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

Start date
Feb 23, 2026
Status verified
Jun 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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