What side effects are associated with this type of intervention?

Common treatments for Long QT Syndrome (LQTS) include beta-blockers, potassium supplements, and antiarrhythmic drugs like dofetilide. Beta-blockers can cause fatigue, cold extremities, and bradycardia. Potassium supplements may lead to gastrointestinal discomfort and hyperkalemia. Dofetilide can cause torsades de pointes, a life-threatening arrhythmia, as well as dizziness and headache.

What prior FDA approvals exist?

FDA-approved treatments for Long QT Syndrome primarily include beta-blockers such as propranolol and nadolol, which are commonly used to manage symptoms and reduce the risk of arrhythmias. Additionally, combined alpha- and beta-blocking agents like labetalol have been used in specific cases to suppress arrhythmias associated with LQTS. These treatments aim to mitigate QTc prolongation, similar to the investigational drug LQT-1213 being studied for its effects on dofetilide-induced QTc prolongation.

What other types of research exist?

Promising novel alternatives to LQT-1213 for Long QT Syndrome patients in 2024 include dofetilide, which has been studied for its effects on QTc prolongation, and other emerging therapies such as gene therapy and CRISPR-based approaches that target the underlying genetic mutations causing LQTS. Additionally, advancements in personalized medicine and the use of wearable technology for continuous monitoring and early intervention may also play a significant role in the management of LQTS.

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Potassium Channel Modulator

LQT-1213 for Long QT Syndrome

Phase 1 & 2

Recruiting

Led By Jan Matousek, DO

Research Sponsored by Thryv Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 0,1,1.5, 2, 3, 4, 5, 6, 8, 10. 12 after administration of placebo on day1 and treatment day 2 and day4.

Summary

This trial tests a new medication, LQT-1213, to help with heart rhythm problems. It involves healthy adults and patients with specific heart rhythm disorders (LQT2 or LQT3). The medication aims to keep the heart's electrical signals normal, preventing irregular heartbeats.

Who is the study for?

Healthy adults and patients with Type 2 or 3 Long QT Syndrome (LQT2 or LQT3) can join this trial. Healthy participants must be 19-60 years old, have a BMI of 18-35 kg/m^2, not have used tobacco recently, and agree to use effective contraception. Patients with LQT need an ICD implanted and a specific QTcF interval. People with certain health conditions, recent blood donations, drug abuse history, or those on conflicting medications cannot participate.

What is being tested?

The study tests the effects of LQT-1213 on heart rhythm prolongation caused by Dofetilide in healthy subjects (Part1), followed by its safety in patients with congenital Long QT syndrome types LQT2 or LQT3 (Part2). Part1 is a crossover study where subjects receive both treatments at different times; Part2 involves multiple doses for safety evaluation.

What are the potential side effects?

Potential side effects are not detailed but would typically include reactions related to heart rhythm changes due to the nature of the drugs being tested which affect cardiac electrical activity.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 0,1,1.5, 2, 3, 4, 5, 6, 8, 10. 12 after administration of placebo on day1 and treatment day 2 and day4.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~0,1,1.5, 2, 3, 4, 5, 6, 8, 10. 12 after administration of placebo on day1 and treatment day 2 and day4.

This trial's timeline: 3 weeks for screening, Varies for treatment, and 0,1,1.5, 2, 3, 4, 5, 6, 8, 10. 12 after administration of placebo on day1 and treatment day 2 and day4. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Part 1: Pharmacodynamics: by-time point analysis for QTc on LQT-1213 versus placebo

Part 2: Safety and tolerability of oral LQT-1213 in participants with LQT-2 or LQT-3

Secondary study objectives

Part 1: Pharmacokinetic Dofetilide AUC0-t

Part 1: Pharmacokinetic Dofetilide AUCtau

Part 1: Pharmacokinetic Dofetilide Cmax

+14 more

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 2: TID dosing of PlaceboExperimental Treatment1 Intervention

Placebo matched to LQT-1213 (Day 1)TID

Group II: Part 2: TID dosing of LQT-1213Experimental Treatment1 Intervention

LQT-1213 2.24 mg/kg/day TID (at time 0, 8 and 16 hours) on Days 2-4, with a final single morning dose on Day 4 at time 0, though these time points may be adjusted based upon emerging data.

Group III: Part 1: Arm A: 500 μg of Dofetilide BID in combination with dose-escalating LQT-1213 TID, orallyExperimental Treatment2 Interventions

Dofetilide 500 μg BID (2 × 250 μg capsules), orally (Days 1-8) and LQT-1213 3 times a day (TID) low dose (Days 3 and 4), mid dose (Days 5 and 6), and high dose (not to exceed 0.747 mg/kg TID, daily dose 2.24 mg/kg/day) on Days 7 and 8. The specific doses will be determined before administration of the first dose of LQT-1213, but the high dose will not exceed 0.747 mg/kg TID or 2.24 mg/kg/day. Only 1 dose of dofetilide and LQT-1213 will be administered on Day 8.

Group IV: Part 1: Arm B: 500 μg of Dofetilide BID in combination with placeboPlacebo Group2 Interventions

Dofetilide 500 μg BID (2 × 250 μg capsules), orally (Days 1-8) and placebo matched to LQT-1213 TID (Days 3-8). Only 1 dose of dofetilide and placebo matched to LQT-1213 will be administered on Day 8.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

LQT-1213

2022

Completed Phase 1

~50

Placebo

1995

Completed Phase 3

~2670

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Long QT Syndrome (LQTS) include beta-blockers, potassium supplements, and antiarrhythmic drugs like mexiletine. Beta-blockers work by reducing the heart rate and the force of contraction, which helps to prevent arrhythmias. Potassium supplements help to normalize the potassium levels in the blood, which can reduce the risk of prolonged QT intervals. Mexiletine, a sodium channel blocker, shortens the QT interval by stabilizing the cardiac cell membrane and reducing the duration of the action potential. These mechanisms are crucial for LQTS patients as they help to mitigate the risk of life-threatening arrhythmias by preventing excessive prolongation of the QT interval, similar to the investigational drug LQT-1213, which aims to reduce QTc prolongation.