Recruiting
Phase 1

SGT-501

Sponsor:

Solid Biosciences Inc.

Code:

NCT07148089

Conditions

Catecholaminergic Polymorphic Ventricular Tachycardia

Eligibility Criteria

Sex: All

Age: 7+

Healthy Volunteers: Not accepted

Interventions

SGT-501

Study Details

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

Study ID

NCT07148089

Start date

Feb 23, 2026

Status verified date

Aug, 2026

Completion date

May, 2031

Anticipated

Primary completion date

Jun, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 7+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

18 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1 (≥ 18 years of age)

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

experimental: Cohort 2 (≥ 18 years of age)

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

experimental: 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.

Interventions

SGT-501

IV for infusion

Primary outcome measure

  • Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360 [ Time Frame: First dose through Day 360 ]

Central Contacts and Locations

Central contacts

Solid Bio Clinical Trials

617-337-4680clinicaltrials@solidbio.com

Locations

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02459

Contacts

Elizabeth DeWitt, MD

gene.therapy@childrens.harvard.edu

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Kolawole Olarinoye

507-293-1564Olarinoye.Kolawole@mayo.edu

Principal Investigator:

John Giudicessi, MD

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Contacts

Paige Ewing

216-445-8798ewingp@ccf.org

Principal Investigator:

Peter Aziz, MD

St. Paul's Hospital

Recruiting

Vancouver, British Columbia, Canada, BC V6Z 1Y6

Contacts

Brianna Davies

604-682-2344brianna.davies@phc.ca

Principal Investigator:

Thomas Roston, MD

More Information

Sponsor

Solid Biosciences Inc.

Last update posted

Aug 10, 2026

Last verified

Aug, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Solid Biosciences Inc. on 2026-08-10.