Aom0304 in Adult Patients With Symptomatic Hypertrophic Cardiomyopathy
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-70
SponsorAmckaus PTY LTD.
About this trial
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.
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
QG101-23-0 enteric coated capsule (60/120/240/360 mg BID)
DrugThe 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)
DrugThe 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)
DrugThe 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)
DrugThe 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.
Treatment groups
56 Participants
are divided into 3 treatment groupsGroup A: oHCM whose post-Valsalva LVOT-G ≥ 30 mmHg and < 50 mmHgExperimental treatment 1 intervention
Group B: oHCM whose resting LVOT-G ≥ 50 mmHg, or resting ≥ 30 mmHg accompanying with post-Valsalva ≥ 50 mmHgExperimental treatment 2 interventions
Group C: resting, post-Valsalva, or exercise LVOT-G < 30 mmHg and NT-proBNP > 300 pg/mLExperimental treatment 1 intervention
Trial outcomes
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.
Time frame
Baseline to week 12Secondary outcomes
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.
Time frame
Baseline to week 122
Change from Baseline in Peak Oxygen Consumption (pVO2)
Change in pVO2 measured using cardiopulmonary exercise testing (CPET).
Time frame
Baseline to week 123
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.
Time frame
Baseline to week 124
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
Time frame
Baseline to week 12Locations
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Amckaus PTY LTD.
Lead sponsor
Novotech (Australia) Pty Limited
Collaborator
Source ClinicalTrials.gov
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