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

November 3, 2026 updated by: Haya Therapeutics, Inc.

A Two-Part, Phase 1, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and WISPER Target Engagement of HTX-001 in Adult Healthy Participants (Part 1) and in Adult Participants With Nonobstructive Hypertrophic Cardiomyopathy (Part 2)

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 01805
        • Recruiting
        • Lahey Clinic
        • Contact:
        • Principal Investigator:
          • Martin Maron, MD
    • Missouri
      • Kansas City, Missouri, United States, 64111
        • Recruiting
        • Saint Luke's Health System
        • Contact:
        • Principal Investigator:
          • Erika Hutt Centeno, MD
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Recruiting
        • The Lindner Research Center at The Christ Hospital
        • Contact:
        • Principal Investigator:
          • Ankit Bhatia,, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Open-label evaluation of multiple doses of HTX-001 in participants with symptomatic nHCM

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

HTX-001 is an investigational antisense oligonucleotide (ASO)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and Tolerability
Time Frame: 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)
Through Day 43

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Determination of pharmacokinetic parameters
Time Frame: Through Day 43

Maximum (peak) plasma drug concentration (Cmax)
Through Day 43
Determination of pharmacokinetic parameters
Time Frame: Through Day 43

Time to reach maximum (peak) plasma concentration (Tmax)
Through Day 43
Determination of pharmacokinetic parameters
Time Frame: Through Day 43

Area under the plasma concentration time curve (AUC)
Through Day 43

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Haya Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 30, 2026

Primary Completion (Estimated)

March 1, 2027

Study Completion (Estimated)

March 1, 2027

Study Registration Dates

First Submitted

October 5, 2026

First Submitted That Met QC Criteria

October 5, 2026

First Posted (Actual)

October 9, 2026

Study Record Updates

Last Update Posted (Actual)

November 4, 2026

Last Update Submitted That Met QC Criteria

November 3, 2026

Last Verified

November 1, 2026

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • HTX-001-A-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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