Evidence map›Paper›PMID 41347307›Full record

ArticleCirculation. Heart failure2026

Efficacy and Safety of Aficamten in Children and Adolescents With Obstructive Hypertrophic Cardiomyopathy: Study Design and Rationale of CEDAR-HCM.

Juan Pablo Kaski, Paul F Kantor, Stephanie J Nakano, Iacopo Olivotto, Mark W Russell, Justin Godown, Michael Chiu, Polina German, Stephen B Heitner, Daniel L Jacoby and 10 more

2 registry-linked trialsAbstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in Circulation. Heart failure, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–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
NCT06412666 phase2 / phase3active not recruitingnot on this map

A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled and Open-Label Extension Trial to Evaluate the Efficacy and Safety of Aficamten in a Pediatric Population With Symptomatic Obstructive Hypertrophic Cardiomyopathy

TypeinterventionalSponsorCytokineticsRan2024 to 2030Enrolled65ConditionsPediatric, Symptomatic Obstructive Hypertrophic CardiomyopathyArmsAficamten, Placebo
3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
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

20 authors.

Juan Pablo KaskiCentre for Paediatric Inherited and Rare Cardiovascular Disease, UCL Institute of Cardiovascular Science, University College London, United Kingdom (J.P.K.).ORCID 0000-0002-0014-9927
Paul F KantorDivision of Pediatric Cardiology, Children's Hospital Los Angeles, University of Southern California (P.F.K.).ORCID 0000-0002-4244-1563
Stephanie J NakanoDivision of Pediatric Cardiology, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora (S.J.N.).ORCID 0000-0002-2493-5842
Iacopo OlivottoCardiology Unit, Meyer Children's Hospital, Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Florence, Italy (I.O.).ORCID 0000-0003-1751-9266
Mark W RussellDivision of Pediatric Cardiology, University of Michigan Medical School, Ann Arbor (M.W.R.).
Justin GodownCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).
Michael ChiuCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).
Polina GermanCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).
Stephen B HeitnerCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).ORCID 0000-0003-2189-1474
Daniel L JacobyCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).ORCID 0000-0002-9182-275X
Stuart KupferCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).ORCID 0000-0002-5469-1287
Justin LutzCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).ORCID 0009-0009-9353-9139
Neha MaharaoCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).
Fady I MalikCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).ORCID 0000-0002-5597-5759
Chiara MelloniCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).
Paula F Nieto MoralesDepartment of Biomedical Sciences, College of Medicine, Florida State University, Tallahassee (P.F.N.M.).
Tyrell SimkinsCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).ORCID 0000-0002-6063-174X
Jenny WeiCytokinetics Inc, South San Francisco, CA (J.G., M.C., P.G., S.B.H., D.L.J., S.K., J.L., N.M., F.I.M., C.M., T.S., J.W.).ORCID 0009-0000-5828-5705
Carolyn Y HoDepartment of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA (C.Y.H.).ORCID 0000-0002-7334-7924
CEDAR-HCM Investigators

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundHypertrophic cardiomyopathy (HCM) is an important cause of morbidity and mortality in children, but treatment options are limited. Aficamten, a next-in-class cardiac myosin inhibitor, directly targets the hypercontractility underlying HCM. Aficamten improved exercise capacity, health status, and symptoms in adults with obstructive HCM in the pivotal, phase 3 SEQUOIA-HCM trial (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM; NCT05186818).

methodsCEDAR-HCM (Clinical Evaluation of dosing With Aficamten to Reduce Obstruction in Pediatric Population With HCM) is an international, multicenter, randomized, double-blind, placebo-controlled trial followed by an open-label extension to evaluate the efficacy, safety, and pharmacokinetics of aficamten in pediatric participants with symptomatic obstructive HCM. The trial will enroll ≈55 adolescents (12 to <18 years) and subsequently expand to include at least 10 children (6 to <12 years) with nonsyndromic obstructive HCM, left ventricular ejection fraction ≥60%, Valsalva left ventricular outflow tract gradient ≥50 mm Hg, and New York Heart Association functional class ≥II. Participants will be randomized 2:1 to aficamten or placebo in addition to standard of care therapy or as monotherapy, with echocardiogram-guided dose adjustments targeting a Valsalva left ventricular outflow tract gradient <30 mm Hg while maintaining left ventricular ejection fraction ≥50%. The primary end point is the change in Valsalva left ventricular outflow tract gradient from baseline to week 12. Secondary end points include change in resting left ventricular outflow tract gradient, cardiac biomarkers, New York Heart Association functional class, and assessment of pharmacokinetics. After completing the 12-week randomized period, eligible participants will continue into a long-term open-label extension.

resultsThe trial is currently enrolling.

conclusionsResults of CEDAR-HCM will provide insight into the safety and efficacy of aficamten in adolescents and in children as young as 6 years of age. REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifier: NCT06412666.

Indexed as

Cardiomyopathy, HypertrophicVentricular Function, LeftAdolescentChildDouble-Blind MethodExercise ToleranceFemaleHumansMaleMulticenter Studies as TopicRandomized Controlled Trials as TopicStroke VolumeTreatment Outcomeadolescentaficamtenechocardiographymorbidityventricular remodeling

Identifiers

PMID41347307
PMCPMC12908643

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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.