Nonobstructive Hypertrophic CardiomyopathyPhase 1NCT07867288
Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001
A Phase 1 study testing HTX-001 in people with Nonobstructive Hypertrophic Cardiomyopathy, run by Haya Therapeutics, Inc. at 2 sites in United States.
Trial summary
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Nonobstructive Hypertrophic Cardiomyopathy
- What is being tested?
- HTX-001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- Oct 2026to Mar 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Lahey Clinic
Boston, Massachusetts, 01805
Amanda Kirshkaln-Leahy, MS, ACRP-PM, 781-825-8656, Amanda.Kirshkaln-Leahy@lahey.org
- Recruiting
Saint Luke's Health System
Kansas City, Missouri, 64111
Claire Clinical Research Nurse, 816-932-1627, claire.pederson@bjc.org
Erika Hutt Centeno, MD
About this study
Official title: 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.
Read the 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.
- Being tested
- HTX-001
- Keywords
- HAYA, Haya Therapeutics, HTX-001, ASO, nHCM, nonobstructive HCM, HCM
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HTX-001Drug
HTX-001 is an investigational antisense oligonucleotide (ASO)
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: HTX-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Through Day 43
Secondary outcomes (3)
Determination of pharmacokinetic parameters
Maximum (peak) plasma drug concentration (Cmax)
Measured: Through Day 43
Determination of pharmacokinetic parameters
Time to reach maximum (peak) plasma concentration (Tmax)
Measured: Through Day 43
Determination of pharmacokinetic parameters
Area under the plasma concentration time curve (AUC)
Measured: Through Day 43
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2026
- Study startOct 2026 (estimated)
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nonobstructive Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Haya Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07867288
- Sponsor's protocol ID
- HTX-001-A-101
- Status last verified
- Oct 2026
- First submitted
- 5 Oct 2026
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No