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Résztvevőt keres itt: Kanada1. fázis

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

A legfontosabb adatok

Ki végzi
Solid Biosciences Inc.
Hol
Vancouver (Kanada)A helyszín megnézéseMég 1 másik országban, 4 helyszínen
Azonosító
Állapot
Toboroz
Fázis
1. fázis
Résztvevők
18 fő, tervezett
Kezdés
2026. febr. 23.
Várható befejezés
2031. máj.
A vizsgálat típusa
Beavatkozással járó, 3 csoport
A vizsgált kezelések
  • SGT-501
A regiszteradat frissítve
2026. október 26.

Ki vehet részt

MI-összefoglaló

A vizsgálat saját részvételi feltételeiből. Gép olvasta ki az oldalon álló regiszterszövegből, ezért tévedhet. Hogy ki vehet részt, azt a lenti regiszterszöveg és a vizsgálat csapata dönti el.

The trial is looking for people aged 7 or older with CPVT, an inherited heart rhythm disorder, caused by a RYR2 gene change. They get a single IV dose of the gene therapy SGT-501.

Életkor
7 years and older. Cohorts 1 and 2: 18 years and older. Cohort 3: 7 to under 18 years.
Nem
All sexes can join.
A betegség stádiuma
CPVT shown by fast, irregular heartbeats from the lower heart chambers during exercise or on an exercise stress test (EST). People must have had a life-threatening rhythm event, such as surviving sudden cardiac arrest, an ICD shock, fainting from the heart rhythm, or ventricular tachycardia lasting 30 seconds or more. A past EST must show a ventricular arrhythmia score (VAS) of ≥ 2.
Korábbi kezelés
Must be on a stable dose of a beta-blocker and/or flecainide (dose not changed by more than 50% for at least 1 month before screening). Must not have had any gene transfer drug before. The first 2 people in each cohort must have a working implanted defibrillator (ICD). Must be up to date with meningococcal vaccination or willing to get it.
Biomarker vagy leletérték
A RYR2 gene variant that is pathogenic or likely pathogenic for CPVT, confirmed by the central screening lab.

A részvétel fő kizáró okai

  • Abnormal liver function (GGT > 1.5 × ULN or total bilirubin > ULN), or kidney function (eGFR < 60 mL/min/1.73 m^2)
  • Blood clotting problems (INR or aPTT > 1.2 × ULN, or platelets < 150,000 cells/mm^3)
  • A disease-causing gene change other than RYR2 pointing to another heart muscle or rhythm disease
  • Another investigational drug within 90 days or 5 half-lives before screening, whichever is longer
  • Body mass index ≥ 30 kg/m^2, or cannot or will not take the required immune-suppressing medicines

A regiszter feltételei, szó szerint

Mind a 18 feltétel megnézéseKevesebb

Ki vehet részt

  • 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

Ki nem vehet részt

  • 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
  • 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
  • Abnormal liver function: gamma-glutamyl transferase (GGT) > 1.5 × upper limit of normal [ULN] or total bilirubin > ULN)
  • 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)
  • 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

Helyszínek itt: Kanada

Még 1 másik országban, 4 helyszínen

  • St. Paul's HospitalVancouver, KanadaContact: Brianna DaviesRésztvevőt keres

A vizsgálatról

Mit akar kideríteni a vizsgálat

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.

A vizsgálat 3 csoportja

  • Cohort 1 (≥ 18 years of age)A vizsgált kezelés

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

    Kezelés: SGT-501

  • Cohort 2 (≥ 18 years of age)A vizsgált kezelés

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

    Kezelés: SGT-501

  • Cohort 3 (≥ 7 to < 18 years)A vizsgált kezelés

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

    Kezelés: SGT-501

Kérdések a vizsgálat csapatának

  1. Milyen gyakran kell bejárnom, és egy-egy alkalom mennyi ideig tart?
  2. Megtérítik az utazási költséget?
  3. Megtarthatom a saját orvosomat?
  4. Mi történik, ha ki akarok lépni?

Contact the study team: see the registry record → Contacts and Locations

Oldal nyomtatása az orvosomnak

Adatok és források

Regiszteradat
Szerepel a ClinicalTrials.gov-on.
Hivatalos cím
A Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in Patients With Catecholaminergic Polymorphic Ventricular Tachycardia
MI-összefoglaló
A vizsgálat saját részvételi feltételeiből. Gépi kiolvasás: 2026. november 14.
A regiszteradat frissítve
2026. október 26.