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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
Enrollment Count 12 (ESTIMATED)
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
Delivery System Viral transduction
Vector Type AAV1
Editor Type none
Dose 1 1E13 vg
Dose 2 3E13 vg

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
Eligibility Criteria
Inclusion Criteria: * Diagnosis of DMD with confirmatory genetic testing * Cardiomyopathy with left ventricular scar in at least 3 of 16 segments * Left ventricular ejection fraction \< 40% * Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment * Willing and able to provide informed consent Exclusion Criteria: * Abnormal blood pressure * Non-DMD-related liver function test elevations * Cystatin C ≥ 1.2 mg/L * Thrombocytopenia * Anemia * Inadequate pulmonary function
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

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