Catecholaminergic Polymorphic Ventricular TachycardiaPhase 1ARTEMISNCT07148089
A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
A Phase 1 study testing SGT-501 in people with Catecholaminergic Polymorphic Ventricular Tachycardia, run by Solid Biosciences Inc. at 5 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Catecholaminergic Polymorphic Ventricular Tachycardia
- What is being tested?
- SGT-501
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 23 Feb 2026to May 2031
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
5 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 2 countries
United States4 sites, 4 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02459
Elizabeth DeWitt, MD, gene.therapy@childrens.harvard.edu
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
Paige Ewing, 216-445-8798, ewingp@ccf.org
Peter Aziz, MD
- Not yet recruiting
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Sierra Slade, 507-422-5433, Slade.Sierra@mayo.edu
John Giudicessi, MD
Canada1 site, 1 recruiting
- Recruiting
St. Paul's Hospital
Vancouver, British Columbia, BC V6Z 1Y6
Brianna Davies, 604-682-2344 ext. 64252, brianna.davies@phc.ca
Thomas Roston, MD
About this study
Official title: 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
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.
- Being tested
- SGT-501
- Keywords
- Catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-501, Ryanodine Receptor 2 (RYR2), adeno-associated virus serotype 8 (AAV8), Cardiac, Gene Therapy
- Medical subject headings
- Polymorphic Catecholaminergic Ventricular Tachycardia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SGT-501Drug
IV for infusion
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (≥ 18 years of age) | Experimental | Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501. Receives: Drug: SGT-501 |
| Cohort 2 (≥ 18 years of age) | Experimental | Participants will receive a single IV infusion (dose 2) of SGT-501. Receives: 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. Receives: Drug: SGT-501 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360
Measured: First dose through Day 360
Secondary outcomes (1)
Change from baseline in ventricular arrhythmia score (VAS) on exercise stress test (EST) at Day 180
An EST will be used to evaluate ventricular arrhythmia scores. The EST will be performed using the standard Bruce treadmill protocol.
Measured: Baseline, Day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2025
- Study start23 Feb 2026
- Primary completionJun 2027 (estimated)
- Study completionMay 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Catecholaminergic Polymorphic Ventricular TachycardiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Solid Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07148089
- Sponsor's protocol ID
- SGT-501-101
- Acronym
- ARTEMIS
- Status last verified
- Oct 2026
- First submitted
- 22 Aug 2025
- Last updated
- 26 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07148089
- Acronym / sponsor ID
- ARTEMIS / SGT-501-101
- Keywords
- Catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-501, Ryanodine Receptor 2 (RYR2), adeno-associated virus serotype 8 (AAV8), Cardiac, Gene Therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Solid Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 23 Feb 2026 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- May 2031 (estimated)
- First posted
- 29 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (≥ 18 years of age) | Experimental | Drug: SGT-501Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501. |
| Cohort 2 (≥ 18 years of age) | Experimental | Drug: SGT-501Participants will receive a single IV infusion (dose 2) of SGT-501. |
| Cohort 3 (≥ 7 to < 18 years) | Experimental | Drug: SGT-501Participants will receive a single IV infusion (level at or below dose(s) assessed in adults) of SGT-501. |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SGT-501IV for infusion | Drug | Cohort 1 (≥ 18 years of age), Cohort 2 (≥ 18 years of age), Cohort 3 (≥ 7 to < 18 years) |
Primary outcome
Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360
Time frame: First dose through Day 360
1 secondary outcome
Change from baseline in ventricular arrhythmia score (VAS) on exercise stress test (EST) at Day 180
Time frame: Baseline, Day 180
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SGT-501 in people with Catecholaminergic Polymorphic Ventricular Tachycardia, run by Solid Biosciences Inc. at 5 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Solid Bio Clinical Trials (617-337-4680, clinicaltrials@solidbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Treatment-Emergent Adverse Event (TEAEs)s through Day 360 (time frame: First dose through Day 360). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.