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Gene Therapy Trial Report

Summary

A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)


NCTID NCT07148089 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication Catecholaminergic Polymorphic Ventricular Tachycardia
Disease Ontology Term 🔄 DOID:0060675
Drug Product Name 🔄 SGT-501
Drug Product Description 🔄 AAV8-DES-hCASQ2
Sponsor Solid Biosciences Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 18 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 CASQ2
Therapeutic Modality 🔄 Gene transfer
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Overexpression of protective allele/gene
Route of Administration 🔄 Intravenous
Drug Product Type 🔄 Viral vector
Target Tissue/Cell 🔄 Cardiomyocytes
Gene Delivery System Type 🔄 Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV8
Dose 1 🔄 Undisclosed dose escalation, 2 levels

Study Record Dates


Current Phase Phase1
Submit Date 2025-08-22
Completion Date 2031-05
Last Update 🔄 2026-08-10

Participation Criteria


Eligible Age >=7 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: Type of Participant and Disease Characteristics: * Clinical diagnosis of CPVT, based on documented history of polymorphic or bidirectional non-sustained ventricular tachycardia with exercise or ventricular ectopy in a pattern consistent with CPVT on EST. * Central Screening laboratory determination of a RYR2 variant that is pathogenic or likely pathogenic for CPVT. * Documented history of life-threatening ventricular arrhythmic event defined as: survived sudden cardiac arrest, sudden cardiac arrest with appropriate implantable cardioverter defibrillator (ICD) shock, arrhythmic syncope, or sustained ventricular tachycardia (30 seconds or more) with or without ICD shock. * On stable dose (defined as no change in dose by more than 50% for at least 1 month prior to Screening) of standard-of-care therapy defined as a beta-blocker and/or flecainide. * Documented prior history of EST demonstrating a ventricular arrythmia score (VAS) score of ≥ 2. * For the first 2 participants in each cohort only: a properly functioning ICD device in place. Following review of data from Cohorts 1 and 2, the Data Safety and Monitoring Board (DSMB) will determine if this criterion is required for participants in Cohort 3. * Must be up to date with meningococcal vaccination per national guidelines or willing to receive meningococcal vaccine to achieve this. * Other inclusion criteria to be applied as per protocol. Exclusion Criteria: * Abnormal liver function: gamma-glutamyl transferase (GGT) \> 1.5 × upper limit of normal \[ULN\] or total bilirubin \> ULN). * Abnormal renal function defined by estimated glomerular filtration rate \< 60 milliliter /minute (mL/min)/1.73-square meter (m\^2) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Formula. * Clinically significant abnormalities of coagulation including international normalized ratio or activated partial thromboplastin time \> 1.2 × ULN or platelets \< 150,000 cells/cubic millimeter (mm\^3). * Potential concomitant cardiomyopathy or inherited arrhythmia as evidenced by pathogenic or likely pathogenic mutation other than RYR2 obtained on cardiac panel during Screening. * Current or prior treatment with an approved or investigational gene transfer drug. * Exposure to another investigational drug within 90 days prior to Screening or 5 half-lives since last administration, whichever is longer. * Contraindication or unwillingness to receive required immunosuppression regimen. * Body mass index ≥ 30 kilograms per square meter (kg/m\^2). * Other exclusion criteria to be applied as per protocol.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 5
Locations Canada,United States

Regulatory Information


Has US IND True
FDA Designations 🔄 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Company anticipates dosing the first participant in the Phase 1b ARTEMIS clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT) in H2 2026

Resources/Links