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 3 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
- 3 sitesUnited States
- Study dates
- 30 Oct 2026to Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Lahey Clinic
Boston, Massachusetts, 01805
Amanda Kirshkaln-Leahy, MS, ACRP-PM, 781-825-8656, Amanda.Kirshkaln-Leahy@lahey.org
Martin Maron, MD
- Recruiting
The Lindner Research Center at The Christ Hospital
Cincinnati, Ohio, 45219
Kasey Wright, 513-585-1777, kasey.wright@thechristhospital.com
Ankit Bhatia,, MD
- 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 start30 Oct 2026
- 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
- Nov 2026
- First submitted
- 5 Oct 2026
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT07867288
- Acronym / sponsor ID
- HTX-001-A-101
- Keywords
- HAYA, Haya Therapeutics, HTX-001, ASO, nHCM, nonobstructive HCM, HCM
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Haya Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Oct 2026 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 9 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-label evaluation of multiple doses of HTX-001 in participants with symptomatic nHCM | Experimental | Drug: HTX-001Open 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 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HTX-001HTX-001 is an investigational antisense oligonucleotide (ASO) | Drug | Open-label evaluation of multiple doses of HTX-001 in participants with symptomatic nHCM |
Primary outcome
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)
3 secondary outcomes
Determination of pharmacokinetic parameters
Time frame: Through Day 43
Determination of pharmacokinetic parameters
Time frame: Through Day 43
Determination of pharmacokinetic parameters
Time frame: Through Day 43
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The Lindner Research Center at The Christ Hospital
Cincinnati, United States
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HTX-001 in people with Nonobstructive Hypertrophic Cardiomyopathy, run by Haya Therapeutics, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability (time frame: Through Day 43). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2026.