ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-70
SponsorAlexion Pharmaceuticals, Inc.
About this trial
This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.
Eligibility criteria
Qualifiers
Pathogenic or likely pathogenic mutation in BAG3
Medical history of diagnosis of DCM
Stable combination of HF SoC medications
Adequate acoustic windows for echocardiography
Disqualifiers
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
Trial design
Treatments tested in this trial
- ALXN2350
Treatment groups
6 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov