Optimal drug therapy for the suppression of ventricular arrhythmias in Andersen-Tawil syndrome and multifocal ectopic Purkinje-related premature contractions: a series of N-of-1 trials

Phase 1

Conditions: Andersen-Tawil syndrome, Multifocal Ectopic Purkinje-related Premature Contractions
Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

Registration Number: CTIS2024-510682-42-01

Lead Sponsor: Amsterdam UMC

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 10

Inclusion Criteria

One of the following two primary diagnostic criteria A.Clinical diagnosis of ATS. Genetically confirmed diagnosis (i.e. class 4 or 5 KCNJ2 variant) is not required B.Clinical diagnosis of MEPPC and carrier of associated class 4 or 5 SCN5A variant, Has demonstrated a disease phenotype of ATS or MEPPC including ventricular arrhythmia burden at any point during follow-up on Holter monitor or other rhythm monitoring device (i.e. loop recorder, ECG patch), Is currently treated with a stable (at least 3 months) dose of flecainide, Age = 18 years

Exclusion Criteria

Pregnancy, Inability to take orally administered tablets, Inability to provide informed consent, Contra-indication to study medication, Significant structural heart disease (left ventricular ejection fraction <50%, history or signs of coronary ischemia, suspicion or definitive diagnosis of cardiomyopathy, or moderate/severe valve regurgitation), Suspicion or definitive diagnosis of another (heritable) arrhythmia syndrome, e.g. Brugada syndrome, early repolarization syndrome or catecholaminergic polymorphic ventricular tachycardia, Presence of a short (<350 ms) or prolonged (>480 ms) heart-rate corrected QT interval on the resting ECG at baseline, History of therapy refractory ventricular arrhythmia or intolerable side-effects on an adequate dose of any study medication, as determined by the treating cardiologist, Serious known comorbid disease with a life expectancy of less than two years, Ongoing medical condition that is deemed by the principal investigator to interfere with the conduct or assessments of the study or safety of the subjects, Circumstances that prevent follow-up