Ninerafaxstat for Hypertrophic Cardiomyopathy
(FORTITUDE-HCM Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the effectiveness of a new drug, ninerafaxstat, for individuals with hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens excessively. Researchers seek to determine if ninerafaxstat, combined with usual care, is more effective than a placebo in reducing symptoms. The trial seeks participants diagnosed with HCM who experience symptoms like shortness of breath or chest pain during physical activity and can engage in walking or cycling for exercise. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to the development of a potentially effective therapy.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. It mentions that the study will assess ninerafaxstat on top of Standard of Care, which suggests you may continue your existing treatment, but it's best to confirm with the trial organizers.
Is there any evidence suggesting that ninerafaxstat is likely to be safe for humans?
Research has shown that ninerafaxstat is generally safe and well-tolerated in people with non-obstructive hypertrophic cardiomyopathy (nHCM). Studies have found that this drug not only has a good safety record but also improves symptoms and exercise capacity. Participants in earlier studies reported feeling healthier, suggesting the drug might offer benefits beyond safety alone. It is important to discuss any concerns with a doctor before joining a clinical trial.12345
Why do researchers think this study treatment might be promising?
Unlike the standard treatments for hypertrophic cardiomyopathy, which often include beta-blockers, calcium channel blockers, or surgical options, Ninerafaxstat offers a novel approach. Researchers are excited about Ninerafaxstat because it targets the underlying molecular pathways of the disease, potentially offering more precise management of symptoms. This experimental drug could provide an alternative for patients who don't respond well to existing therapies, paving the way for improved quality of life and better outcomes.
What evidence suggests that ninerafaxstat might be an effective treatment for hypertrophic cardiomyopathy?
Research has shown that ninerafaxstat, which participants in this trial may receive, may help people with hypertrophic cardiomyopathy (HCM). Studies have found that it can improve exercise ability and reduce symptoms in patients with non-obstructive HCM. The drug helps the heart use energy more efficiently, making activity easier for patients. Trials have shown it to be well tolerated, with a good safety record. Additionally, no major negative changes in heart function were observed, suggesting it could be a promising way to manage HCM symptoms.12367
Are You a Good Fit for This Trial?
This trial is for patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM), confirmed by echocardiography. Participants should experience functional limitations, classified as NYHA Class II or III. They must meet specific diagnostic criteria and have their condition verified by a core laboratory.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ninerafaxstat or placebo on top of Standard of Care for 12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Ninerafaxstat
Find a Clinic Near You
Who Is Running the Clinical Trial?
Imbria Pharmaceuticals, Inc.
Lead Sponsor