A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age7+
SponsorSolid Biosciences Inc.
About this trial
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. Cohort 1 and Cohort 2 (optional for dose exploration) will include participants ≥ 18 years of age. Cohort 3 will include participants ≥ 7 to \< 18 years of age and will be initiated following data and safety monitoring board (DSMB) recommendations. Participants will be monitored for 5 years post-administration of SGT-501 including the active treatment period (1 year) and long-term follow-up (LTFU) (4 years) period.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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 (m²) 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.
Trial design
Design model
Parallel
Treatments tested in this trial
SGT-501
DrugIV for infusion
Treatment groups
18 Participants
are divided into 3 treatment groupsGroup A: Cohort 1 (≥ 18 years of age)Experimental treatment 1 intervention
Group B: Cohort 2 (≥ 18 years of age)Experimental treatment 1 intervention
Group C: Cohort 3 (≥ 7 to < 18 years)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360
Time frame
First dose through Day 360Secondary outcomes
1
Change from baseline in ventricular arrhythmia score (VAS) on exercise stress test (EST) at Day 180
An EST will be used to evaluate ventricular arrhythmia scores. The EST will be performed using the standard Bruce treadmill protocol.
Time frame
Baseline, Day 180Sponsors and contacts
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Source ClinicalTrials.gov