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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
Indication DMD-Associated Dilated Cardiomyopathy
Compound Name AAV1-CMV-SERCA2a (SRD-001)
Sponsor Sardocor Corp.
Funder Type Industry
Status
Not yet recruiting
Enrollment Count 12

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
Dose 1 1 x 10^13 vg
Dose 2 3 x 10^13 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2024-01-11
Completion Date 2030-02
Last Update 2024-01-25

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex MALE

Locations


No.of Trial Sites
Locations

Regulatory Information


Has US IND True
Recent Updates Granted Orphan Drug Designation by FDA 2/28/24

Resources/Links