Study of AGP100 Safety and Effectiveness in Patients with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
- Participants:Adults
Rare diseases
Investigational molecules
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DNS-6288 is an investigational substance being developed as a potential treatment for catecholaminergic polymorphic ventricular tachycardia (CPVT). It is a chemically synthesised substance, meaning it is a small-molecule compound produced through chemical processes rather than derived from biological sources.[1]
The substance is supplied by Agiana Pharmaceuticals AS. It has no marketing authorisation in the product records, which means it is not yet approved as a medicine and remains in the research stage.[1] In the trial records, DNS-6288 appears as the test substance in two trials, indicating it is the active compound under investigation.[1]
The registry data do not describe the mechanism of action of DNS-6288. What is known from the trial record is that the substance is being tested in people with CPVT who continue to have residual ventricular ectopy — extra or abnormal heartbeats originating in the lower chambers of the heart — during exercise testing, despite being on stable doses of current standard of care.[1] This suggests the research is exploring whether DNS-6288 can provide additional benefit when existing treatments are not fully controlling the abnormal heart rhythms.
DNS-6288 is given as a capsule taken by mouth. The trial record lists a 50 milligram oral capsule formulation.[1] The trial is designed to assess increasing doses of the substance, starting from this 50 mg strength.
One authorised Phase 2a trial is listed in the registry for DNS-6288. The trial is interventional, meaning participants receive the study drug as part of the research.[1]
The trial aims to enrol 10 participants. It is currently authorised but not yet recruiting, so no results are available yet.[1]
The primary outcomes focus on safety and tolerability. Safety is measured by the incidence of adverse events from the first dose and changes in standard safety assessments, including physical examinations, vital signs, body weight, 12-lead electrocardiogram (ECG), and safety laboratory tests. Tolerability is assessed as a yes-or-no measure: if a dose adjustment, dose interruption, or discontinuation of treatment is deemed necessary by the investigator, the participant is classified as not tolerating the treatment.[1]
Participants must have genetically confirmed CPVT and residual ventricular ectopy during exercise testing while on stable doses of current standard of care.[1]
No side effect data are available from the sources provided. The trial is specifically designed to measure safety and tolerability, and its results will provide the first information on side effects of DNS-6288 in people with CPVT.[1]
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