EDG-7500 for Hypertrophic Cardiomyopathy

Recruiting at 17 trial locations

Overseen By Edgewise Therapeutics, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Edgewise Therapeutics, Inc.

Must not be taking: Cardiac myosin inhibitors

Disqualifiers: Coronary artery disease, Atrial fibrillation, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

75 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This study is being conducted in order to understand the safety and effects of different doses of EDG-7500 as a single dose in adults with obstructive hypertrophic cardiomyopathy (oHCM) and as multiple doses in adults with obstructive or nonobstructive hypertrophic cardiomyopathy (nHCM).

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, it does exclude participants who have used cardiac myosin inhibitors before, so you may need to discuss your current medications with the trial team.

How does the drug EDG-7500 differ from other treatments for hypertrophic cardiomyopathy?

EDG-7500 is unique because it may target the specific pathophysiology of hypertrophic cardiomyopathy, potentially offering a more precise treatment compared to traditional options like beta-blockers or septal reduction therapies. This approach could improve symptoms and functional capacity by addressing the underlying disease mechanisms. ^{1 2 3 4 5}

Who Is on the Research Team?

Medical Director

Principal Investigator

Edgewise Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for adults with obstructive hypertrophic cardiomyopathy, a condition where the heart muscle becomes abnormally thick. Participants should not have other serious medical conditions and must be able to follow the study procedures.

Inclusion Criteria

Documented left ventricular ejection fraction (LVEF) ≥0.60 at Screening

LVOT peak gradient ≥30 mmHg measured at rest and ≥50 mmHg post-Valsalva as determined by echocardiography at Screening

Body mass index (BMI) ≥18 to <35 kg/m2; weight ≥50 kg at Screening

Exclusion Criteria

A history of syncope or sustained ventricular tachyarrhythmia <180 days prior to Screening

My heart valve condition is at least Stage B.

I have been diagnosed with atrial fibrillation recently or had treatment for it.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Participants receive a single dose of EDG-7500

Up to 10 days

Treatment Part B

Participants receive EDG-7500 once daily for up to 28 days

Up to 38 days

Treatment Part C

Participants receive EDG-7500 once daily for up to 28 days

Up to 28 days

Treatment Part D

Participants receive EDG-7500 daily for up to 48 weeks

Up to 49 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• EDG-7500

Trial Overview The study tests different doses of EDG-7500, administered either once or multiple times, to see how safe it is and what effects it has on patients with obstructive hypertrophic cardiomyopathy.

How Is the Trial Designed?

Group I: Part D: EDG-7500 Multiple Dose in Adults with Hypertrophic Cardiomyopathy Experimental Treatment 1 Intervention

EDG-7500 daily for up to 18 months in new participants and participants who have completed Part B or C.

Group II: Part C: EDG-7500 Multiple Dose in Adults with Nonobstructive Hypertrophic Cardiomyopathy Experimental Treatment 1 Intervention

EDG-7500 once daily for up to 28 days.

Group III: Part B: EDG-7500 Multiple Dose in Adults with Obstructive Hypertrophic Cardiomyopathy Experimental Treatment 1 Intervention

EDG-7500 once daily for up to 28 days.

Group IV: Part A: EDG-7500 Single Dose Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Edgewise Therapeutics, Inc.

Lead Sponsor

Trials

13

Recruited

1,000+

Published Research Related to This Trial

Hypertrophic cardiomyopathy (HCM) is the most common inherited heart disease, characterized by unexplained thickening of the heart muscle, which can lead to serious complications like heart failure and arrhythmias.

Recent advancements in pharmacological treatments specifically targeting HCM's underlying mechanisms have shown promising results in improving patients' functional capacity and symptoms, indicating a shift towards more effective management strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stage-specific therapy for hypertrophic cardiomyopathy. Argirò, A., Zampieri, M., Marchi, A., et al.[2023]

Hypertrophic cardiomyopathy (HCM) is primarily caused by mutations in sarcomere genes, leading to altered heart cell function and a range of symptoms, including heart failure and sudden cardiac death, making early identification and management crucial.

Current management strategies focus on preventing sudden death through risk stratification, genetic diagnosis, and timely interventions, which have significantly reduced mortality rates in the last decade, although no causal therapy exists for this inherited condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hypertrophic cardiomyopathy: genetics and clinical perspectives. Wolf, CM.[2020]

Myosin inhibitors have emerged as a promising new treatment for obstructive hypertrophic cardiomyopathy, showing effectiveness in relieving symptoms and improving patients' quality of life based on recent clinical trials.

Current treatment options for left ventricular outflow tract obstruction include both pharmacological therapies and surgical interventions, highlighting the need for a comprehensive approach to manage this common genetic condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Therapeutic approaches in hypertrophic cardiomyopathy: from symptom relief to precision therapy]. Argirò, A., Zampieri, M., Marchi, A., et al.[2023]

Hypertrophic cardiomyopathy is a genetic condition that can lead to serious heart issues, including sudden cardiac death, making it crucial to identify high-risk patients based on clinical factors like age, syncope, and family history.

The implantable cardioverter-defibrillator (ICD) is an effective treatment for reducing the risk of sudden cardiac death in high-risk patients with hypertrophic cardiomyopathy, replacing amiodarone as the preferred prophylactic measure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sudden death and hypertrophic cardiomyopathy: a review. Ly, HQ., Greiss, I., Talakic, M., et al.[2013]

Hypertrophic cardiomyopathy affects about 0.2% of the population and can lead to serious symptoms like angina and heart failure; effective diagnosis involves ECG, echocardiography, and cardiac MRI for risk assessment.

Treatment options include beta-blockers for symptom management, and for patients with significant outflow tract obstruction, surgical myectomy or catheter-based septum ablation can provide significant relief.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Diagnosis and treatment of hypertrophic cardiomyopathies]. Kühl, C., Luedde, M., Langer, C., et al.[2013]

Citations

1. England pubmed.ncbi.nlm.nih.gov

Stage-specific therapy for hypertrophic cardiomyopathy. [2023]

2. China pubmed.ncbi.nlm.nih.gov

Hypertrophic cardiomyopathy: genetics and clinical perspectives. [2020]

3. Italy pubmed.ncbi.nlm.nih.gov

[Therapeutic approaches in hypertrophic cardiomyopathy: from symptom relief to precision therapy]. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Sudden death and hypertrophic cardiomyopathy: a review. [2013]

5. Germany pubmed.ncbi.nlm.nih.gov

[Diagnosis and treatment of hypertrophic cardiomyopathies]. [2013]