Treatment of an Inherited Ventricular Arrhythmia (NCT05122975) | Clinical Trial Compass
TerminatedPhase 2
Treatment of an Inherited Ventricular Arrhythmia
Stopped: Due to challenges in recruiting patients.
United States, Netherlands8 participantsStarted 2023-08-01
Plain-language summary
The goal of the proposed project is to determine the safety and tolerability as well as the preliminary efficacy of a novel small molecule drug, S48168 (ARM210), for the treatment of Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT1). This disease is associated with fatal changes in heart rhythms leading to sudden death with exercise or excessive excitement. It is due to mutations in the Ryanodine Receptor calcium release channel, which cause leaky channels leading to the disease. S48168 (ARM210) repairs these leaky channels and can be a disease-modifying therapy restoring normal function to the channels. This result would allow patients with CPVT to live normal, active lives. Funding Source- FDA OOPD.
Who can participate
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Participant must be 18 to 65 years of age inclusive, at the time of signing the informed consent.
. Participants who are willing and able to comply with scheduled visits, study drug administration plan, study restrictions, and study procedures.
. Participants have a confirmed genetic diagnosis of CPVT1 and supporting clinical phenotype, including residual ventricular ectopy (a complexity score ≥ 2; requiring at minimum the presence of PVCs in bigeminy on exercise stress test) on a stable (at least 1 month) standard-of-care, CPVT1-directed treatment regimen as decided by their CPVT treating physician.
. Have a body mass index (BMI) ≤ 36 kg/m2 (inclusive) at screening.
. Male participants agree to not donate sperm from the first day of dosing of study drug until 5 half-lives plus 90 days (approximately 94 days) after the last dose of study drug.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The effect of S48168 (ARM210) treatment on the amount and complexity of exercise-• Change in ectopy score from baseline to Day 28 versus placebo (pre-dose Period 1 baseline to Day 28 Period 1 versus Day 28 Period 2
. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
. Daily use of medicines and dietary supplements need to be approved by the PI and Sponsor, or a drug/supplement-dependent wash-out prior to inclusion.
Exclusion criteria
. History or presence of alcoholism or drug abuse within the past 2 years prior to the first dose of study drug.
. History or presence of hypersensitivity or idiosyncratic reaction to the study drug, related compounds, or inactive ingredients.
. ALT or AST levels three times above the upper limits of normal (ULN) at screening (isolated elevations of total bilirubin \< 2 X ULN with direct bilirubin below the ULN will be included). A recheck for confirmation is allowed.
. History of documented, EEG-confirmed epileptic seizures.
. History of cancer (malignancy). Exceptions: (1) Subjects with adequately treated non-melanomatous carcinoma or carcinoma in situ of the cervix may participate in the trial; (2) Subjects with other malignancies who have been successfully treated \> 10 years prior to the screening where in the judgment of the investigator has revealed no evidence of recurrence from the time of treatment through the time of the screening except those identified at the beginning of the exclusion criterion; or (3) Subjects who in the opinion of the investigator are highly unlikely to sustain a recurrence for the duration of the trial.
. Currently has uncontrolled diabetes defined as HbA1c \> 7% at screening visit or diabetic neuropathy.
. Estimated creatinine clearance \< 40mL/minute at screening visit.
. Clinically significant abnormality on their screening and/or prior to first dosing resting ECG, other than hypertensive related, or heart failure (ejection fraction \< 30%) or other clinically significant structural heart disease.