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ClinicalTrials.gov public records Last synced Sep 6, 2026, 5:01 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
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
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
Completed Phase 2Phase 3 Interventional Results available

Phenylbutyrate Therapy for Maple Syrup Urine Disease

NCT01529060
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
Completed Not applicable Interventional Accepts healthy volunteers Results available

Effect of Endoplasmic Reticulum Stress on Metabolic Function

NCT00771901
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
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
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
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
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
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
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
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
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
Conditions
Alzheimer's Disease, Pseudobulbar Affect (PBA)
Interventions
Nuedexta (20/10)
Drug
Lead sponsor
St. Joseph's Hospital and Medical Center, Phoenix
Other
Eligibility
55 Years to 90 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Phoenix, Arizona
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
4-PBA
Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Gainesville, Florida
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
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
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
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
Conditions
ACHROMATOPSIA 7, Achromatopsia
Interventions
PBA
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
New York, New York
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
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
Recruiting Not applicable Interventional Accepts healthy volunteers

Process-Based Approach Case Series For Anxiety Problems

NCT07352592
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
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
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