Evidence map›Paper›PMID 41120118›Full record

Trial reportEuropean heart journal2025

Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial.

Martin S Maron, Juan Ramon Gimeno, Josef Veselka, Roberto Barriales-Villa, Brian L Claggett, Caroline J Coats, Sheila M Hegde, James L Januzzi, Ian J Kulac, Ahmad Masri and 8 more

Registry-linked trialAbstract readClinical Trial, Phase IIIMulticenter StudyRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in European heart journal, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05186818 (A Phase 3, Multi-Center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy and Safety of CK-3773274 in Adults With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction), which is not on this map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05186818 phase3completednot on this map

A Phase 3, Multi-Center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy and Safety of CK-3773274 in Adults With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

TypeinterventionalSponsorCytokineticsRan2022 to 2023Enrolled282ConditionsObstructive Hypertrophic Cardiomyopathy (oHCM)ArmsAficamten (5 mg, 10 mg, 15 mg, and 20 mg), Placebo to match aficamten
3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Review
  4. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors.

Martin S MaronCardiovascular Division, Lahey Hospital and Medical Center, HCM Center at Lahey Clinic, 41 Mall Road, Burlington, MA 01805, USA.
Juan Ramon GimenoCardiac Department, University Hospital Virgen Arrixaca, CIBERCV, ERN Guard-Heart, Murcia, Spain.
Josef VeselkaInstitute of Health Information and Statistics, Prague, Czech Republic.
Roberto Barriales-VillaComplexo Hospitalario Universitario A Coruña, INIBIC, CIBERCV-ISCIII, A Coruña, Spain.ORCID 0000-0002-6721-3487
Brian L ClaggettCardiovascular Division, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
Caroline J CoatsSchool of Cardiovascular and Metabolic Health, University of Glasgow, Glasgow, Scotland.
Sheila M HegdeBrigham and Women's Hospital, Boston, MA, USA.
James L JanuzziDivision of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Ian J KulacCardiovascular Division, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
Ahmad MasriCardiovascular Division, Oregon Health and Science University, Portland, OR, USA.ORCID 0000-0002-6390-6526
Michael E NassifUniversity of Missouri Kansas City Healthcare Institute for Innovations in Quality and Saint Luke's Mid America Heart Institute, Kansas City, MO, USA.
John A SpertusSaint Luke's Mid America Heart Institute, Kansas City, MO, USA.
Daniel L JacobyCytokinetics, Incorporated, South San Francisco, CA, USA.
Stephen B HeitnerCytokinetics, Incorporated, South San Francisco, CA, USA.
Stuart KupferCytokinetics, Incorporated, South San Francisco, CA, USA.ORCID 0000-0002-5469-1287
Fady I MalikCytokinetics, Incorporated, South San Francisco, CA, USA.
Amy WohltmanCytokinetics, Incorporated, South San Francisco, CA, USA.
Iacopo OlivottoMeyer Children's Hospital, Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Florence, Italy.ORCID 0000-0003-1751-9266

Funding

Cytokinetics, Incorporated
6 · The paper itself

Abstract

BACKGROUND AND

aimsPatients with obstructive hypertrophic cardiomyopathy (oHCM) treated with aficamten in SEQUOIA-HCM (NCT05186818) demonstrated marked improvement in symptoms and functional capacity. This analysis explores whether oHCM and mild symptoms patients experience similar clinical benefits with aficamten as patients with more advanced limitations.

methodsPatients in SEQUOIA-HCM (N = 282) were grouped at baseline according to symptom severity. Mild symptoms (n = 118) were defined as New York Heart Association (NYHA) class II and Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) ≥ 80, and moderate to severe symptoms (n = 150) as NYHA class II/III/IV and KCCQ-CSS <80. Primary endpoint was change in peak oxygen uptake (pVO2) from baseline to Week 24; secondary endpoints included change in NYHA class, KCCQ-CSS, outflow tract gradients, and N-terminal pro-B-type natriuretic peptide (NT-proBNP).

resultsIn aficamten-treated patients, change at Week 24 was not different between moderate to severe (1.8 mL/kg/min; n = 71) and mild (1.6 mL/kg/min; n = 62) symptom groups (P = .8). Likewise, the change in secondary endpoints (NYHA class, resting or Valsalva gradients, and NT-proBNP) did not differ significantly between the two symptom groups. Both groups experienced statistically significant improvements in KCCQ-CSS, but the extent of improvement was greater in the advanced symptom group (P = .02 for interaction). Treatment-emergent serious adverse events were infrequent in both groups.

conclusionsPatients with oHCM and mild symptoms treated with aficamten achieved significant improvement across a range of clinically relevant outcomes and generally similar to patients with more advanced symptoms. Less severely symptomatic patients could be considered for aficamten treatment.

Indexed as

Cardiomyopathy, HypertrophicPyrazolesAgedFemaleHumansMaleMiddle AgedNatriuretic Peptide, BrainPeptide FragmentsTreatment OutcomeaficamtenNatriuretic Peptide, BrainPeptide Fragmentspro-brain natriuretic peptide (1-76)PyrazolesaficamtenClinical trialHypertrophic cardiomyopathyObstructive hypertrophic cardiomyopathyTherapy

Identifiers

PMID41120118
PMCPMC12539928

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.