Aom0304 in Adult Patients With Symptomatic Hypertrophic Cardiomyopathy

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. 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).

Conditions

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

Interventions

DRUG

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

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.

DRUG

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

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.

DRUG

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

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.

DRUG

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

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.

Sponsors & Collaborators

• Novotech (Australia) Pty Limited

  collaborator INDUSTRY

• Amckaus PTY LTD.

  lead INDUSTRY

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SEQUENTIAL

Eligibility

Min Age

18 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2026-10-01

Primary Completion

2027-12-30

Completion

2028-04-30