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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Solid Biosciences Inc.
Study ID
NCT07148089
Phase
PHASE1
Status
Recruiting
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Conditions

  • Catecholaminergic Polymorphic Ventricular Tachycardia

Eligibility Criteria

Sex
ALL
Age
7 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • SGT-501 — DRUG
    IV for infusion

Study Details

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.

Key Dates

First listed
Aug 29, 2025
Start date
Feb 23, 2026
Status verified
Jul 2026
Primary completion
Jun 30, 2027
Completion
May 31, 2031

Study Design

Enrollment
18 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

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.

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

Locations (4)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02459
Elizabeth DeWitt, MD
[email protected]
Mayo ClinicRochesterMinnesota55905
Kolawole Olarinoye
[email protected]
507-293-1564
John Giudicessi, MD (PRINCIPAL_INVESTIGATOR)
Cleveland ClinicClevelandOhio44195
Paige Ewing
[email protected]
216-445-8798
Peter Aziz, MD (PRINCIPAL_INVESTIGATOR)
University of PennsylvaniaPhiladelphiaPennsylvania19104-

Find similar trials in Boston, MA

By research site
Boston Children's Hospital· Boston, MAMayo Clinic· Rochester, MNCleveland Clinic· Cleveland, OHUniversity of Pennsylvania· Philadelphia, PA

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