Recruiting
Phase 1

HTX-001

Sponsor:

Haya Therapeutics, Inc.

Code:

NCT07867288

Conditions

Nonobstructive Hypertrophic Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Accepted

Interventions

HTX-001

Study Details

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

Study ID

NCT07867288

Start date

Oct, 2026

Status verified date

Oct, 2026

Completion date

Mar, 2027

Anticipated

Primary completion date

Mar, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70

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

Study Design

Enrollment

10 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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

Interventions

HTX-001

HTX-001 is an investigational antisense oligonucleotide (ASO)

Primary outcome measure

  • Safety and Tolerability [ Time Frame: Through Day 43 ]

Central Contacts and Locations

Locations

Saint Luke's Health System

Recruiting

Kansas City, Missouri, United States, 64111

Contacts

Claire Clinical Research Nurse

816-932-1627claire.pederson@bjc.org

Principal Investigator:

Erika Hutt Centeno, MD

More Information

Sponsor

Haya Therapeutics, Inc.

Last update posted

Oct 9, 2026

Last verified

Oct, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-10. This information was provided to ClinicalTrials.gov by Haya Therapeutics, Inc. on 2026-10-09. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.