Clinical Trials for Andersen-Tawil Syndrome
There is currently 1 ongoing clinical trial investigating treatment options for Andersen-Tawil syndrome, a rare genetic disorder affecting heart rhythm and muscle function. This trial is testing combinations of medications to control irregular heartbeats in patients with this condition and a related disorder called Multifocal Ectopic Purkinje-related Premature Contractions (MEPPC).
Clinical trial locations
Study of flecainide alone or with beta-blockers or calcium channel blockers and quinidine for treating ventricular arrhythmias in patients with Andersen-Tawil syndrome and MEPPC
This clinical trial is investigating the most effective medication approaches for controlling irregular heartbeats in patients with Andersen-Tawil syndrome and Multifocal Ectopic Purkinje-related Premature Contractions. These are rare conditions that cause abnormal heart rhythms originating from specific areas of the heart’s electrical system.
Main focus and goal: The study aims to identify which medication or combination of medications works best for controlling ventricular arrhythmias in these patient populations. Researchers will compare the effectiveness of flecainide used alone versus flecainide combined with other heart medications. The study will monitor the number of irregular heartbeats over 24-hour periods to determine which treatment approach is most effective for each individual patient.
Investigational drugs: The trial tests several medications including:
- Flecainide – a medication that slows down electrical signals in the heart to maintain normal heart rhythm by blocking sodium channels in heart cells
- Beta-blockers (including bisoprolol, metoprolol, nebivolol, propranolol, and atenolol) – medications that reduce the heart’s workload and help control heart rate by blocking stress hormones
- Calcium channel blockers (such as verapamil) – medications that help relax blood vessels and control irregular heart rhythms by affecting calcium movement in heart cells
- Quinidine – a medication specifically tested in MEPPC patients that stabilizes electrical activity in the heart
Who can participate: To be eligible for this trial, you must:
- Have a confirmed diagnosis of either Andersen-Tawil syndrome or Multifocal Ectopic Purkinje-related Premature Contractions
- Have shown signs of irregular heart rhythms during previous heart monitoring
- Be currently taking flecainide at a stable dose for at least 3 months
- Be at least 18 years old
- For MEPPC patients: have a confirmed genetic test showing a specific variation in the SCN5A gene
Who cannot participate: You cannot join this study if you:
- Are under 18 years old or over 65 years old
- Are pregnant or breastfeeding
- Have known allergies to flecainide, quinidine, beta-blockers, or calcium channel blockers
- Have severe kidney or liver problems
- Have a history of severe heart failure or significant structural heart abnormalities
- Have severe psychiatric conditions or a history of substance abuse in the past 6 months
- Have uncontrolled high blood pressure or significant bleeding disorders
- Have a life expectancy shorter than the study duration
What the study involves: Participants will continue taking their current stable dose of flecainide for at least 3 months while their heart rhythm is monitored. They will then receive either flecainide alone or flecainide combined with beta-blockers or calcium channel blockers. MEPPC patients will also have the opportunity to try quinidine as an alternative treatment. Throughout the study, regular heart rhythm monitoring will track the number of irregular heartbeats occurring in each 24-hour period. All medications are taken as oral tablets, and the study runs from April 2024 to May 2025.
Summary
Currently, there is one clinical trial available for patients with Andersen-Tawil syndrome, taking place in the Netherlands. This trial focuses on optimizing treatment approaches for controlling irregular heart rhythms by testing various medication combinations. The study is particularly notable for including patients with both Andersen-Tawil syndrome and the related condition MEPPC, and for systematically comparing different drug combinations to determine the most effective treatment strategy for each patient. The trial emphasizes a personalized approach to managing these rare cardiac conditions by monitoring individual responses to different medication regimens.
