Actively Recruiting
Evaluation of Efficacy and Safety of THRV-1268 in Long QT Syndrome Type 2 (LQTS 2)
Led by Thryv Therapeutics, Inc. · Updated on 2026-03-30
64
Participants Needed
6
Research Sites
65 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluation of Efficacy and Safety of THRV-1268 in Long QT Syndrome Type 2 (LQTS 2)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female participants over 15 years of age at Screening (first 5 participants in Part A must be 18 or older)
- Confirmed LQTS 2 genotype with a pathologic or likely pathologic KCNH2 mutation
- QTcF interval greater than 480 ms and less than or equal to 600 ms on Screening ECG
- Body weight at least 45 kg and body mass index between 18.0 and 40.0 kg/m2 at Screening
- Male and female participants of childbearing potential agree to use highly effective contraception throughout the study
- Capable of giving signed informed consent and complying with study requirements
- Participants aged 15 to under 18 years capable of providing signed assent
You will not qualify if you...
- History within 2 months prior to Screening of aborted cardiac arrest, ICD implantation, or syncopal episode due to ventricular arrhythmia
- History of structural or functional cardiovascular disease, myocardial infarction, stroke or transient ischemic attack, atrial fibrillation or flutter, heart failure, bundle branch block, angina pectoris, or significant ventricular tachycardia not due to TdP
- Active or known liver disease
- Active or past cancer except non-melanoma skin cancer
- Advanced pulmonary disease requiring more than a steroid inhaler
- Pulmonary artery hypertension
- Pregnant, lactating, breastfeeding, or planning pregnancy
- Positive pregnancy test at Screening or Day -7 Visit
- Clinically significant abnormal findings on physical exam or medical history at Screening or Day -21
- Recent acute illness resolved less than 14 days before first study drug dose or major illness/hospitalization within 1 month prior
- Recent history of alcohol or substance abuse posing safety or compliance risk
- Active pacemaker or ICD for ventricular pacing
- Current or planned use of restricted drugs
- Scheduled elective surgery during study
- Participation in another interventional clinical study within 1 month
- Abnormal blood pressure readings at Screening (diastolic >95 mm Hg; systolic <90 or >150 mm Hg)
- ECG abnormalities at Screening such as PR >280 ms; QRS >110 ms; QTcF >600 ms or <480 ms; bundle branch block; significant ST-T wave changes; resting heart rate <50 bpm (or <40 bpm if on beta-blocker) or >100 bpm
- Atrial pacing rate set above 80 bpm
- Abnormal renal function with eGFR <70 mL/min/1.73 m2
- Abnormal liver function tests exceeding specified limits
- Clinically significant abnormal serum chemistry values for hemoglobin, potassium, magnesium, or calcium
- Any condition that would interfere with safety, tolerability, or efficacy evaluation or study compliance
- Consumption of more than 21 alcoholic drinks per week for men or 14 for women over the past 2 years
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 6 locations
1
Honor Health Research and Innovation Institute
Scottsdale, Arizona, United States, 85258
Actively Recruiting
2
University of California San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
3
University of Illinois Chicago
Chicago, Illinois, United States, 60612
Actively Recruiting
4
Heart Center Clinical Research Program | MGH
Boston, Massachusetts, United States, 02114
Actively Recruiting
5
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
6
Wilmington Health
Wilmington, North Carolina, United States, 28401
Actively Recruiting
Research Team
S
Study Director
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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