A Phase 1, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001

NCT07867288 | Recruiting Phase 1 FDA Drug

• 1 other identifier: HTX-001-A-101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Nonobstructive Hypertrophic Cardiomyopathy

Keywords

HAYA; Haya Therapeutics; HTX-001; ASO; nHCM; nonobstructive HCM; HCM

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

  Adverse events (AEs), serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and AEs of special interest (AESIs)

  Through Day 43

Secondary Outcomes (3)

• Determination of pharmacokinetic parameters

  Through Day 43

• Determination of pharmacokinetic parameters

  Through Day 43

• Determination of pharmacokinetic parameters

  Through Day 43

Study Arms (1)

Open-label evaluation of multiple doses of HTX-001 in participants with symptomatic nHCM

EXPERIMENTAL

Open label evaluation of multiple doses of HTX-001, across 2-3 dose levels, in participants with symptomatic nHCM who are receiving standard of care therapy

Drug: HTX-001

Interventions

HTX-001 DRUG

HTX-001 is an investigational antisense oligonucleotide (ASO)

Open-label evaluation of multiple doses of HTX-001 in participants with symptomatic nHCM

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Haya Therapeutics, Inc.: lead

Study Sites (1)

Saint Luke's Health System

Kansas City, Missouri, 64111, United States

RECRUITING

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 5, 2026
• First Posted: October 9, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: October 9, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)