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ClinicalTrials.gov public records Last synced Sep 5, 2026, 7:15 PM EDT

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Showing 1–24 of 60 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Urea Cycle Disorders
Interventions
HPN-100, Buphenyl (NaPBA)
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
21
States / cities
Long Beach, California • Los Angeles, California • Stanford, California + 18 more
Completed Not applicable Interventional Accepts healthy volunteers Results available

A Skills-based RCT for Physical Activity Using Peer Mentors

NCT02329262
Conditions
Health Behavior, Obesity, Motor Activity
Interventions
Mentoring to be Active with Accelerometers, Planning to be Active with Accelerometers
Behavioral
Lead sponsor
Ohio State University
Other
Eligibility
14 Years to 64 Years
Enrollment
571 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2018
U.S. locations
20
States / cities
Bidwell, Ohio • Chillicothe, Ohio • Coal Grove, Ohio + 13 more
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
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
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
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
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
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
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
Pseudobulbar Affect (Involuntary Laughing and/or Crying)
Interventions
NUEDEXTA®
Drug
Lead sponsor
Avanir Pharmaceuticals
Industry
Eligibility
18 Years to 90 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Miami, Florida • Centerville, Ohio
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
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
Conditions
Spinal Muscular Atrophy
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
University of Utah
Other
Eligibility
1 Day to 6 Months
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
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
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
5
States / cities
Stanford, California • St Louis, Missouri • New York, New York + 2 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
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
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
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
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
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
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
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
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