Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 30, 2026, 11:51 AM EDT

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Showing 1–23 of 23 matching trials from the live ClinicalTrials.gov search.
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
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
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
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
Urea Cycle Disorders
Interventions
HPN-100, NaPBA
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
7
States / cities
Los Angeles, California • Washington D.C., District of Columbia • New York, New York + 4 more
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
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
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
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
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
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
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
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
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
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
Amyotrophic Lateral Sclerosis
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
San Francisco, California • Boston, Massachusetts • Worcester, Massachusetts + 2 more
Active, not recruiting Phase 2 Interventional

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034
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
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
Terminated Phase 1Phase 2 Interventional Results available

Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)

NCT02323100
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
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
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
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
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