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Disease

Long QT Syndrome

Late-stage therapeutic developmentEmerging research
MeSHLong QT Syndrome
3
Publications
10
Clinical trials
2023
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Risk stratification of sudden cardiac death: a review.

Research2023-08-01Europace : European pacing, arrhythmias, and cardiac electrophysiology : journal of the working groups on cardiac pacing, arrhythmias, and cardiac cellular electrophysiology of the European Society of Cardiology

Risk of QT Interval Prolongation Associated With Use of Hydroxychloroquine With or Without Concomitant Azithromycin Among Hospitalized Patients Testing Positive for Coronavirus Disease 2019 (COVID-19).

Research2020-09-01JAMA cardiology

An International, Multicentered, Evidence-Based Reappraisal of Genes Reported to Cause Congenital Long QT Syndrome.

Research2020-01-27Circulation

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial expected to report results, the earliest in Q2 2027.
Upcoming Milestones
Clinical Milestones1View
Results expected 1
Activity timeline1

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Idursulfaseapproved

Approval — Elaprase is indicated for the long-term treatment of patients with Hunter syndrome (mucop… (2007)

Galsulfaseapproved

Approval — Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confir… (2006)

Growth Hormoneapproved

Approval — Long-term treatment of children with growth failure due to inadequate endogenous growth h… (2001)

Clinical trials

5 sponsors · 1 new · 0 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
10
All trials
1
Active
4
Late-stage
6
Completed
Recently completed

Novel Approaches for Minimizing Drug-Induced QT Interval Lengthening

Phase 4 · Completed · Indiana University

Mutation-specific Therapy for the Long QT Syndrome

Phase 2 · Completed · Istituto Auxologico Italiano

Novel Approaches for Minimizing Drug-Induced QT Interval Lengthening: Reducing the Risk of Drug-Induced QT Interval Lengthening in Women

Phase 4 · Completed · Indiana University

A Phase 1, Randomized, Double-blinded, Placebo-controlled, Single and Multiple Ascending Dose Study to Evaluated the Safety, Tolerability, Pharmacokinetics, Food Effect, and Pharmacodynamics of LQT-1213 in Healthy Adult Participants

Phase 1 · Completed · Thryv Therapeutics, Inc.

Efficacy of Ranolazine in LQT3 Patients

Phase 2 · Completed · University of Rochester

A Phase 1, Open-Label, Parallel-Group, Adaptive, Single Dose Study to Evaluate the Pharmacokinetics of GS-6615 in Subjects With Normal and Impaired Hepatic Function

Phase 1 · Completed · Gilead Sciences

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2007emaApproval — Idursulfase· Elaprase is indicated for the long-term treatment of patients with Hunter syndrome (mucopolysaccharidosis II, MPS II). Heterozygous females were not studied in the clinical trials. source ↗
2006emaApproval — Galsulfase· Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis VI (MPS VI; N-acetylgalactosamine-4-sulfatase deficiency; Maroteaux-Lamy syndrome) (see section 5.1). As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease. A key issue is to treat young patients aged <5 years suffering from a severe form of the disease, even though patients <5 years were not included in the pivotal phase-3 study. source ↗
2001emaApproval — Growth Hormone· Long-term treatment of children with growth failure due to inadequate endogenous growth hormone secretion. Long-term treatment of growth failure associated with Turner syndrome. Treatment of prepubertal children with growth failure associated with chronic renal insufficiency up to the time of renal transplantation. Replacement of endogenous growth hormone in adults with growth hormone deficiency of either childhood or adult-onset etiology. Growth hormone deficiency should be confirmed appropriately prior to treatment. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

3 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20202023
Most influential

Risk of QT Interval Prolongation Associated With Use of Hydroxychloroquine With or Without Concomitant Azithromycin Among Hospitalized Patients Testing Positive for Coronavirus Disease 2019 (COVID-19).

JAMA cardiology · 2020 · 465 cites

An International, Multicentered, Evidence-Based Reappraisal of Genes Reported to Cause Congenital Long QT Syndrome.

Circulation · 2020 · 295 cites

Risk stratification of sudden cardiac death: a review.

Europace : European pacing, arrhythmias, and cardiac electrophysiology : journal of the working groups on cardiac pacing, arrhythmias, and cardiac cellular electrophysiology of the European Society of Cardiology · 2023 · 46 cites
Recent publications

Risk stratification of sudden cardiac death: a review.

Europace : European pacing, arrhythmias, and cardiac electrophysiology : journal of the working groups on cardiac pacing, arrhythmias, and cardiac cellular electrophysiology of the European Society of Cardiology · 2023 · 46 cites

Risk of QT Interval Prolongation Associated With Use of Hydroxychloroquine With or Without Concomitant Azithromycin Among Hospitalized Patients Testing Positive for Coronavirus Disease 2019 (COVID-19).

JAMA cardiology · 2020 · 465 cites

An International, Multicentered, Evidence-Based Reappraisal of Genes Reported to Cause Congenital Long QT Syndrome.

Circulation · 2020 · 295 cites
Major themes5
  • Betacoronavirus1
  • Defibrillators, Implantable1
  • Genetic Predisposition to Disease1
  • Heart Diseases1
  • Long QT Syndrome1
Leading journals3
  • Circulation1
  • Europace : European pacing, arrhythmias, and cardiac electrophysiology : journal of the working groups on cardiac pacing, arrhythmias, and cardiac cellular electrophysiology of the European Society of Cardiology1
  • JAMA cardiology1
Leading researchers8
  • Wilde AAM2
  • Abiusi E1
  • Ackerman MJ1
  • Adler A1
  • Albert C1
  • Amenta S1
  • Amin AS1
  • Basso C1
Affiliations (unnormalised)6
  • Amsterdam UMC location University of Amsterdam1
  • Beth Israel Deaconess Medical Center1
  • Centre d'Investigation Clinique 14021
  • Centre for Cardiovascular Innovation1
  • Columbia University Irving Medical Center1
  • Copenhagen University1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A condition that is characterized by episodes of fainting (SYNCOPE) and varying degree of ventricular arrhythmia as indicated by the prolonged QT interval. The inherited forms are caused by mutation of genes encoding cardiac ion channel proteins. The two major forms are ROMANO-WARD SYNDROME and JERVELL-LANGE NIELSEN SYNDROME.

Identifiers
MeSHLong QT Syndrome
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.