Status:
NOT_YET_RECRUITING
N-of-1 in ATS and MEPPC
Lead Sponsor:
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Conditions:
Andersen Tawil Syndrome
Multifocal Ectopic Purkinje-related Premature Contractions
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
Rationale: Andersen-Tawil syndrome (ATS) is a very rare heritable cardiac arrhythmia syndrome that is characterized by the triad of periodic paralysis, physical dysmorphisms, and ventricular arrhythmi...
Eligibility Criteria
Inclusion
- 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 flecainide
- Age ≥ 18 years
Exclusion
- Pregnancy
- Contra-indication to study medication (see section 7.4)
- 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
- Inability to take orally administered tablets
- Inability to provide informed consent
Key Trial Info
Start Date :
March 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2025
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT06205550
Start Date
March 1 2025
End Date
October 1 2025
Last Update
January 29 2025
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