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Study of ALXN2350 gene therapy in adults with BAG3 mutation-associated dilated cardiomyopathy: A safety and efficacy trial

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial studies BAG3 Mutation-Associated Dilated Cardiomyopathy, a heart condition where the heart muscle becomes enlarged and weak due to a specific genetic mutation. The study will test a new gene therapy called ALXN2350, which is given as a single intravenous infusion. The therapy uses a modified virus called adeno-associated virus serotype 9 to deliver a corrective gene to heart cells.

The study aims to determine if this treatment is safe and can help improve heart function in adults with this specific type of heart disease. During the trial, participants will receive either the gene therapy or standard heart failure medications including sirolimus, prednisone, and co-trimoxazole. The medications will be given in tablet form or as oral solutions.

Throughout the study, doctors will monitor participants' heart health using various tests including blood samples and heart measurements. They will check for changes in heart function and track important markers of heart health in the blood. The study will also measure how the body responds to the treatment and watch for any side effects.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial medication phase

    You will receive heart failure standard medications for at least 12 weeks before the main treatment. These medications need to remain stable during this period.

    If you are taking water pills (diuretics), the dose should be stable for at least 2 weeks before proceeding to the next phase.

  2. Step 2

    Main treatment administration

    You will receive a single dose of ALXN2350 through an intravenous infusion (through a vein).

    The treatment will be administered in the form of a solution containing a modified virus (AAV9) that helps deliver the medicine.

  3. Step 3

    Additional medications

    You will receive several oral medications including:

    Rapamune (sirolimus) in various forms: coated tablets (0.5 mg, 1 mg, 2 mg) or oral solution

    Prednisone in different doses (1 mg, 2.5 mg, 5 mg, 10 mg, 20 mg, 50 mg tablets)

    Co-Trimoxazole tablets for infection prevention

  4. Step 4

    Monitoring period

    Your health will be monitored through regular check-ups for 52 and 78 weeks after treatment.

    The monitoring includes:

    Physical examinations

    Vital sign measurements

    Blood tests

    Heart tests (ECG - electrical heart activity recording)

    Testing for virus presence in blood, stool, saliva, and urine samples

    Checking for antibodies against the treatment

  5. Step 5

    Long-term follow-up

    The study will continue monitoring your heart health through various measurements until January 2034.

    Regular checks will track your heart function and any heart-related events that may occur.

Who can join the trial?

9 criteria

  • Age requirement: Must be between 18 and 70 years old when signing the consent form
  • Must have a confirmed BAG3 gene mutation (a genetic change that affects heart function) verified by laboratory testing
  • Must have been diagnosed with dilated cardiomyopathy (a condition where the heart muscle becomes enlarged and weakened)
  • Must be on stable heart failure medications for at least 12 weeks before joining the study. These medications include:
    • Heart failure standard treatments
    • Blood pressure medications
    • Beta-blockers (medications that control heart rate)
    • Other heart medications
    • If taking water pills (diuretics), the dose must be stable for at least 2 weeks before joining the study (small adjustments up to 50% are allowed if doctor approves)
    • Must have suitable heart imaging quality when performing echocardiography (an ultrasound test of the heart)

Who cannot join the trial?

16 criteria

  • History of heart transplant or currently on a heart transplant waiting list
  • Current or recent (within 3 months) treatment with any experimental drug therapy
  • Active infections requiring treatment with antibiotics
  • Severe kidney problems (as measured by estimated glomerular filtration rate below 30 mL/min)
  • Severe liver problems (liver enzyme levels more than 3 times the normal upper limit)
  • Pregnant women or women planning pregnancy during the study period
  • Currently breastfeeding mothers
  • Known allergies to similar medications or their components
  • Major surgery within 3 months before the start of the study
  • History of drug or alcohol abuse within the past year
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
  • Unable to provide informed consent
  • Participation in another clinical trial within the past 30 days
  • Unstable heart rhythm problems (cardiac arrhythmias)
  • Recent heart attack (within past 3 months)
  • Presence of blood clotting disorders
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Investigated drugs

ALXN2350 is a gene therapy product administered through intravenous (IV) infusion. This treatment is designed to help patients who have dilated cardiomyopathy (a heart condition) that is associated with BAG3 gene mutations. The therapy aims to address the underlying genetic cause of the heart condition by delivering genetic material directly into the bloodstream through a single infusion.

What is already known about the treatment

ALXN2350 - A gene therapy medication administered through intravenous (IV) infusion, currently being investigated in clinical trials for treating BAG3 mutation-associated dilated cardiomyopathy (DCM). This experimental therapy is designed to target the genetic cause of heart muscle weakness in patients with BAG3 mutations, representing a novel therapeutic approach in cardiology. The treatment consists of a single IV infusion aimed at addressing the underlying genetic defect responsible for the cardiac condition, with ongoing studies evaluating both its safety profile and potential effectiveness compared to conventional treatments.

Investigated diseases

BAG3 Mutation Associated Dilated Cardiomyopathy – A genetic heart condition caused by mutations in the BAG3 gene that affects the heart muscle. The disease causes the heart chambers to enlarge and the heart muscle to become thin and weak, reducing its ability to pump blood effectively throughout the body. This condition leads to progressive weakening of heart function and decreased exercise capacity. The disease typically affects the left ventricle of the heart, though it can involve both ventricles. This inherited form of cardiomyopathy can occur at any age and may progress at different rates in different individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-519674-40-00Protocol codeALXN2350-DCM-201Estimated enrolment18 patientsSponsorAlexion Pharmaceuticals Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).