Haya Therapeutics, Inc.
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.
HTX-001 is an investigational antisense oligonucleotide (ASO) administered subcutaneously. The purpose of this study is to evaluate safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple doses of HTX-001. This will evaluate multiple doses of HTX-001 in participants with symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM) who are receiving standard of care therapy.
Inclusion Criteria: * Diagnosis of nHCM * On a stable regimen of guideline-directed medical therapy for nHCM * NYHA functional class II or III * NT-proBNP \>300 pg/mL * Body weight of 50 to 100 kg, inclusive, and BMI ≤40 kg/m2, inclusive * Adequate renal function * Not pregnant, and willing to use acceptable birth control methods * Able and willing to comply with study procedures Exclusion Criteria: * Any evidence of left ventricular outflow tract obstruction (LVOTO) * Implantation of an implantable cardioverter-defibrillator (ICD) within 6 months * History or current evidence of a clinical…
HTX-001 is an investigational antisense oligonucleotide (ASO)
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