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Gene Therapy Trial Browser
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
This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.
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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
News and Press Releases
Medera's Sardocor Granted FDA Orphan Drug Designation for DMD-associated cardiomyopathy Gene Therapy - February 2024
Preclinical Publications
Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy