Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults with HCM



Sponsor:   Cytokinetics


Sommario: The purpose of this study is to collect long-term safety and tolerability data for
aficamten.




TIPOLOGIA STUDIO

 
Interventistico



FASE

 




TRATTAMENTO

  
    
  






OBIETTIVO PRIMARIO


  
  • Misura: Incidence of adverse events observed during dosing of aficamten in patients with HCM
  • Tempo: Baseline to End of study, up to 5 years
  • Descrizione: Patient incidence of reported Adverse Events (AEs)




OBIETTIVO SECONDARIO


  
  • Misura: Incidence of serious adverse events observed during dosing of aficamten in patients with HCM
  • Tempo: Baseline to End of study, up to 5 years
  • Descrizione: Patient incidence of reported Serious Adverse Events (SAEs)
  • Misura: Incidence of left ventricular ejection fraction (LVEF) < 50% observed during dosing of aficamten in patients with HCM
  • Tempo: Baseline to End of study, up to 5 years
  • Descrizione: Patient incidence of reported LVEF <50%
  • Misura: Long-term effects of aficamten on left ventricular outflow tract gradient (LVOT G) in patients with oHCM
  • Tempo: Baseline through the end of participation at 12-24 week intervals
  • Descrizione: Peak LVOT-G at rest; applicable only to patients with oHCM
  • Misura: Long-term effects of aficamten on resting LVOT-G
  • Tempo: Baseline through the end of participation at 12-24 week intervals
  • Descrizione: Proportion of patients with resting LVOT-G < 30 mmHg; applicable only to patients with
oHCM
  • Misura: Long-term effects of aficamten on post Valsalva LVOT-G
  • Tempo: Baseline through the end of participation at 12-24 week intervals
  • Descrizione: Proportion of patients with post-Valsalva LVOT-G < 50 mmHg; applicable only to patients
with oHCM
  • Misura: Long-term effects of aficamten on post Valsalva LVOT-G
  • Tempo: Baseline through the end of participation at 12-24 week intervals
  • Descrizione: Proportion of patients with post-Valsalva LVOT-G < 30 mmHg; applicable only to patients
with oHCM
  • Misura: Long-term effects of aficamten on left ventricular ejection fraction (LVEF) and post-Valsalva LVOT-G
  • Tempo: Baseline through the end of participation at 12-24 week intervals
  • Descrizione: Proportion of patients with LVEF ≥ 50%, resting LVOT-G < 30 mmHg, and post-Valsalva
LVOT-G < 50 mmHg; applicable only to patients with oHCM
  • Misura: Long-term effects of aficamten on time to first resting LVOT-G < 30 mmHg through last follow-up
  • Tempo: Time to the following event through last follow-up, up to 5 years
  • Descrizione: Applicable only to patients with oHCM
  • Misura: Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 50 mmHg through last follow-up
  • Tempo: Time to the following event through last follow-up, up to 5 years
  • Descrizione: Applicable only to patients with oHCM
  • Misura: Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 30 mmHg through last follow-up
  • Tempo: Time to the following event through last follow-up, up to 5 years
  • Descrizione: Applicable only to patients with oHCM
  • Misura: Long-term effects of aficamten on time to first LVEF ≥ 50%, resting LVOT-G < 30 mmHg, and post-Valsalva LVOT-G < 50 mmHg through last follow-up
  • Tempo: Time to the following event through last follow-up, up to 5 years
  • Descrizione: Applicable only to patients with oHCM




CRITERI DI ELIGIBILITA'


    
             
  • 
        Criteri di inclusione e esclusione
        
    Inclusion Criteria:

  -  Completion of a Cytokinetics trial investigating aficamten

  -  LVEF ≥ 55% at the Screening Visit

Exclusion Criteria:

  -  Has received treatment with mavacamten: (a) within 56 days prior to dosing and (b)
     has not received approval for participation from the Medical Monitor.

  -  Has participated in another investigational device or drug study or received an
     investigational device or drug < 1 month (or 5 half-lives for drugs, whichever is
     longer) prior to screening. Other investigational procedures while participating in
     this study are not permitted.

  -  Since completion of a previous trial of aficamten has:

       -  Developed new-onset paroxysmal or permanent atrial fibrillation requiring
          rhythm restoring treatment (eg, direct-current cardioversion, ablation
          procedure, or antiarrhythmic therapy) < 30 days prior to screening. Patient may
          re-screen for CY 6022 after 30 days if heart rate (HR) < 100 bpm and/or rhythm
          is stable > 30 days

       -  Undergone septal reduction therapy (surgical myectomy or transcatheter alcohol
          ablation)

  -  Had a confirmed LVEF < 40% with an associated dose interruption during participation
     in a prior study with aficamten

  -  History of implantable ICD placement within 30 days prior to screening.
    
        
    • 
    




SESSO

  
Tutti




  


ETA' MINIMA



Età Minima: 18 Years

Età Massima: 85 Years






LUOGO


  
Unit Cardiomiopatie, Dipartimento Cardio ToracoVascolare
Firenze, 50134
Fondazione toscana Gabriele Monesterio per la ricarca medica Dipartimento Cardiotoracico UOC Cardiologia e Medicina Cardiovasculare Ospedale San Cataldo
Pisa, 56124
Dipartimento di Medicina Clinica e Molecolare Universita Sapienza di Roma Unita di Terapia Intensiva Cardiologica Azienda Ospedaliero Universitaria Sant'Andrea
Roma, 00189