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 study about?

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.

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 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 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 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 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 Study?

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 Study?

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

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Fondazione Toscana Gabriele Monasterio	Pisa	Italy
Hyyphnej Vozn dtpmwcwx	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Germany	Not yet recruiting	01.12.2025
Italy	Recruiting	01.12.2025
Spain	Recruiting	01.12.2025

Trial locations

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.

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 ID:

2024-519674-40-00

Protocol code:

ALXN2350-DCM-201

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?