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Clinical Trial Report

Gene Therapy Trial Report

Summary

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy


NCTID NCT06224660 (View at clinicaltrials.gov)
Description
Development Status Active
Indication DMD-Associated Dilated Cardiomyopathy
Disease Ontology Term DOID:0110461
Compound Name SRD-001
Compound Description AAV1-CMV-SERCA2a
Sponsor Sardocor Corp.
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 12
Results Posted Not Available

Therapy Information


Target Gene/Variant SERCA2a
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intraarterial
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV1
Editor Type none
Dose 1 1E13 vg
Dose 2 3E13 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2024-01-11
Completion Date 2030-10
Last Update 2025-02-27

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Anticipated Phase 2b in 2H2025

Resources/Links