NCT03965351

TRPV2 Agonists in the Fontan Circulation

TRPV2 Agonists in Fontan Circulation Patients

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

There are currently very few proven pharmacologic options available for these patients. The recent discovery of transient receptor potential vanilloid (TRPV) channels, particularly TRPV2 channels, in the cardiovascular system is promising as a potential pathway for pharmacologic intervention for Fontan patients. Probenecid, a drug best known as a treatment for gout or as a penicillin adjunct, acts as a TRPV2 agonist and has recently become the subject of study as a model therapy for the treatment of cardiomyopathy due to its positive inotropic and lusitropic effects. The purpose of this pilot study is to determine if probenecid will improve magnetic resonance (MRI) parameters of systolic and/or diastolic dysfunction as well as associated symptoms in patients with a Fontan circulation. The investigators will quantitatively assess functional improvement with pre- and post-treatment cardiopulmonary exercise testing.

Conditions

• Fontan

Interventions

Type	Name	Description
DRUG	Probenecid	This is a cross-over pilot study where participants will receive both the study medication (probenecid), as well as a placebo to see if the study medication improves magnetic resonance parameters of systolic and diastolic dysfunction that occurs in the Fontan population.
OTHER	Placebo	placebo

Timeline

• Start date: 2017-03-21
• Primary completion: 2020-06-30
• Completion: 2020-12-31
• First posted: 2019-05-29
• Last updated: 2021-07-09
• Results posted: 2021-07-09

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study