RecruitingPhase 2/3

Evaluation of Efficacy and Safety of THRV-1268 in Long QT Syndrome Type 2 (LQTS 2)

United States64 participantsStarted 2026-03-07

Plain-language summary

The goal of this clinical trial is to learn whether the study drug THRV-1268 can safely and effectively shorten the QT interval in people diagnosed with Long QT Syndrome Type 2 (LQTS 2). The study will also learn about the safety and tolerability of THRV-1268 at different doses. The main questions this study aims to answer are: Does THRV-1268 reduce the QTc interval (a measure of the heart's electrical recovery time)? What side effects or medical problems occur when participants take THRV-1268? Which dose of THRV-1268 works best and is safest? Participants will: Complete a 3-week observation period with ECG and Holter monitoring to establish baseline QTc measurements Take THRV-1268 tablets twice daily at two dose levels for 6 weeks (Part A) or be randomly assigned to a dose group for 6 weeks (Part B) Have clinic visits and tests to monitor safety and changes in their heart rhythm May continue taking THRV-1268 for up to 1 year for ongoing safety and efficacy evaluation Researchers will compare changes in QTc over time and evaluate side effects to determine whether THRV-1268 can help reduce the risk of abnormal heart rhythms and sudden cardiac events in people with LQTS 2.

Who can participate

Age range

15 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male and female participants over 15 years of age at Screening (at least the first 5 participants enrolled in Part A must be 18 years of age or older). * LQTS 2 genotype: Demonstration of KCNH2 mutation by clinical laboratory test result that is autosomal dominant (heterozygous) and considered to be pathologic or likely pathologic can be included after approval from the sponsor medical monitor or qualified delegate. * QTcF interval \>480 ms and ≤600 ms based on Screening ECG. * Body weight of at least 45 kg with body mass index between 18.0 and 40.0 kg/m2, inclusively at Screening. * Male and female participants of childbearing potential must agree to use highly effective contraception throughout the duration of the study. * Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. * Participants age 15 to \<18 years capable of providing signed assent. Exclusion Criteria: * Within 2 months prior to Screening, Participant has a history of an aborted cardiac arrest, ICD implantation, or syncopal episode due to a ventricular arrhythmia. Participants can be enrolled after the 2-month period has lapsed. * History of structural or functional cardiovascular disease, myocardial infarction or stroke or transient ischemic attack (TIA), atrial fibrillation or atrial flutter, heart failure, bundle branch block, angina pectoris, or hemodynamically signi…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part A: To evaluate the effect of oral THRV-1268 on QTcF interval by AUC method in participants with LQTS 2

Timeframe: Day 42 and Day 84

Part A: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])

Timeframe: up to 52 weeks

Trial details

NCT IDNCT07277582

SponsorThryv Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-06-30

Contact for this trial

Study Director

1-855-958-WAVE (9283) waveiistudyteam@thryvtrx.com

View on ClinicalTrials.gov More Long QT Syndrome (LQTS) 2 trials