Health TrialsbyHealthPanel

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

ConditionBAG3 Mutation 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

This trial does not accept healthy volunteers

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

Cardiac ventricular arrhythmia that requires treatment

Trial design

Design model

Single group

Treatments tested in this trial

  • ALXN2350

    Drug

    ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.

Treatment groups

6 Participants
are divided into 1 treatment group
Group A: ALXN2350Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Time frame
Baseline up to Week 78

Secondary outcomes

1

Part B: Number of Participants With TEAEs and SAEs

Time frame
Baseline up to Week 78
2

Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78

Time frame
Weeks 52 and 78
3

Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death

Time frame
Baseline up to Week 78
4

CFB in cardiac serum biomarker NT-pro-BNP

Time frame
Weeks 52 and 78

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alexion Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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