What side effects are associated with this type of intervention?

Gene therapies and targeted molecular therapies for Hypertrophic Cardiomyopathy, such as those targeting the MYBPC3 mutation, can cause immune reactions, leading to inflammation or other immune responses. Other common side effects include fatigue, gastrointestinal issues like nausea and diarrhea, and liver enzyme abnormalities. Severe but less common side effects may involve cardiovascular complications and off-target effects impacting other organs.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved gene therapies or targeted molecular therapies for Hypertrophic Cardiomyopathy (HCM) aimed at correcting or mitigating the effects of the MYBPC3 mutation. Current treatments for HCM primarily include medications such as beta-blockers, calcium channel blockers, and antiarrhythmic drugs, as well as surgical options like septal myectomy and alcohol septal ablation. The trial TN-201 represents an investigational approach, focusing on gene therapy for MYBPC3 mutation-associated HCM, which is still under clinical evaluation.

What other types of research exist?

Promising alternatives to TN-201 for Hypertrophic Cardiomyopathy patients include: 1) Mavacamten, a myosin inhibitor that has shown efficacy in reducing left ventricular outflow tract obstruction and improving symptoms in HCM patients. 2) CRISPR/Cas9-based gene editing therapies targeting MYBPC3 mutations, which are in early stages of research but hold potential for precise genetic correction. 3) RNA interference (RNAi) therapies aimed at reducing the expression of mutant MYBPC3 protein. These alternatives are in various stages of development and may offer new avenues for treatment in the near future.

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Gene Therapy

TN-201 for Hypertrophic Cardiomyopathy (MyPEAK-1 Trial)

Phase 1

Recruiting

Research Sponsored by Tenaya Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Nonobstructive Hypertrophic Cardiomyopathy

MYBPC3 mutation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new drug, TN-201, in adults with a specific genetic heart condition. It aims to see if the drug is safe and how it affects their health over several years.

Who is the study for?

This trial is for adults with symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM) linked to MYBPC3 mutation. Participants must have certain heart function markers like NT-proBNP levels of at least 300pg/ml and a left ventricular ejection fraction of 50% or more, as well as an implantable cardiac defibrillator.

What is being tested?

The study is testing TN-201, a new treatment for nHCM. It's the first time this drug is being given to humans. The trial will look at how safe it is, how well people can tolerate it, and what effects it has on the body.

What are the potential side effects?

Since TN-201 is new and this is its first use in humans, potential side effects are not yet fully known. However, participants will be closely monitored for any signs of organ inflammation, allergic reactions or other adverse events.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a thickened heart muscle that isn't blocking blood flow.

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I have a MYBPC3 gene mutation.

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I have moderate to moderately severe heart symptoms.

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I have a working heart defibrillator and moderate heart symptoms.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment1 Intervention

Dose for Cohort 2 will be 6E13 vg/kg

Group II: Cohort 1Experimental Treatment1 Intervention

Dose for Cohort 1 will be 3E13 vg/kg

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Hypertrophic Cardiomyopathy (HCM) treatments often include beta-blockers, calcium channel blockers, and surgical interventions like septal myectomy. However, emerging treatments such as gene therapy and targeted molecular therapies, like TN-201, aim to correct or mitigate the effects of specific genetic mutations such as MYBPC3. These therapies work by either restoring normal function to the mutated gene or compensating for its dysfunction, thereby addressing the root cause of the disease rather than just alleviating symptoms. This is crucial for HCM patients as it offers the potential for more effective and long-lasting treatment outcomes, reducing the risk of complications such as heart failure and sudden cardiac death.

Variable cardiac myosin binding protein-C expression in the myofilaments due to MYBPC3 mutations in hypertrophic cardiomyopathy.Human molecular genetic and functional studies identify TRIM63, encoding Muscle RING Finger Protein 1, as a novel gene for human hypertrophic cardiomyopathy.