HTX-001: a first-in-human test to see if this daily shot is safe for adults with nonobstructive hypertrophic cardiomyopathy
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)
Run by Haya Therapeutics, Inc. · NCT07867288 · Data updated 2026-11-12
This study tests multiple doses of HTX-001, an injectable antisense therapy, to see if it is safe and how it behaves in the body for adults with nonobstructive hypertrophic cardiomyopathy already on standard care.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Enrollment | 10 (estimated) |
| Sponsor | Haya Therapeutics, Inc. Industry-sponsored |
| Locations | 3 sites (Boston, Massachusetts and 2 other locations) |
| Status | Recruiting |
| Started | October 30, 2026 |
| Main results expected | March 2027 |
| Study ends | March 2027 |
| Registry record updated | November 4, 2026 |
| Trial ID | NCT07867288 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults with symptomatic nonobstructive hypertrophic cardiomyopathy, on stable standard therapy, NYHA class II-III, and NT-proBNP > 300 pg/mL.
✕Probably not a fit
Patients without nonobstructive hypertrophic cardiomyopathy or those not meeting the stability or NYHA criteria may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a Phase 1, interventional study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of HTX-001 given by subcutaneous injection. Eligible participants have symptomatic nonobstructive hypertrophic cardiomyopathy and are on guideline-directed medical therapy, with NYHA class II-III and elevated NT-proBNP. The trial will examine multiple dosing regimens to characterize how the drug is absorbed, distributed, metabolized and excreted, and its pharmacodynamic effects. Key exclusion criteria include recent LVOT obstruction, recent ICD implantation, and conditions that could interfere with protocol adherence. The study is sponsored by Haya Therapeutics, with sites in Boston, Kansas City, and Cincinnati.
Why it matters
Potential benefit: If successful, HTX-001 could offer a new therapeutic option that safely improves disease biology and symptoms for adults with nonobstructive hypertrophic cardiomyopathy.
How similar studies have gone: Early-phase antisense therapies in cardiomyopathy have shown safety signals in other cardiac trials but HTX-001 in nHCM is a novel approach with no confirmed efficacy to date.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
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Lahey Clinic
Boston, Massachusetts, United States Recruiting -
Saint Luke's Health System
Kansas City, Missouri, United States Recruiting -
The Lindner Research Center at The Christ Hospital
Cincinnati, Ohio, United States Recruiting
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Enavogliflozin for treating diastolic dysfunction in nonobstructive hypertrophic cardiomyopathy
This study is testing if the medication enavogliflozin can help people with nonobstructive hypertrophic cardiomyopathy feel better and improve their heart function compared to a placebo over 24 weeks.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.