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

NCT07148089 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: SGT-501-101
• Study Type: interventional
• Target: 18
• Locations: 2 countries
• Sites: 5

Brief Summary

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.

Conditions

Catecholaminergic Polymorphic Ventricular Tachycardia

Keywords

Catecholaminergic polymorphic ventricular tachycardia (CPVT); SGT-501; Ryanodine Receptor 2 (RYR2); adeno-associated virus serotype 8 (AAV8); Cardiac; Gene Therapy

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360

  First dose through Day 360

Secondary Outcomes (1)

• Change from baseline in ventricular arrhythmia score (VAS) on exercise stress test (EST) at Day 180

  Baseline, Day 180

Study Arms (3)

Cohort 1 (≥ 18 years of age)

EXPERIMENTAL

Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501.

Drug: SGT-501

Cohort 2 (≥ 18 years of age)

EXPERIMENTAL

Participants will receive a single IV infusion (dose 2) of SGT-501.

Drug: SGT-501

Cohort 3 (≥ 7 to < 18 years)

EXPERIMENTAL

Participants will receive a single IV infusion (level at or below dose(s) assessed in adults) of SGT-501.

Drug: SGT-501

Interventions

SGT-501 DRUG

IV for infusion

Cohort 1 (≥ 18 years of age); Cohort 2 (≥ 18 years of age); Cohort 3 (≥ 7 to < 18 years)

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify 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 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.

You may not qualify 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 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).

Sponsors & Collaborators

• Solid Biosciences Inc.: lead

Study Sites (5)

Boston Children's Hospital

Boston, Massachusetts, 02459, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

NOT YET RECRUITING

St. Paul's Hospital

Vancouver, British Columbia, BC V6Z 1Y6, Canada

RECRUITING

MeSH Terms

Conditions

Polymorphic Catecholaminergic Ventricular Tachycardia

Condition Hierarchy (Ancestors)

Tachycardia, Ventricular; Tachycardia; Arrhythmias, Cardiac; Heart Diseases; Cardiovascular Diseases; Cardiac Conduction System Disease; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Solid Bio Clinical Trials

CONTACT

617-337-4680; clinicaltrials@solidbio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2025
• First Posted: August 29, 2025
• Study Start: February 23, 2026
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): May 1, 2031
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (4); CA (1)