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Gene Therapy Trial Report

Summary

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy


NCTID NCT07218887 (View at clinicaltrials.gov)
Description
Development Status πŸ”„ Active
Indication BAG3 Mutation Associated Dilated Cardiomyopathy
Disease Ontology Term πŸ”„ DOID:0110448
Drug Product Name πŸ”„ ALXN2350
Sponsor Alexion Pharmaceuticals, Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 6 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant πŸ”„ BAG3
Therapeutic Modality πŸ”„ Gene transfer
Therapy Route πŸ”„ in vivo
Mechanism of Action πŸ”„ Functional gene replacement
Route of Administration πŸ”„ Intravenous
Drug Product Type πŸ”„ Viral vector
Gene Delivery System Type πŸ”„ Viral transduction
Vector Type πŸ”„ Adeno-associated virus
Viral Vector Subtype AAV9
Dose 1 πŸ”„ Undisclosed dose escalation, 3 levels

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2025-10-17
Completion Date 2032-01-27
Last Update πŸ”„ 2026-08-28

Participation Criteria


Eligible Age 18 Years - 70 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites πŸ”„ 11
Locations πŸ”„ United States,Italy,United Kingdom,Spain

Regulatory Information


Has US IND True
FDA Designations
Recent Updates First patient dosed September 2026

Resources/Links