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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 🔄 Active
Indication BAG3 Mutation Associated Dilated Cardiomyopathy
Disease Ontology Term 🔄 DOID:0110448
Drug Product Name 🔄 ALXN2350
Sponsor Alexion Pharmaceuticals, Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 6 (ESTIMATED)
Results Posted Not Available

Therapy Information


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

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2025-10-17
Completion Date 2032-01-27
Last Update 🔄 2026-06-15

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 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 Note: Other inclusion and exclusion criteria may apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 10
Locations 🔄 United States,Italy,United Kingdom,Spain

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links