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Enrolling by invitation NCT04848506

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

Phase II / Phase III Interventional Symptomatic Hypertrophic Cardiomyopathy (HCM)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Aficamten (5 - 20 mg).
Who it may be relevant to
Registry conditions: Symptomatic Hypertrophic Cardiomyopathy (HCM). Basic parameters: 18 years — 85 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Argentina, Australia, Brazil, Canada +14
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Follow-Up, Open-Label, Research Evaluation of Sustained Treatment With Aficamten (CK-3773274) in Hypertrophic Cardiomyopathy (HCM)

Overview

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

Interventions

  • Drug Aficamten (5 - 20 mg)
    Aficamten tablets administered orally. During titration phase, clinic visits will occur approximately every 2-6 weeks. In the maintenance phase clinic visits will occur every 24 weeks, with safety check-in occurring every 12 weeks between visits by phone or, if desirable, in the clinic.

Primary outcome measures

  • Incidence of adverse events observed during dosing of aficamten in patients with HCM [Time frame: Baseline to End of study, up to 5 years]
Secondary outcome measures (11)
  • Incidence of serious adverse events observed during dosing of aficamten in patients with HCM [Time frame: Baseline to End of study, up to 5 years]
  • Incidence of left ventricular ejection fraction (LVEF) < 50% observed during dosing of aficamten in patients with HCM [Time frame: Baseline to End of study, up to 5 years]
  • Long-term effects of aficamten on left ventricular outflow tract gradient (LVOT G) in patients with oHCM [Time frame: Baseline through the end of participation at 12-24 week intervals]
  • Long-term effects of aficamten on resting LVOT-G [Time frame: Baseline through the end of participation at 12-24 week intervals]
  • Long-term effects of aficamten on post Valsalva LVOT-G [Time frame: Baseline through the end of participation at 12-24 week intervals]
  • Long-term effects of aficamten on post Valsalva LVOT-G [Time frame: Baseline through the end of participation at 12-24 week intervals]
  • Long-term effects of aficamten on left ventricular ejection fraction (LVEF) and post-Valsalva LVOT-G [Time frame: Baseline through the end of participation at 12-24 week intervals]
  • Long-term effects of aficamten on time to first resting LVOT-G < 30 mmHg through last follow-up [Time frame: Time to the following event through last follow-up, up to 5 years]
  • Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 50 mmHg through last follow-up [Time frame: Time to the following event through last follow-up, up to 5 years]
  • Long-term effects of aficamten on time to first post-Valsalva LVOT-G < 30 mmHg through last follow-up [Time frame: Time to the following event through last follow-up, up to 5 years]
  • 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 [Time frame: Time to the following event through last follow-up, up to 5 years]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 52 centers
  • University of Alabama at Birmingham — Birmingham
  • Alaska Heart and Vascular Institute — Anchorage
  • Mayo Clinic Building - Phoenix — Phoenix
  • UC San Diego Health - Sulpizio Cardiovascular Center — La Jolla
  • Cedar-Sinai Medical Center — Los Angeles
  • Cedars-Sinai Medical Center (Smidt Heart Institute) — Los Angeles
  • UCSF Medical Center — San Francisco
  • Yale New Haven Hospital — New Haven
  • … and 44 more centers
Spain · 12 centers

Center list to be confirmed — check the primary protocol.

France · 9 centers
  • Hopital de la Timone Service de cardiologie — Marseille
  • Clinique Du Millenaire — Montpellier
  • CHU Montpellier - Hopital Arnaud de Villeneuve — Montpellier
  • CHU de Nantes Institut Du Thorax et du systeme nerveux Clinique Cardiologique et des Malad — Nantes
  • Hopital Lariboisiere — Paris
  • Hopital Europeen Georges Pompidou — Paris
  • Hopital cardiologique de Haut-Leveque — Pessac
  • Centre hospitalier universitaire (CHU) de Rennes-Hopital Pontchaillou — Rennes
  • … and 1 more center
United Kingdom · 8 centers

Center list to be confirmed — check the primary protocol.

Italy · 7 centers

Center list to be confirmed — check the primary protocol.

Germany · 6 centers
  • Charite-Universitaetsmedizin Berlin — Berlin
  • Universitaetsklinikum Essen — Essen
  • Universitaetsmedizin Goettingen Klinik fur Kardiologie und Pneumologie — Goettigen
  • Universitaesklinkum Heidelberg — Heidelberg
  • Universitaetsklinikum Jena — Jena
  • … and 1 more center
Denmark · 5 centers
  • Department of Cardiology Aalborg University Hospital — Aalborg
  • Department of Cardiology Aarhus University Hospital — Aarhus
  • The Heart Center, Department of Cardiology — Copenhagen
  • Copenhagen University Hospital — Copenhagen
  • Odense University Hospital — Odense
Israel · 5 centers

Center list to be confirmed — check the primary protocol.

Greece · 4 centers

Center list to be confirmed — check the primary protocol.

Portugal · 4 centers

Center list to be confirmed — check the primary protocol.

Australia · 3 centers
  • Flinders Medical Centre, Cardiology Research — Bedford Park
  • The Prince Charles Hospital — Chermside
  • Fiona Stanley Hospital, AHF Unit — Murdoch
Canada · 3 centers
  • Institut universitaire de cardiologie et de pneumologie de Quebec-Universite Laval — Québec
  • Royal Alexandra Hospital — Edmonton
  • QEII Health Science Centre (Halifax Infirmary site) — Halifax
Netherlands · 3 centers

Center list to be confirmed — check the primary protocol.

Hungary · 2 centers

Center list to be confirmed — check the primary protocol.

Poland · 2 centers

Center list to be confirmed — check the primary protocol.

Argentina · 1 center
  • Instituto Cardiovascular de Buenos Aires(ICBA) — Buenos Aires
Brazil · 1 center
  • Instituto Dante Pazzanese de Cardiologia — São Paulo
Czechia · 1 center
  • Il. Interni klinika kardiologie a angiologie — Prague
Iceland · 1 center

Center list to be confirmed — check the primary protocol.

Publications

  • Saberi S, Abraham TP, Choudhury L, Barriales-Villa R, Elliott PM, Nassif ME, Oreziak A, Owens AT, Tower-Rader A, Rader F, Garcia-Pavia P, Olivotto I, Coats CJ, Fifer MA, Sherrid MV, Solomon SD, Watkins H, Heitner SB, Jacoby DL, Kupfer S, Malik FI, Melloni C, Meng L, Wei J, Maron MS, Masri A; FOREST-HCM Steering Committee and Investigators. Aficamten Treatment for Symptomatic Obstructive Hypertroph PMID 40540987
  • Masri A, Maron MS, Abraham TP, Nassif ME, Barriales-Villa R, Bilen O, Coats CJ, Elliott P, Garcia-Pavia P, Massera D, Olivotto I, Oreziak A, Owens AT, Saberi S, Solomon SD, Tower-Rader A, Heitner SB, Jacoby DL, Melloni C, Wei J, Sherrid MV; REDWOOD-HCM, SEQUOIA-HCM, and FOREST-HCM Investigators. Concomitant Aficamten and Disopyramide in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC Hea PMID 40285763
  • Masri A, Choudhury L, Barriales-Villa R, Elliott P, Maron MS, Nassif ME, Oreziak A, Owens AT, Saberi S, Tower-Rader A, Rader F, Garcia-Pavia P, Olivotto I, Nagueh SF, Wang A, Heitner SB, Jacoby DL, Kupfer S, Malik FI, Melloni C, Meng L, Wei J, Sherrid MV, Abraham TP; FOREST-HCM Investigators. Standard-of-Care Medication Withdrawal in Patients With Obstructive Hypertrophic Cardiomyopathy Receiving PMID 39477631

Identifiers

NCT: NCT04848506 · CY 6022 · 2020-003571-17 · 2023-508963-58-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗