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Clinical trials for Sodium phenylbutyrate

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44336   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    12 result(s) found for: Sodium phenylbutyrate. Displaying page 1 of 1.
    EudraCT Number: 2015-000075-27 Sponsor Protocol Number: HPN-100-021 Start Date*: 2016-03-07
    Sponsor Name:Horizon Therapeutics, LLC
    Full Title: A Randomised, Controlled, Open-Label Parallel Arm Study of the Safety, Pharmacokinetics and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate [GPB]) Oral Liquid and Sodium Phenylbutyrate (NaPBA)...
    Medical condition: Urea Cycle Disorders (UCDs)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000161833 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) AT (Completed) IT (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2014-003247-36 Sponsor Protocol Number: HPN-100-005SE Start Date*: 2015-02-10
    Sponsor Name:Hyperion Therapeutics, Inc.
    Full Title: A Phase 2, Fixed-Sequence, Open-Label, Switch-Over Study of the Safety and Tolerability of HPN 100 Compared to Sodium Phenylbutyrate in Children 6–17 Years of Age with Urea Cycle Disorders, with a ...
    Medical condition: Urea Cycle Disorders
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-003246-26 Sponsor Protocol Number: HPN-100-005SO Start Date*: 2015-02-10
    Sponsor Name:Hyperion Therapeutics, Inc.
    Full Title: A Phase 2, Fixed-Sequence, Open-Label, Switch-Over Study of the Safety and Tolerability of HPN 100 Compared to Sodium Phenylbutyrate in Children 6–17 Years of Age with Urea Cycle Disorders, with a ...
    Medical condition: Urea Cycle Disorders (UCDs)
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2008-003865-23 Sponsor Protocol Number: HPN-100-004 Start Date*: 2008-09-08
    Sponsor Name:Hyperion Therapeutics Limited
    Full Title: A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea...
    Medical condition: Urea Cycle Disorder (urea cycle enzyme or transporter deficiency)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-005971-11 Sponsor Protocol Number: AP101-02 Start Date*: 2021-10-20
    Sponsor Name:AL-S Pharma, AG
    Full Title: A Phase 2a, multicenter, randomized, double-blind, placebo-controlled study to evaluate safety, tolerability, pharmacodynamic markers, and pharmacokinetics of AP-101 in patients with familial amyot...
    Medical condition: Amyotrophic Lateral Sclerosis (ALS)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Trial now transitioned) DE (Completed) BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-005069-15 Sponsor Protocol Number: LIGHTHOUSEII Start Date*: 2021-06-23
    Sponsor Name:Stichting TRICALS Foundation
    Full Title: RANDOMISED DOUBLE-BLIND PLACEBO-CONTROLLED PHASE 3 TRIAL OF TRIUMEQ IN AMYOTROPHIC LATERAL SCLEROSIS
    Medical condition: Amyotrophic Lateral Sclerosis (ALS)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) IE (Trial now transitioned) ES (Ongoing) SI (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-004156-42 Sponsor Protocol Number: ACT16970 Start Date*: 2022-04-12
    Sponsor Name:Sanofi-aventis recherche & développement
    Full Title: A Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of SAR443820 in adult participants with amyotrophic lateral sclerosis, followed by an ...
    Medical condition: Amyotrophic lateral sclerosis
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) DE (Completed) IT (Prematurely Ended) NL (Completed) BE (Completed) ES (Prematurely Ended) FR (Completed) PL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-005522-28 Sponsor Protocol Number: ION363-CS1 Start Date*: Information not available in EudraCT
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients with Fused in Sarcoma Mutati...
    Medical condition: Amyotrophic Lateral Sclerosis with Fused in Sarcoma mutations
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) IT (Trial now transitioned) BE (Trial now transitioned) NL (Trial now transitioned) SE (Trial now transitioned) IE (Trial now transitioned) PL (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-006511-29 Sponsor Protocol Number: PTC857-CNS-001-ALS Start Date*: 2022-08-26
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL STUDY TO ASSESS THE EFFICACY, SAFETY, TOLERABILITY, PK, AND BIOMARKER EFFECTS OF PTC857 IN ADULT SUBJECTS WITH AMYOTROPHIC LATERAL ...
    Medical condition: Amyotrophic Lateral Sclerosis (ALS)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DE (Ongoing) BE (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing) CZ (Trial now transitioned) PL (Trial now transitioned) NL (Ongoing) NO (Trial now transitioned) SE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-005611-31 Sponsor Protocol Number: CORT113176-652 Start Date*: 2022-09-07
    Sponsor Name:Corcept Therapeutics Incorporated
    Full Title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of CORT113176 (Dazucorilant) in Patients with Amyotrophic Lateral Sclerosis (DAZALS)
    Medical condition: Amyotrophic Lateral Sclerosis
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) DE (Trial now transitioned) FR (Trial now transitioned) ES (Ongoing) PL (Trial now transitioned) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2022-002185-32 Sponsor Protocol Number: NST003 Start Date*: 2022-11-21
    Sponsor Name:NeuroSense Therapeutics Ltd
    Full Title: A Phase IIb, Randomized, Multi-center, Multinational, Prospective, Double-Blind, Placebo- Controlled Study, with an Open Label Extension, to Evaluate Safety, Tolerability and Efficacy of PrimeC in ...
    Medical condition: Amyotrophic lateral sclerosis (ALS)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10002026 Amyotrophic lateral sclerosis PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-001550-34 Sponsor Protocol Number: TIGEM2-PDH Start Date*: Information not available in EudraCT
    Sponsor Name:FONDAZIONE TELETHON
    Full Title: Studio clinico pilota per valutare l’efficacia della terapia con fenilbutirrato in pazienti affetti da deficit di piruvato deidrogenasi
    Medical condition: La Sperimentazione verrà eseguita su pazienti affetti da difetto dell’enzima piruvato deidrogenasi.I pazienti affetti da questa malattia manifestano aumento dei livelli di lattato nel sangue e nell...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10027433 - Metabolism and nutrition disorders 10058097 Metabolic disorder PT
    20.1 10027433 - Metabolism and nutrition disorders 10000491 Acidosis lactic LLT
    20.0 10022891 - Investigations 10037677 Pyruvate LLT
    20.0 10022891 - Investigations 10023660 Lactate increased LLT
    20.0 10022891 - Investigations 10057975 Lactate pyruvate ratio PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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