Aom0304 in Adult Patients With Symptomatic Hypertrophic Cardiomyopathy

NCT07675668 | Not Yet Recruiting Phase 2

• 1 other identifier: Aom0304-CT02-01
• Study Type: interventional
• Target: 56
• Locations: 0 countries
• Sites: N/A

Brief 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. 1.Which dose is safe and tolerant of Aom0304 in participants with HCM?
2. 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.
3. 3.Undergo screening up to 28 days before enrollment to confirm eligibility
4. 4.Adjust dose every 2 weeks assessed by the Investigator according to predefined criteria at Titration Phase (Week 1 Day 1 to Week 8).
5. 5.Continuation of the last tolerated and effective dose; no further escalation is permitted at Maintenance Phase (Week 8 to Week 12).
6. 6.Study drug discontinued and follow up at Off-treatment Follow-up Period (at the end of Week 12 to Week 16).

Conditions

Hypertrophic Cardiomyopathy (HCM); Obstructive HCM (oHCM); Non-obstructive HCM (nHCM)

Keywords

Hypertrophic cardiomyopathy (HCM); Aom0304; Left ventricular outflow tract gradient (LVOT-G); Left ventricular ejection fraction (LVEF)

Outcome Measures

Primary Outcomes (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.

  Baseline to week 12

Secondary Outcomes (7)

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

  Baseline to week 12

• Change from Baseline in Peak Oxygen Consumption (pVO2)

  Baseline to week 12

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

  Baseline to week 12

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

  Baseline to week 12

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

  Baseline to week 12.

Study Arms (3)

oHCM whose post-Valsalva LVOT-G ≥ 30 mmHg and < 50 mmHg

EXPERIMENTAL

treat adult participants with oHCM whose post-Valsalva LVOT-G ≥ 30 mmHg and \< 50 mmHg

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 mmHg

EXPERIMENTAL

treat adult participants with oHCM whose resting LVOT-G ≥ 50 mmHg, or resting ≥ 30 mmHg accompanying with post-Valsalva ≥ 50 mmHg

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/mL

EXPERIMENTAL

treat adult participants with nHCM, defined as resting, post-Valsalva, or exercise LVOT-G \< 30 mmHg and NT-proBNP \> 300 pg/mL

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

Interventions

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

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.

Also known as: Aom0304

oHCM whose resting LVOT-G ≥ 50 mmHg, or resting ≥ 30 mmHg accompanying with post-Valsalva ≥ 50 mmHg

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

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.

Also known as: Aom0304

oHCM whose post-Valsalva LVOT-G ≥ 30 mmHg and < 50 mmHg

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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).

Sponsors & Collaborators

• Amckaus PTY LTD.: lead
• Novotech (Australia) Pty Limited: collaborator

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic

Interventions

BID protein, human

Condition Hierarchy (Ancestors)

Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 16, 2026
• First Posted: June 30, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): April 30, 2028
• Last Updated: June 30, 2026

Record last verified: 2026-06