ALXN2350 for adults with BAG3-related dilated cardiomyopathy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an open-label Phase 1/2 dose-finding and dose-expansion study that gives a single intravenous infusion of ALXN2350 to adults with genetically confirmed BAG3-associated DCM. The primary focus is safety and tolerability across escalating doses, with expansion cohorts used to look for preliminary signs of clinical benefit using cardiac imaging and heart-failure measures. Key eligibility includes a pathogenic or likely pathogenic BAG3 variant, stable guideline-directed heart failure therapy, and adequate echocardiographic windows, while participants with pre-existing AAV9 antibodies or another known genetic cause of DCM are excluded. Study procedures include baseline and follow-up clinical assessments and imaging at U.S. sites to monitor efficacy signals and adverse events after the infusion.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Pathogenic or likely pathogenic mutation in BAG3
* Medical history of diagnosis of DCM
* Stable combination of HF SoC medications
* Adequate acoustic windows for echocardiography
* LVEF \> 15% and ≤ 45% at Screening by echocardiography
* Diagnosis of chronic HF for at least 3 months
* NYHA Class I/II/III at Screening

Exclusion Criteria:

* Presence of antibodies to AAV9
* Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
* Decompensated HF
* Cardiac ventricular arrhythmia that requires treatment
* History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency
* NYHA Class IV
* History of substance abuse that could contribute to cardiac dysfunction

Note: Other inclusion and exclusion criteria may apply.

Where it's running

• Research Site
  Birmingham, Alabama, United States Recruiting
• Research Site
  Palo Alto, California, United States Recruiting
• Research Site
  Boston, Massachusetts, United States Recruiting
• Research Site
  Cincinnati, Ohio, United States Recruiting
• Research Site
  Portland, Oregon, United States Recruiting
• Research Site
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
• Email: clinicaltrials@alexion.com
• Phone: 1-855-752-2356

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• AAV Gene Therapy for BAG3-Related Dilated Cardiomyopathy in Adults

  This Phase 1/2 trial tests a single IV dose of AFTX-201 to see if it is safe and potentially helps adults with BAG3-related dilated cardiomyopathy.

  Recruiting at 5 sites Phase 1/2 Affinia Therapeutics · Miami, Florida and 4 other locations · NCT07426419

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.