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Hypertrophic Cardiomyopathy (HCM)Phase 2NCT07675668

Aom0304 in Adult Patients With Symptomatic Hypertrophic Cardiomyopathy

A Phase 2 study testing QG101-23-0 enteric coated capsule (60/120/240/360 mg BID) and QG101-23-0 enteric coated capsule (120/240/360/480mg BID) in people with Hypertrophic Cardiomyopathy (HCM) and Obstructive HCM (oHCM), run by Amckaus PTY LTD..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial summary

The goal of this clinical trial is designed to characterise the safety, tolerability, efficacy, and PK of Aom0304 across oHCM and nHCM populations and to inform dose selection for future Phase 3 development. The main questions it aims to answer are: 1. Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
QG101-23-0 enteric coated capsule (60/120/240/360 mg BID) and QG101-23-0 enteric coated capsule (120/240/360/480mg BID)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
56 participants
Where
No sites listed yet
Study dates
1 Oct 2026to 30 Apr 2028
Phase
Phase 2
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
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to federal, local, and institutional guidelines before the first study-specific procedure.
  • Men or women participants aged 18 to 70 years (both inclusive) at Screening.
  • Documented diagnosed with HCM based on European Society of Cardiology/American College of Cardiology Foundation criteria: hypertrophied and non-dilated left ventricle in absence of other systemic or cardiac causes, with left ventricular wall thickness ≥ 15 mm at diagnosis or ≥ 13 mm with a positive family history of HCM, or a known gene mutation related to HCM.
  • Participants who are already treated with β-blockers, verapamil, diltiazem, or ranolazine should have been on stable doses for at least 4 weeks prior to Day 1 and anticipate remaining on the same medication regimen during the study.
  • Body weight must be ≥ 45 kg and body mass index 18 to 35 kg/m2 (both inclusive) at Screening.
  • LVEF ≥ 60% at Screening.
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  • Symptomatic HCM defined as NYHA functional Class II or III at Screening.
  • Part-specific requirements at Screening:
  • Part 1: Post-Valsalva LVOT-G ≥ 30 mmHg and < 50 mmHg.
  • Part 2: Resting LVOT-G ≥ 50 mmHg or resting ≥ 30 mmHg with post-Valsalva ≥ 50 mmHg.
  • Part 3: Resting/post-Valsalva or exercise LVOT-G < 30 mmHg with NT-proBNP > 300 pg/mL.
  • Have adequate acoustic windows for accurate TTEs.
  • Female participants must not be pregnant or lactating and if sexually active, must be using 1 of the following acceptable contraceptive methods from the Screening through 3 months after the last dose of the study drug. Hormonal contraceptives are not considered highly effective contraceptive methods for this study. Acceptable contraceptive methods are listed as below.
  • Double-barrier method (eg, vasectomy or male using a condom and female using a diaphragm or cervical cap).
  • Barrier (eg, male using a condom) plus non-hormonal intrauterine device or intrauterine system.
  • Females are surgically sterile for 6 months or postmenopausal for 2 years. Permanent sterilisation includes hysterectomy, bilateral oophorectomy, bilateral salpingectomy, and/or documented bilateral tubal occlusion at least 6 months prior to Screening.
  • Females are considered postmenopausal if they have had amenorrhea for at least 2 years or more following cessation of all exogenous hormonal treatments and follicle-stimulating hormone (FSH) levels are ≥ 40 IU/L at Screening (confirmed by at least 2 separate occasions during Screening Period).
  • Male participants with female partners (including postmenopausal partners) must agree to use highly effective contraceptive measures. from the Screening through 3 months after the last dose of study drug. Highly effective contraception is presented in Inclusion criterion #10.
  • As there may be a risk of drug being secreted in the ejaculate, male participants (including men who have had vasectomies) whose partners are currently pregnant, or not pregnant or capable of becoming pregnant, should use barrier methods for the duration of the study and 3 months following the last dose of study drug in order to prevent passing Aom0304 to the partner in the ejaculate.
  • Male and female participants must agree to not donate sperm or ova after the first dose of study drug until at least 3 months following last dose of study drug.
  • Must be able to complete the Dyspnoea Numeric Rating Scale (NRS) and the KCCQ per established guidelines.
  • Participants must be able to safely undergo CPET at Baseline and follow-up.

Who cannot join

  • Participants with clinically significant haematology or chemistry abnormalities at Screening, as determined by the Investigator, including but not limited to:
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN).
  • Total bilirubin > 2 × ULN.
  • Haemoglobin < 9 g/dL.
  • Platelet count < 100000/µL.
  • Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m².
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  • Known hypersensitivity to Aom0304, or any of the components of the formulation of Aom0304, or alcohol.
  • History of sustained ventricular tachyarrhythmia or cardiac arrest.
  • Implanted cardioverter defibrillator (ICD) placement within 3 months prior to Screening or planned ICD placement during the study.
  • History of myocardial diseases other than HCM, including but not limited to: myocarditis, ischemic cardiomyopathy, dilated cardiomyopathy, cardiac amyloidosis, restrictive cardiomyopathy, Takotsubo syndrome, arrhythmogenic right ventricular cardiomyopathy.
  • Poor controlled hypertension, defined as systolic blood pressure (BP) ≥ 160 mmHg or diastolic BP ≥ 100 mmHg at Screening or Baseline despite stable antihypertensive therapy.
  • Participants who have been treated with septal reduction therapy (surgical myectomy or percutaneous alcohol septal ablation) or have plans for either treatment during the study period.
  • History of syncope with exercise within past 6 months prior to Screening.
  • Active infection, defined as any acute bacterial, viral, or fungal infection of any organ system (eg, respiratory, urinary, gastrointestinal, skin/soft tissue, cardiovascular, or central nervous system) requiring systemic antimicrobial therapy or considered to be clinically significant by the Investigator.
  • Current or recent (within 3 months prior to Screening) treatment with cardiac myosin inhibitors (eg, mavacamten, aficamten).
  • Persistent atrial fibrillation (AF), or paroxysmal AF with resting heart rate (HR) > 100 bpm within 1 year prior to Screening.
  • Have QT interval corrected by Fridericia's (QTcF) formula > 500 ms, or any other ECG abnormality considered by the Investigator to pose a risk to participant safety (eg, second degree atrioventricular block type II).
  • Aortic stenosis or fixed subaortic obstruction.
  • History of left ventricular systolic dysfunction (LVEF < 45%).
  • History of obstructive coronary artery disease (stenosis of > 70% of luminal diameter or > 50% of luminal diameter with ischemic symptoms in one or more coronary arteries), documented history of myocardial infarction or stroke.
  • History of malignancy of any type, with the following exceptions: in situ cervical cancer more than 5 years prior to Screening or surgically excised non-melanomatous skin cancers more than 2 years prior to Screening.
  • Positive serologic test at Screening for infection with human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV).
  • Participants with positive alcohol or drug screen results considered clinically significant or indicative of ongoing abuse, as judged by the Investigator.
  • History or evidence of any other clinically significant disorder, condition, or disease (with the exception of those outlined above) that, in the opinion of the Investigator, would pose a risk to participants' safety or interfere with the study evaluation, procedures, or completion.
  • Participated in a clinical trial where the participants received any investigational drug (or is currently using an investigational device) within 30 days prior to Screening, or at least 5 times the respective elimination half-life (whichever is longer).
  • Current use of tobacco- or nicotine-containing products > 20 cigarettes/day or equivalent.
  • Prior treatment with cardiotoxic agents such as doxorubicin or similar, or current treatment with antiarrhythmic drugs that have negative inotropic activity, eg, flecainide or propafenone.
  • Unable to comply with the study restrictions/requirements, including the number of required visits to the clinical site.

About this study

Official title: A Phase 2, Multi-Regional, Open-label, Three-Part Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Aom0304 in Adult Patients With Symptomatic Hypertrophic Cardiomyopathy

The goal of this clinical trial is designed to characterise the safety, tolerability, efficacy, and PK of Aom0304 across oHCM and nHCM populations and to inform dose selection for future Phase 3 development. The main questions it aims to answer are:

  1. Which dose is safe and tolerant of Aom0304 in participants with HCM?
  2. Which dose is effective of 12 weeks of Aom0304 treatment in participants with oHCM or nHCM? Researchers will compare different doses of Aom0304 to see which works best. All participants will receive Aom0304, but at different dose levels depending on which cohort they joined.
Read more

Participants will:

  1. Undergo screening up to 28 days before enrollment to confirm eligibility
  2. Adjust dose every 2 weeks assessed by the Investigator according to predefined criteria at Titration Phase (Week 1 Day 1 to Week 8).
  3. Continuation of the last tolerated and effective dose; no further escalation is permitted at Maintenance Phase (Week 8 to Week 12).
  4. Study drug discontinued and follow up at Off-treatment Follow-up Period (at the end of Week 12 to Week 16).
Being tested
QG101-23-0 enteric coated capsule (60/120/240/360 mg BID), QG101-23-0 enteric coated capsule (120/240/360/480mg BID)
Keywords
Hypertrophic cardiomyopathy (HCM), Aom0304, Left ventricular outflow tract gradient (LVOT-G), Left ventricular ejection fraction (LVEF)
Medical subject headings
Cardiomyopathy, Hypertrophic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • QG101-23-0 enteric coated capsule (60/120/240/360 mg BID)Drug

    Also called Aom0304

    The first dose of Aom0304 is to be administered orally under supervision, the second dose it to be administered orally at home. Participants will be supplied with Aom0304 for BID dosing orally at home. During the treatment period of 12 weeks, participants will receive the assigned dosing according to cohort allocation and dose titration strategy. There will be a Dose titration phase and Maintenance phase from Day 2 to Week 12. The dose titration will be assessed by the Investigator every 2 weeks (ie, at Week 2, Week 4, Week 6, and Week 8) according to predefined safety and efficacy criteria. Dose adjustment for Aom0304 will follow the sequence of 60 mg, 120 mg, 240 mg, and a maximum of 360 mg BID.

  • QG101-23-0 enteric coated capsule (60/120/240/360 mg BID)Drug

    Also called Aom0304

    The first dose of Aom0304 is to be administered orally under supervision, the second dose it to be administered orally at home. Participants will be supplied with Aom0304 for BID dosing orally at home. During the treatment period of 12 weeks, participants will receive the assigned dosing according to cohort allocation and dose titration strategy. There will be a Dose titration phase and Maintenance phase from Day 2 to Week 12. The dose titration will be assessed by the Investigator every 2 weeks (ie, at Week 2, Week 4, Week 6, and Week 8) according to predefined safety and efficacy criteria. Dose adjustment for Aom0304 will follow the sequence of 60 mg, 120 mg, 240 mg, and a maximum of 360 mg BID.

  • QG101-23-0 enteric coated capsule (120/240/360/480mg BID)Drug

    Also called Aom0304

    The first dose of Aom0304 is to be administered orally under supervision, the second dose it to be administered orally at home. Participants will be supplied with Aom0304 for BID dosing orally at home. During the treatment period of 12 weeks, participants will receive the assigned dosing according to cohort allocation and dose titration strategy. There will be a Dose titration phase and Maintenance phase from Day 2 to Week 12. The dose titration will be assessed by the Investigator every 2 weeks (ie, at Week 2, Week 4, Week 6, and Week 8) according to predefined safety and efficacy criteria. Dose adjustment for Aom0304 will follow the sequence of 120 mg, 240 mg, 360mg and a maximum of 480 mg BID.

  • QG101-23-0 enteric coated capsule (60/120/240/360 mg BID)Drug

    Also called Aom0304

    The first dose of Aom0304 is to be administered orally under supervision, the second dose it to be administered orally at home. Participants will be supplied with Aom0304 for BID dosing orally at home. During the treatment period of 12 weeks, participants will receive the assigned dosing according to cohort allocation and dose titration strategy. There will be a Dose titration phase and Maintenance phase from Day 2 to Week 12. The dose titration will be assessed by the Investigator every 2 weeks (ie, at Week 2, Week 4, Week 6, and Week 8) according to predefined safety and efficacy criteria. Dose adjustment for Aom0304 will follow the sequence of 60 mg, 120 mg, 240 mg, and a maximum of 360 mg BID.

Study groups in NCT07675668
Study groupRoleWhat they receive
oHCM whose post-Valsalva LVOT-G ≥ 30 mmHg and < 50 mmHgExperimentaltreat adult participants with oHCM whose post-Valsalva LVOT-G ≥ 30 mmHg and < 50 mmHg
Receives: Drug: QG101-23-0 enteric coated capsule (60/120/240/360 mg BID)
oHCM whose resting LVOT-G ≥ 50 mmHg, or resting ≥ 30 mmHg accompanying with post-Valsalva ≥ 50 mmHgExperimentaltreat adult participants with oHCM whose resting LVOT-G ≥ 50 mmHg, or resting ≥ 30 mmHg accompanying with post-Valsalva ≥ 50 mmHg
Receives: Drug: QG101-23-0 enteric coated capsule (60/120/240/360 mg BID); Drug: QG101-23-0 enteric coated capsule (120/240/360/480mg BID)
resting, post-Valsalva, or exercise LVOT-G < 30 mmHg and NT-proBNP > 300 pg/mLExperimentaltreat adult participants with nHCM, defined as resting, post-Valsalva, or exercise LVOT-G < 30 mmHg and NT-proBNP > 300 pg/mL
Receives: Drug: QG101-23-0 enteric coated capsule (60/120/240/360 mg BID)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To evaluate the safety and tolerability of Aom0304 in participants with HCM

    Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), with LVEF < 50% with abnormal vital signs, abnormal laboratory tests results, abnormal electrocardiograms (ECGs) and with changes in LVEF.

    Measured: Baseline to week 12

Secondary outcomes (7)

  1. Change from Baseline in Left Ventricular Outflow Tract Gradient (LVOT-G) at Rest and Post-Valsalva in Participants with oHCM

    Change in LVOT-G (mmHg) measured by echocardiography at rest and after Valsalva maneuver.

    Measured: Baseline to week 12

  2. Change from Baseline in Peak Oxygen Consumption (pVO2)

    Change in pVO2 measured using cardiopulmonary exercise testing (CPET).

    Measured: Baseline to week 12

  3. Change from Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS)

    The KCCQ is a 23-item, self-administered disease-specific questionnaire. The Clinical Summary Score combines the Physical Limitation and Symptom domains. Scores are transformed to a range of 0 to 100, where higher scores indicate better health status and fewer symptoms.

    Measured: Baseline to week 12

  4. To assess the effects of 12 weeks of Aom0304 treatment on N-terminal pro B type natriuretic peptide (NT-proBNP)

    Changes in NT-proBNP from Baseline

    Measured: Baseline to week 12

  5. Proportion of participants achieving a LVOT-G response of resting LVOT-G < 30 mm Hg and post-Valsalva LVOT-Gs < 50 mm Hg

    Proportion of participants with resting left ventricular outflow tract gradient (LVOT-G) < 30 mmHg and post-Valsalva LVOT-Gs < 50 mm Hg measured by echocardiography at Week 12.

    Measured: Baseline to week 12.

  6. Proportion of Participants Achieving Post-Valsalva LVOT-G < 30 mmHg at Week 12

    Proportion of participants with post-Valsalva left ventricular outflow tract gradient (LVOT-G) < 30 mmHg measured by echocardiography at Week 12.

    Measured: Baseline to week 12.

  7. Proportion of Participants Achieving ≥ 20 mmHg Reduction in Post-Valsalva LVOT-G at Week 12

    Proportion of participants showing a reduction of ≥ 20 mmHg in post-Valsalva left ventricular outflow tract gradient (LVOT-G) from Baseline to Week 12, measured by echocardiography.

    Measured: Baseline to week 12.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Jun 2026
  2. Study start1 Oct 2026 (estimated)
  3. Primary completion30 Dec 2027 (estimated)
  4. Study completion30 Apr 2028 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
56 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07675668
Sponsor's protocol ID
Aom0304-CT02-01
Status last verified
Jun 2026
First submitted
16 Jun 2026
Last updated
30 Jun 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing QG101-23-0 enteric coated capsule (60/120/240/360 mg BID) and QG101-23-0 enteric coated capsule (120/240/360/480mg BID) in people with Hypertrophic Cardiomyopathy (HCM) and Obstructive HCM (oHCM), run by Amckaus PTY LTD..

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 22 inclusion and 28 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: opening soon, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To evaluate the safety and tolerability of Aom0304 in participants with HCM (time frame: Baseline to week 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07675668.

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 30 Jun 2026.

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