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Clinical Trials in the USA / NCT07867288
Recruiting Phase 1

Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001

NCT07867288 · tracked via the Priya Life Science USA tracker
Sponsor
Haya Therapeutics, Inc
Phase
Phase 1
Started
2026-10
Last updated
2026-10-27

Condition(s) studied

Nonobstructive Hypertrophic Cardiomyopathy

Investigational drug(s) / intervention(s)

HTX-001

HTX-001: HTX-001 is an investigational antisense oligonucleotide (ASO)

Study summary

The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.

Eligibility

Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
Accepted
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 clinically significant or uncontrolled underlying condition * Unable to tolerate subcutaneous dosing * Any reason that in the opinion of the Investigator would lead to the inability of the participant to comply with the protocol

Primary outcome measure(s)

  • Safety and Tolerability — Through Day 43
    Adverse events (AEs), serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and AEs of special interest (AESIs)

Trial sites (3)

FacilityCityRegionStatus
Lahey Clinic Boston Massachusetts Recruiting
Saint Luke's Health System Kansas City Missouri Recruiting
The Lindner Research Center at The Christ Hospital Cincinnati Ohio Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07867288 on ClinicalTrials.gov ↗ ← All trials in the USA