Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

One-Time gene infusion aims to repair hearts in rare genetic condition

NCT ID NCT07426419

Disease control Sponsor: Affinia Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Sep 02, 2026 · Updated 10 times

Summary

This early-stage trial tests a single intravenous dose of AFTX-201, a gene therapy designed to deliver a working copy of the BAG3 gene to heart cells. It enrolls 22 adults aged 18–55 with dilated cardiomyopathy and a BAG3 mutation. The study primarily checks safety and tolerability over 12 months, while also measuring changes in heart function and biomarkers.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
AFTX-201 (gene therapy)
What this could lead to
If successful, this could point toward a one-time gene therapy treatment to improve heart function in people with BAG3-related dilated cardiomyopathy.
What could go wrong
This is an early Phase 1/2 trial with only 22 participants, so safety and effectiveness are not yet proven. Gene therapies can have unexpected side effects, and long-term benefits are uncertain.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 22 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Dec 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: Male or female, 18 to 70 years of age Truncating mutation in BAG3 Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) \< 45% NYHA Class II or III heart failure symptoms NT-proBNP ≥ 300 pg/mL for subjects in sinus rhythm or paced rhythm, or ≥ 600 pg/mL for subjects in atrial fibrillation Willing and able to sign informed consent and comply with study procedures Exclusion Criteria: Prior myocardial infarction, heart transplant, or presence/requirement of a left ventricular assist device (LVAD) IV therapy with positive inotropes, vasodilators, or diuretics for heart failure within 30 days prior to enrollment Positive neutralizing antibodies to ATC-0187 Any condition that, in the investigator's opinion, would place the subject at undue risk or interfere with study participation or interpretation of results

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for BAG3 mutation associated dilated cardiomyopathy are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

dilated cardiomyopathy dilated cardiomyopathy 1HH

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BAG3 mutation associated dilated cardiomyopathy Dilated cardiomyopathy (DCM)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    4 sites in 2 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Houston Methodist Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • Institut de Cardiologie de Montréal / Montreal Heart Institute

    RECRUITING

    Montreal, Quebec, QC H1T 1C8, Canada

  • St. Lukes Hospital

    RECRUITING

    Kansas City, Missouri, 64111, United States

  • University of Miami Hospital and Clinics

    RECRUITING

    Miami, Florida, 33136, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.