About this data
Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.
HPN-100
- Company
- Amgen
- Mechanism
- Mechanism not yet reported
- Route
- Route not yet reported
- Recruiting trials
- 2 recruiting
Efficacy results: Not yet reported
2 recruiting studies currently listed on ClinicalTrials.gov.
Data source: ClinicalTrials.gov · trial data as of Sep 11, 2026
Find a Clinical Trial Near Me
See recruiting studies for HPN-100 and check eligibility with the study team.
Trial details
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Recruiting NCT NCT06887777
Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI)
Not yet reported Assistance Publique - Hôpitaux de Paris 15 participants Est. completion 2027-04-01 1 siteOther conditions: Pyruvate Dehydrogenase Complex Deficiency DiseaseView official trial listing → -
Recruiting NCT NCT06904027
A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
Tongji Hospital 40 participants Est. completion 2031-07 5 sitesOther conditions: Urea Cycle DisordersView official trial listing → -
Completed NCT NCT00551200
Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders
Not yet reported Amgen 14 participants Est. completion 2008-12 2 sitesOther conditions: Urea Cycle DisordersView official trial listing → -
Completed NCT NCT00947297
Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)
Not yet reported Amgen 60 participants Est. completion 2011-11 22 sites Stand-aloneOther conditions: Urea Cycle DisordersView official trial listing → -
Completed NCT NCT00947544
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Not yet reported Amgen 17 participants Est. completion 2011-08 8 sites Stand-aloneOther conditions: Urea Cycle DisordersView official trial listing → -
Completed NCT NCT00977600
A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)
Not yet reported Amgen 24 participants Est. completion 2005-07 1 site Stand-aloneOther conditions: HealthyView official trial listing → -
Completed NCT NCT00986895
A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group
Not yet reported Amgen 32 participants Est. completion 2007-06 2 sitesOther conditions: Hepatic Encephalopathy, Urea Cycle DisordersView official trial listing → -
Completed NCT NCT00992459
Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
Not yet reported Amgen 46 participants Est. completion 2010-09 22 sites Stand-aloneOther conditions: Urea Cycle DisordersView official trial listing → -
Completed NCT NCT00999167
A Study of Safety and Efficacy of HPN-100 in Subjects With Cirrhosis and Episodic Hepatic Encephalopathy
Not yet reported Amgen 189 participants Est. completion 2012-04 28 sites Stand-aloneOther conditions: Cirrhosis, Hepatic EncephalopathyView official trial listing → -
Completed NCT NCT01135680
Double-Blind Randomized Crossover Trial to Access Electrocardiogram Effects of HPN-100
Not yet reported Amgen 98 participants Est. completion 2010-09 1 siteOther conditions: Drug ToxicityView official trial listing → -
Completed NCT NCT01257737
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
Not yet reported Amgen 88 participants Est. completion 2017-02-16 16 sites Stand-aloneOther conditions: Urea Cycle DisordersView official trial listing → -
Completed NCT NCT01347073
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
Not yet reported Amgen 23 participants Est. completion 2013-03 8 sites Stand-aloneOther conditions: Urea Cycle DisordersView official trial listing → -
Completed NCT NCT01881984
Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
Not yet reported University of Pittsburgh 4 participants Est. completion 2016-02 1 siteOther conditions: Medium-chain Acyl-CoA Dehydrogenase (MCAD) DeficiencyView official trial listing → -
Terminated NCT NCT01948427
Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Amgen 203 participants Est. completion 2020-02-24 1 siteOther conditions: Urea Cycle DisorderView official trial listing → -
No Longer Available NCT NCT01949766
Transition From Buphenyl to RAVICTI for the Therapy of Byler Disease
University of PittsburghOther conditions: Byler DiseaseView official trial listing → -
Completed NCT NCT02046434
Phenylbutyrate Response as a Biomarker for Alpha-synuclein Clearance From the Brain
Not yet reported University of Colorado, Denver 38 participants Est. completion 2019-09 1 siteOther conditions: Parkinson's DiseaseView official trial listing → -
No Longer Available NCT NCT02094222
Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease
Robert Squires, Jr.Other conditions: Byler DiseaseView official trial listing → -
Completed NCT NCT02246218
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
Not yet reported Amgen 27 participants Est. completion 2017-07-17 17 sitesOther conditions: Urea Cycle DisorderView official trial listing → -
Terminated NCT NCT02323100
Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)
Not yet reported, Not yet reported National Jewish Health 16 participants Est. completion 2022-03-01 3 sitesOther conditions: Cystic FibrosisView official trial listing → -
Completed NCT NCT03335488
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
Not yet reported Amgen 16 participants Est. completion 2022-12-20 11 sitesOther conditions: Urea Cycle DisorderView official trial listing → -
Suspended NCT NCT04041232
PBA Use for Treatment of ATF6-/- Patients
Not yet reported Columbia University 2 participants Est. completion 2027-08 1 siteOther conditions: ACHROMATOPSIA 7, AchromatopsiaView official trial listing → -
Active, not recruiting NCT NCT04937062
Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy
Not yet reported Weill Medical College of Cornell University 50 participants Est. completion 2026-12-31 2 sitesOther conditions: STXBP1 Encephalopathy With Epilepsy, SLC6A1 Neurodevelopmental Disorder, Developmental and Epileptic EncephalopathyView official trial listing → -
Status unknown NCT NCT05019417
Glycerol-Phenylbutyrate Treatment in Children With MCT Mutation (Allan-Herndon- Dudley Syndrome)
Not yet reported, Not yet reported Kaplan Medical Center 6 participants Est. completion 2022-08-30 1 siteOther conditions: Monocarboxylate Transporter 8 DeficiencyView official trial listing → -
Active, not recruiting NCT NCT05983588
PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS)
Not yet reported Technical University of Munich 32 participants Est. completion 2026-03-30 2 sitesOther conditions: Corticobasal Syndrome (CBS)View official trial listing →
Efficacy history
Not yet reported.
Safety
Not yet reported.
Sources
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This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.
