Ravicti (glycerol phenylbutyrate) / Immedica, Amgen 
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  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Phenylbutyrate for syngap1-related Developmental and Epileptic Encephalopathy (South Hall H - Level 1 - LACC) -  Nov 26, 2024 - Abstract #AES2024AES_2354;    
    Glycerol phenylbutyrate is a promising novel therapy for children with SYNGAP1. In ongoing work, we are (1) reviewing developmental assessment data and (2) enrolling additional children with SYNGAP1 and other monogenetic developmental and epileptic encephalopathies.
  • ||||||||||  Zolinza (vorinostat) / Merck (MSD), Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Mechanistic Insights into Pharmacological Interventions for SLC6A1 Variant-related Disorders (South Hall H - Level 1 - LACC) -  Nov 26, 2024 - Abstract #AES2024AES_2289;    
    These findings suggest that the pharmacological agents tested may offer viable therapeutic options. Ravicti (glycerol-phenylbutyrate) is currently in clinical trials for SLC6A1-related disorders, but this study aims to tease out mechanisms to identify alternative therapeutic treatments.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Journal:  Perinatal management and follow-up in a child with a prenatal diagnosis of OTC deficiency: a case report. (Pubmed Central) -  Oct 15, 2024   
    To date, after 3.5?years of follow up, growth and neurological development have been adequate, biochemical control has been appropriate except for a simple and mild decompensation during the course of a gastroenteritis. This case emphasises the importance of early diagnosis and treatment to avoid potential complications.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Trial completion date, Trial primary completion date:  PBA Use for Treatment of ATF6-/- Patients (clinicaltrials.gov) -  Aug 22, 2024   
    P1,  N=2, Not yet recruiting, 
    This case emphasises the importance of early diagnosis and treatment to avoid potential complications. Trial completion date: Mar 2024 --> May 2025 | Trial primary completion date: Mar 2024 --> May 2025
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen, Xifaxan (rifaximin) / Alfa Wassermann, Bausch Health
    Think Outside the Box! Not All Hyperammonemia is Due to Liver Disease (Exhibit Hall E) -  Aug 20, 2024 - Abstract #ACG2024ACG_5327;    
    Urea cycle disorders should be considered in patients who present with neuropsychiatric symptoms in the emergency room with hyperammonemia. A prompt recognition of symptoms will prevent permanent neurological damage or even death.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    MODELING THE PHARMACOKINETICS OF PHENYLBUTYRATE IN FED AND FASTED STATES (Poster Room | Level 0) -  Jul 30, 2024 - Abstract #SSIEM2024SSIEM_839;    
    NaPBA administration in the fasted state is predicted to increase efficacy proportionally to increased drug exposure. This theoretically allows for a 30% dose decrease while maintaining comparable efficacy outcomes as NaPBA or GPB administered in the fed state.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Journal:  Treatment and management for children with urea cycle disorder in chronic stage. (Pubmed Central) -  Oct 9, 2023   
    Gene therapy, stem cell therapy, enzyme therapy and other novel technologies may offer options for treatment in UCD patients. The regular biochemical assessments like blood ammonia, liver function and plasma amino acid profile are needed, and physical growth, intellectual development, nutritional intake should be also evaluated for adjusting treatment in time.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Enrollment open, Enrollment change, Trial completion date, Trial primary completion date:  STXBP1-E: Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy (clinicaltrials.gov) -  Oct 4, 2023   
    P1,  N=50, Enrolling by invitation, 
    The regular biochemical assessments like blood ammonia, liver function and plasma amino acid profile are needed, and physical growth, intellectual development, nutritional intake should be also evaluated for adjusting treatment in time. Active, not recruiting --> Enrolling by invitation | N=21 --> 50 | Trial completion date: Jan 2025 --> Dec 2025 | Trial primary completion date: Jan 2023 --> Jan 2025
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
    Journal:  Monitoring the treatment of urea cycle disorders using phenylbutyrate metabolite analyses: Still many lessons to learn. (Pubmed Central) -  Sep 17, 2023   
    Medications that elicit an alternate pathway for nitrogen excretion such as oral sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB) and intravenous sodium phenylacetate (NaPAA) are important for the management of urea cycle disorders (UCDs)...These results demonstrate that during routine clinical management, the majority of patients have PAA levels that are deemed safe. As age is negatively associated with PAA levels however, children undergoing treatment with NaPBA may need close monitoring of their phenylbutyrate metabolite levels.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
    Urea cycle disorders in Argentina, analysis from a cohort of 135 patients diagnosed in the last 20 years. (Exhibitions Hall (ICC)) -  Jul 11, 2023 - Abstract #SSIEM2023SSIEM_716;    
    76% required chronic treatment: sodium benzoate 76%, sodium phenylbutyrate 27%, and glycerol phenylbutyrate 12%...The mortality rate was higher than the one reported in other countries. We reported their presenting symptoms and sociocultural characteristics as well as accessibility to diagnosis and treatment, trying to identify the negative correlations in order to portray which variables are most related to worse results.
  • ||||||||||  sodium phenylbutyrate / Generic mfg.
    Journal:  Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders. (Pubmed Central) -  Apr 25, 2023   
    Lower systemic exposure of phenylacetate (PAA) and phenylbutyrate (PBA) was observed when ACER-001 was administered with a high-fat meal relative to a fasting state suggesting that the lower doses of PBA administered under fasting conditions may yield similar efficacy with potentially fewer dose dependent adverse effects relative to higher doses with a meal. ACER-001 appeared to be adequately taste-masked, staying below the aversive taste threshold for the first 3
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica, Xifaxan (rifaximin) / Alfa Wassermann, Bausch Health, Norgine, ASKA Pharma
    Whole body clearance and production of ammonia quantified by constant ammonia infusion  (Poster Area) -  Apr 12, 2023 - Abstract #EASLILC2023EASL_ILC_405;    
    The method identified lower clearance and higher production in cirrhosis patients, and showed that phenylbutyrate increases clearance while lactulose + rifaximin decreases production. The method can be used to examine a range of questions on normo-/pathophysiology and ammonia targeting treatment mechanisms.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
    Severe Hyperammonemia and Coma in a Post-partum Patient Secondary to a Urea Cycle Disorder (Walter E. Washington Convention Center, Area K, Hall C (Lower Level)) -  Mar 25, 2023 - Abstract #ATS2023ATS_8078;    
    RRT was later discontinued after initiating IV ammonia scavenger therapy with Ammonul (sodium phenylacetate and sodium benzoate)... Awareness of late-onset UCD in MICU patients as a cause of encephalopathy and hyperammonemia is necessary for early diagnosis and timely management to prevent life-threatening complications of hyperammonemia.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Trial completion date, Trial primary completion date:  PBA Use for Treatment of ATF6-/- Patients (clinicaltrials.gov) -  Mar 17, 2023   
    P1,  N=2, Not yet recruiting, 
    Awareness of late-onset UCD in MICU patients as a cause of encephalopathy and hyperammonemia is necessary for early diagnosis and timely management to prevent life-threatening complications of hyperammonemia. Trial completion date: Mar 2023 --> Mar 2024 | Trial primary completion date: Mar 2023 --> Mar 2024
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Trial completion:  Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Na (clinicaltrials.gov) -  Mar 3, 2023   
    P4,  N=16, Completed, 
    Trial completion date: Mar 2023 --> Mar 2024 | Trial primary completion date: Mar 2023 --> Mar 2024 Recruiting --> Completed
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
    4-phenylbutyrate for Children with STXBP1 and SLC6A1 Mutations - A Pilot Clinical Trial (Hall B, Level 3) -  Nov 29, 2022 - Abstract #AES2022AES_1846;    
    P1
    Early clinical experience suggests that 4-phenylbutyrate is a promising approach to reduce or eliminate seizures in children with pathogenic mutations of STXBP1 or SLC6A1. Full results are pending trial completion.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Trial completion date, Trial primary completion date:  STXBP1-E: Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy (clinicaltrials.gov) -  Sep 30, 2022   
    P1,  N=21, Active, not recruiting, 
    Full results are pending trial completion. Trial completion date: Jul 2023 --> Jan 2025 | Trial primary completion date: Jul 2022 --> Jan 2023
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
    Clinical, Journal:  Switching to Glycerol Phenylbutyrate in 48 Patients with Urea Cycle Disorders: Clinical Experience in Spain. (Pubmed Central) -  Sep 13, 2022   
    Trial completion date: Jul 2023 --> Jan 2025 | Trial primary completion date: Jul 2022 --> Jan 2023 This study demonstrates the safety of direct switching from other nitrogen scavengers to GPB in clinical practice, which improves efficacy, metabolic control, and RAE compared to previous treatments.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica, Kremezin (AST-120) / Mitsubishi Tanabe, Xifaxan (rifaximin) / Alfa Wassermann, Bausch Health, Norgine, ASKA Pharma
    Review, Journal:  Novel Drugs for the Management of Hepatic Encephalopathy: Still a Long Journey to Travel. (Pubmed Central) -  Jul 12, 2022   
    However, their clinical use is limited due to the presence of major drawbacks in their study design, sample size, safety profile, bias, and heterogenicity. This study will discuss the novel therapeutic targets for HE in liver cirrhosis patients with supporting clinical trial data.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Enrollment closed:  STXBP1-E: Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy (clinicaltrials.gov) -  May 18, 2022   
    P1,  N=20, Active, not recruiting, 
    This study will discuss the novel therapeutic targets for HE in liver cirrhosis patients with supporting clinical trial data. Recruiting --> Active, not recruiting
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Trial completion date, Trial primary completion date:  PBA Use for Treatment of ATF6-/- Patients (clinicaltrials.gov) -  Apr 6, 2022   
    P1,  N=2, Not yet recruiting, 
    Active, not recruiting --> Completed Trial completion date: Mar 2022 --> Mar 2023 | Trial primary completion date: Mar 2022 --> Mar 2023
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Enrollment change, Trial completion date, Trial termination, Trial primary completion date:  Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis) (clinicaltrials.gov) -  Mar 21, 2022   
    P1/2,  N=13, Terminated, 
    Increased awareness of published PB metabolite data and further clinician education on test interpretation may help to inform the use of metabolite testing to optimize UCD care. N=36 --> 13 | Trial completion date: Jun 2022 --> Mar 2022 | Recruiting --> Terminated | Trial primary completion date: Jun 2022 --> Mar 2022; funding ended
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Horizon Therapeutics, Immedica
    Review, Journal:  Ammonia Removal by Metabolic Scavengers for the Prevention and Treatment of Hepatic Encephalopathy in Cirrhosis. (Pubmed Central) -  Oct 27, 2021   
    Effective lowering of circulating ammonia is the mainstay strategy in the prevention and treatment of hepatic encephalopathy in cirrhosis and there is increasing interest in agents with the metabolic potential for the active removal of ammonia by the liver and skeletal muscle by agents including L-ornithine L-aspartate, branched-chain amino acids, as well as the re-purposing of benzoate and phenylacetate currently employed for the control of hyperammonaemia in congenital urea-cycle enzymopathies...Glycerol phenylbutyrate was the only agent with a beneficial effect on both hyperammonaemia and hepatic encephalopathy. None were superior to lactulose for the lowering of blood ammonia.
  • ||||||||||  lorazepam / Generic mfg.
    [VIRTUAL] Arginase Deficiency in an Immigrant Patient Without Medical Care () -  Sep 25, 2021 - Abstract #AAPNCE2021AAP_NCE_1892;    
    Progression of arginase deficiency and similar conditions can be prevented with early diagnosis and initiation of therapy. Preparing physicians to recognize potential disorders in metabolism can be lifesaving for patients of all ages.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Enrollment change:  STXBP1-E: Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy (clinicaltrials.gov) -  Aug 20, 2021   
    P1,  N=20, Recruiting, 
    Preparing physicians to recognize potential disorders in metabolism can be lifesaving for patients of all ages. N=10 --> 20
  • ||||||||||  Xifaxan (rifaximin) / Alfa Wassermann, Bausch Health, Norgine, ASKA Pharma
    Review, Journal:  Novel Therapies in Hepatic Encephalopathy. (Pubmed Central) -  Jul 21, 2021   
    Several agents have surfaced over recent years with promise to treat hepatic encephalopathy and mitigate the cognitive impairment associated with this disease process. The purpose of this article is to highlight the leading emerging therapies in hepatic encephalopathy as well as their therapeutic targets.
  • ||||||||||  Ravicti (glycerol phenylbutyrate) / Immedica, Amgen
    Trial primary completion date:  PBA Use for Treatment of ATF6-/- Patients (clinicaltrials.gov) -  Jul 6, 2021   
    P1,  N=2, Not yet recruiting, 
    The purpose of this article is to highlight the leading emerging therapies in hepatic encephalopathy as well as their therapeutic targets. Trial primary completion date: Dec 2021 --> Mar 2022