NCT07867288 · Haya Therapeutics, Inc.
A Phase 1, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001
What this study is about
The purpose of this study is to evaluate safety, tolerability, how the drug moves through the body and how the drug affects the body of multiple doses of HTX-001 in human adult participants.
View original scientific description
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.
Interventions
DRUG
HTX-001
HTX-001 is an investigational antisense oligonucleotide (ASO)
Primary outcome measures
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)
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Kansas City, Missouri
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Oct 9, 2026 · Source of record for eligibility and locations