A Phase 1 interventional study of HTX-001 in Nonobstructive Hypertrophic Cardiomyopathy, sponsored by Haya Therapeutics, Inc.. Recruiting at 3 sites in United States. Open to participants aged 18 Years to 70 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-11-04.
Sponsored by Haya Therapeutics, Inc. · Phase 1, Interventional, and Treatment
Among 125 studies in Cardiomyopathy, Hypertrophic with a site in United States — 23 open to participants now, 37 under way, 6 of them phase 3. Cardiomyopathy, Hypertrophic · United States.
Among the 125 studies in Cardiomyopathy, Hypertrophic with a site in United States, this one is the 58th largest by planned enrollment of the 65 interventional ones that give a count, is one of 7 that started in 2026, is one of 10 in phase 1, reaches primary completion later than 15 of the 36 other live ones, and was the 125th registered.
363 studies on the registry are indexed under Cardiomyopathy, Hypertrophic; 116 are open to participants now.
Browse Cardiomyopathy, Hypertrophic studies →23 of the 125 studies in Cardiomyopathy, Hypertrophic with a site in United States are open now. Recruiting first, the newest first:
Braveheart Bio, Inc. · Sites in United States
Christopher Kramer · Sites in United States, United Kingdom
Lexeo Therapeutics · Sites in United States
Oregon Health and Science University · Sites in United States
Prolaio · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 81 Cardiomyopathy, Hypertrophic studies recruiting now, 55 (68%) set no upper age limit and 8 (10%) stop at 75 or younger; 21 take children under 18; 13 take healthy volunteers.
This study: 18 Years to 70 Years · all sexes · healthy volunteers too.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
125 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 11 · Phase 2 27 · Phase 3 14 · Phase 4 5
First in 1987; this study’s year is marked. 7 started in 2026.
This study's planned enrollment of 10 is below the median of 60 across 180 interventional studies indexed under Cardiomyopathy, Hypertrophic.
Median enrollment 50 across the 65 interventional studies, 150 across the 58 observational. Of the 80 that completed or were terminated, 25 (31%) have results posted.
This tab as a report to print or save as PDF → With an account.
49 of 125 have an industry lead sponsor. The 10 most frequent:
This is the only study on the registry with Haya Therapeutics, Inc. as lead sponsor.
Haya Therapeutics, Inc. has 1 study filed under Cardiomyopathy, Hypertrophic — fewer than ten, so there is no comparison to draw.
10 of the studies in Cardiomyopathy, Hypertrophic with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06481891Phase 3Lexicon PharmaceuticalsA Study to Evaluate the Efficacy and Safety of Sotagliflozin in Symptomatic Obstructive and Non-obstructive Hypertrophic Cardiomyopathy | 2027-01 |
| NCT07023614Imbria Pharmaceuticals, Inc.A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy | 2026-12 |
| NCT07600177Oregon Health and Science UniversityMavacamten to Aficamten Transition in Patients With Obstructive Hypertrophic Cardiomyopathy | 2026-12-31 |
| NCT06551129Bristol-Myers SquibbReal-world Patient Reported Outcomes Among Patients Treated With Camzyos | 2026-12-31 |
| NCT07867288Haya Therapeutics, Inc.Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001 | 2027-03 |
Among the 125 studies in Cardiomyopathy, Hypertrophic with a site in United States, from their registry updates:
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The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Drug: HTX-001
HTX-001 is an investigational antisense oligonucleotide (ASO)
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)
Time frame: Through Day 43
Determination of pharmacokinetic parameters
Maximum (peak) plasma drug concentration (Cmax)
Time frame: Through Day 43
Determination of pharmacokinetic parameters
Time to reach maximum (peak) plasma concentration (Tmax)
Time frame: Through Day 43
Determination of pharmacokinetic parameters
Area under the plasma concentration time curve (AUC)
Time frame: Through Day 43
Plan to share: No
No publications or documents are linked to this record.
Started Oct 2026 (actual). Primary completion is estimated for Mar 2027, 5 months after the start.
Of 12 phase 1 interventional studies under Cardiomyopathy, Hypertrophic, half reached primary completion within 5 months of starting (the median), counting those still running; a quarter within 3 and three quarters within 12. This study's planned 5 months is close to that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
No contact was published for this record. The registry link below has the sponsor’s details.
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Cardiomyopathy, Hypertrophic →