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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: phenylbutyrate Clear all
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 13, 2024 · Synced Sep 28, 2026, 9:23 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 4, 2013 · Synced Sep 28, 2026, 9:23 PM 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 21, 2026 · Synced Sep 28, 2026, 9:23 PM 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 8, 2010 · Synced Sep 28, 2026, 9:23 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 29, 2026 · Synced Sep 28, 2026, 9:23 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 6, 2025 · Synced Sep 28, 2026, 9:23 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 Jun 30, 2024 · Synced Sep 28, 2026, 9:23 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 21, 2024 · Synced Sep 28, 2026, 9:23 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 17, 2022 · Synced Sep 28, 2026, 9:23 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 23, 2023 · Synced Sep 28, 2026, 9:23 PM EDT
Completed Phase 2 Interventional

Phenylbutyrate Plus Azacitidine in Treating Patients With Acute Myeloid Leukemia, Myelodysplasia, Non-Hodgkin's Lymphoma, Multiple Myeloma, Non-small Cell Lung Cancer, or Prostate Cancer

NCT00006019
View directory record Official record
Conditions
Leukemia, Lung Cancer, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Prostate Cancer
Interventions
azacitidine, sodium phenylbutyrate
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 23, 2013 · Synced Sep 28, 2026, 9:23 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 30, 2018 · Synced Sep 28, 2026, 9:23 PM 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 7, 2026 · Synced Sep 28, 2026, 9:23 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 9, 2024 · Synced Sep 28, 2026, 9:23 PM 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 24, 2022 · Synced Sep 28, 2026, 9:23 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 5, 2026 · Synced Sep 28, 2026, 9:23 PM 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 28, 2017 · Synced Sep 28, 2026, 9:23 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 10, 2010 · Synced Sep 28, 2026, 9:23 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 16, 2026 · Synced Sep 28, 2026, 9:23 PM EDT
Completed Phase 2 Interventional

Phenylbutyrate, Dexamethasone, and Sargramostim in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia

NCT00006240
View directory record Official record
Conditions
Leukemia
Interventions
sargramostim, dexamethasone, oral sodium phenylbutyrate
Biological · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
4
States / cities
Bethesda, Maryland • New York, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 27, 2015 · Synced Sep 28, 2026, 9:23 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 9, 2024 · Synced Sep 28, 2026, 9:23 PM EDT
Recruiting Phase 2 Interventional

METabolic MODulation to Enhance Insulin Sensitivity and Mitochondrial Function in Type 1 Diabetes (MetMod-T1D)

NCT07699380
View directory record Official record
Conditions
Type 1 Diabetes (T1D), Metabolic Diseases, Glucose Metabolism Disorders, Endocrine System Diseases, Autoimmune Diseases, Immune System Diseases, Diabetes Melletus, Type 1, Nutritional and Metabolic Diseases, Combination Therapy
Interventions
AMX0035, Placebo
Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 69 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 28, 2026, 9:23 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 7, 2026 · Synced Sep 28, 2026, 9:23 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 3, 2008 · Synced Sep 28, 2026, 9:23 PM EDT