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New treatment option for Catecholaminergic Polymorphic Ventricular Tachycardia
Official title A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
ClinicalTrials.gov ID: NCT07148089
What this study is testing
What is SGT-501?
SGT-501 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for catecholaminergic polymorphic ventricular tachycardia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in participants with catecholaminergic polymorphic ventricular tachycardia (CPVT). The first-in-human (FIH) safety study will focus on obtaining safety data in adult participants.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 and older
You may be able to join if
- 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...
- 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...
- 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...
You likely can't join if
- 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...
- Clinically significant abnormalities of coagulation including international normalized ratio or activated partial thromboplastin time \> 1.2 × ULN or...
- Potential concomitant cardiomyopathy or inherited arrhythmia as evidenced by pathogenic or likely pathogenic mutation other than RYR2 obtained on...
- 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.
See the full eligibility 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...
- 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...
- Must be up to date with meningococcal vaccination per national guidelines or willing to receive meningococcal vaccine to achieve this.
- Other to be applied as per protocol.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
- Rochester, Minnesota, United States
- Cleveland, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Vancouver, British Columbia, Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 7 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States; Rochester, Minnesota, United States; Cleveland, Ohio, United States; Philadelphia, Pennsylvania, United States; Vancouver, British Columbia, Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.