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Aficamten

Phase 3

Obstructive Hypertrophic Cardiomyopathy | Small molecule | Cardiovascular |Milestone Pharmaceuticals Inc.|Last Updated: Jan 21, 2026

Success Probability

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment40

FDA Designations

No designations recorded

Clinical trial landscape

Aficamten · 1 trial · 1 indication

Phase 3 1
NCT06116968An Open-Label Study of Aficamten for Chinese Patients With Symptomatic oHCMObstructive Hypertrophic Cardiomyopathy
COMPLETED40 Analytics
PHASE3COMPLETED
An Open-Label Study of Aficamten for Chinese Patients With Symptomatic oHCM
Obstructive Hypertrophic Cardiomyopathy
NCT0611696840
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Study Endpoints

Primary Endpoints

Patient incidence of reported adverse events (AEs)
Baseline to End of study, up to 2 years
Patient incidence of reported serious adverse events (SAEs )
Baseline to End of study, up to 2 years
Patient incidence of LVEF<50% & LVEF <40%
Baseline to End of study, up to 2 years

Secondary Endpoints

Peak LVOT-G at rest and with Valsalva provocation
Change from baseline values to week 48 at 12-week intervals;
Proportion of patients with resting LVOT-G <50 mmHg
Change from baseline values to week 48 at 12-week intervals;
Proportion of patients with resting LVOT-G <30 mmHg
Change from baseline values to week 48 at 12-week intervals;
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AficamtenEXPERIMENTALPatients in this arm take daily dose of Aficamten. Each patient will start at the lowest dose (5 mg) in the pre-specified dose range and undergo dose titration up to a maximum of 20 mg.

Interventions

NameTypeDescription
AficamtenDRUG5-20mg
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Eligibility Criteria

Age Range18 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: 1. Completion of a Cytokinetics trial investigating CK-3773274 2. LVEF ≥55% at the Screening Visit Exclusion Criteria: 1. Has participated in another investigational device or drug study or received an investigational device or drug \<1 month (or 5 half-lives for drugs, whiche...

Countries:China
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Frequently asked questions about Aficamten

What is Aficamten used for?

Aficamten is an investigational small molecule being developed for the treatment of obstructive hypertrophic cardiomyopathy (oHCM), a condition where the heart muscle becomes thickened and obstructs blood flow. It is intended for symptomatic patients with this condition.

Who makes Aficamten?

Aficamten is being developed by Milestone Pharmaceuticals Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol MIST. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with obstructive hypertrophic cardiomyopathy.

What phase is Aficamten in?

Aficamten is currently in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities and is still being studied in clinical trials to determine its safety and effectiveness for treating obstructive hypertrophic cardiomyopathy.

What clinical trials is Aficamten in?

Aficamten has one completed Phase 3 clinical trial, identified as NCT06116968. This was an open-label study conducted in China involving 40 patients with symptomatic obstructive hypertrophic cardiomyopathy. The trial was not randomized or double-blinded and had no control group.

Is Aficamten the same as any other drug?

Aficamten is not known to have any alternative names. It is a distinct investigational small molecule being developed by Milestone Pharmaceuticals Inc. for the treatment of obstructive hypertrophic cardiomyopathy.