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

Conditions

• Long QT Syndrome (LQTS) 2

Eligibility Criteria

Sex

ALL

Age

15 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• baseline lead-in — OTHER
  Participants will undergo a 3 weeks run-in baseline
• THRV-1268 — DRUG
  THRV-1268 50 mg bid (total daily 100 mg) for 6 weeks
• THRV-1268 — DRUG
  THRV-1268 100 mg bid (total daily 200 mg) for 6 weeks

Study Details

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.

Key Dates

Start date

Mar 31, 2026

Status verified

Mar 2026

Primary completion

Jun 30, 2027

Completion

Jun 30, 2027

Study Design

Enrollment

64 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: THRV-1268
  3 weeks lead-in baseline followed by 50 mg BID for 6 weeks with scheduled up-titration to 100 mg BID for another 6 weeks (12 weeks total treatment)

Primary Outcome Measure

Part A: To evaluate the effect of oral THRV-1268 on QTcF interval by AUC method in participants with LQTS 2 [ Time Frame: Day 42 and Day 84 ]

Central Contacts

• Study Director
  1-855-958-WAVE
  [email protected]

Locations (6)

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