lucerastat (ACT-434964) / Idorsia 
Welcome,         Profile    Billing    Logout  
  Diseases    Trials    Trials    News 
  • ||||||||||  lucerastat (ACT-434964) / Idorsia
    Enrollment change, Monotherapy:  Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (clinicaltrials.gov) -  Aug 8, 2024   
    P3,  N=118, Completed, 
    GCS inhibition with lucerastat might be a novel approach to stabilize or slow the progression of kidney dysfunction, a major therapeutic goal in patients with Fabry disease. N=182 --> 118
  • ||||||||||  Humanistic Burden of Fabry Disease and Associated Utility Values () -  Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2023;    
    The results suggest utility values increase with ERT treatment, but patient burden has remained stable over time. Long-term data with existing therapies may provide additional insights on outcomes including pain, disease severity, and quality of life.
  • ||||||||||  Clinical, Review, Journal:  Fabry Disease: current & novel therapeutic strategies. A narrative review. (Pubmed Central) -  Mar 27, 2023   
    Long-term data with existing therapies may provide additional insights on outcomes including pain, disease severity, and quality of life. The therapeutic landscape in FD appears to be actively expanding with more treatment options expected to become available in the near future, allowing for a more personalized approach in FD patients.
  • ||||||||||  Review, Journal:  Developments in the treatment of Fabry disease. (Pubmed Central) -  Oct 7, 2021   
    Chaperone therapy (Migalastat; 1-deoxygalactonojirimycin) is the only other currently approved therapy for Fabry disease...Treatments currently under evaluation in (pre)clinical trials are second generation enzyme replacement therapies (Pegunigalsidase-alfa, Moss-aGal), substrate reduction therapies (Venglustat and Lucerastat), mRNA- and gene-based therapy. This review summarizes the knowledge on currently available and potential future options for the treatment of Fabry disease.
  • ||||||||||  lucerastat (ACT-434964) / Idorsia
    Enrollment status:  A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease (clinicaltrials.gov) -  May 9, 2021   
    P3,  N=108, Enrolling by invitation, 
    Trial registration The study was registered with the ClinicalTrials.gov identifier NCT03832452 (February 6th, 2019, https://clinicaltrials.gov/ct2/show/NCT03832452 ) and the EudraCT number 2018-004546-42 (December 17th, 2018). Recruiting --> Enrolling by invitation
  • ||||||||||  lucerastat (ACT-434964) / Idorsia
    Enrollment closed, Enrollment change, Monotherapy:  Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (clinicaltrials.gov) -  Jan 13, 2021   
    P3,  N=182, Active, not recruiting, 
    Trial completion date: Jul 2025 --> Nov 2025 | Trial primary completion date: Jun 2025 --> Oct 2025 Recruiting --> Active, not recruiting | N=99 --> 182
  • ||||||||||  lucerastat (ACT-434964) / Idorsia
    Trial completion date, Trial primary completion date, Monotherapy:  Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (clinicaltrials.gov) -  Nov 19, 2020   
    P3,  N=99, Recruiting, 
    These results indicate that the single-dose PK of lucerastat are not changed to a clinically relevant extent by cimetidine-mediated OCT2 inhibition, allowing the concomitant use of OCT2 inhibitors with lucerastat without any need for dose adjustment. Trial completion date: Jun 2021 --> Sep 2021 | Trial primary completion date: May 2021 --> Aug 2021
  • ||||||||||  lucerastat (ACT-434964) / Idorsia
    Trial completion date, Trial primary completion date, Monotherapy:  Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (clinicaltrials.gov) -  Dec 22, 2019   
    P3,  N=108, Recruiting, 
    Trial completion date: Jun 2021 --> Sep 2021 | Trial primary completion date: May 2021 --> Aug 2021 Trial completion date: Jun 2020 --> Jun 2021 | Trial primary completion date: May 2020 --> May 2021
  • ||||||||||  lucerastat (ACT-434964) / Idorsia
    Trial completion, Trial completion date, Trial primary completion date:  A Study in Healthy Subjects to Investigate Whether Administration of Lucerastat Can Affect Normal Heart Function (clinicaltrials.gov) -  Nov 1, 2019   
    P1,  N=44, Completed, 
    Trial completion date: Jun 2020 --> Jun 2021 | Trial primary completion date: May 2020 --> May 2021 Active, not recruiting --> Completed | Trial completion date: Sep 2019 --> Apr 2019 | Trial primary completion date: Sep 2019 --> Apr 2019
  • ||||||||||  lucerastat (ACT-434964) / Idorsia
    Trial completion date, Trial primary completion date, Monotherapy:  Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (clinicaltrials.gov) -  Jul 17, 2019   
    P3,  N=108, Recruiting, 
    Active, not recruiting --> Completed | Trial completion date: Sep 2019 --> Apr 2019 | Trial primary completion date: Sep 2019 --> Apr 2019 Trial completion date: Jan 2020 --> Jun 2020 | Trial primary completion date: Dec 2019 --> May 2020