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 range18 Years – 65 Years

SexALL

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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.
✓. 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

What they're measuring

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

Timeframe: 28 days

Trial details

NCT IDNCT05122975

SponsorRyCarma Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-04-01

View on ClinicalTrials.gov More Catecholaminergic Polymorphic Ventricular Tachycardia Type 1 trials