 PM 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 3, 2026, 6:55 PM EDT