A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
NCT ID: NCT07148089
Last Updated: 2025-12-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
18 participants
INTERVENTIONAL
2026-01-05
2031-05-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort 1 (≥ 18 years of age)
Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501.
SGT-501
IV for infusion
Cohort 2 (≥ 18 years of age)
Participants will receive a single IV infusion (dose 2) of SGT-501.
SGT-501
IV for infusion
Cohort 3 (≥ 7 to < 18 years)
Participants will receive a single IV infusion (level at or below dose(s) assessed in adults) of SGT-501.
SGT-501
IV for infusion
Interventions
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SGT-501
IV for infusion
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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).
7 Years
ALL
No
Sponsors
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Solid Biosciences Inc.
INDUSTRY
Responsible Party
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Locations
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Boston Children's Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Cleveland Clinic
Cleveland, Ohio, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
St. Paul's Hospital
Vancouver, British Columbia, Canada
Countries
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Central Contacts
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Facility Contacts
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Kolawole Olarinoye
Role: primary
Brianna Davies
Role: primary
Other Identifiers
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SGT-501-101
Identifier Type: -
Identifier Source: org_study_id