RecruitingPhase 1NCT06324396

IMProving DRug Dosing and Outcomes for Single VEntricle Patients With Fontan Associated Liver Disease


Sponsor

Children's Mercy Hospital Kansas City

Enrollment

15 participants

Start Date

Mar 1, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

This is a single center, open-label, prospective, investigation to quantify the effects liver congestion and fibrosis has on hepatic drug metabolism and transport in children, adolescents, and young adults with Fontan circulation.


Eligibility

Min Age: 8 Years

Inclusion Criteria4

  • > 8 years
  • Status Post Fontan Completion
  • Ability to provide informed permission-assent (<18 years) or consent (≥18 years)
  • Fasting overnight (~8 hours)

Exclusion Criteria9

  • Pregnancy
  • Non-fasting
  • Non-removable metal in body or where magnetic resonance imaging (MRI) is considered unsafe
  • Sildenafil and/or Pravastatin therapy within last 2 months
  • History of underlying or laboratory evidence of underlying intestinal, metabolic, autoimmune, renal disease that can alter Sildenafil and Pravastatin disposition (absorption, metabolism, distribution, or clearance)
  • Pharmacotherapy that interacts with Sildenafil (cytochrome P450 3A4 and P450 3A5 (CYP3A4/5) inducers/inhibitors) and/or Pravastatin (organic anion transporting polypeptide (OATP1B1) inducers/inhibitors)
  • Inability to swallow a tablet
  • >5X the age-specific upper limit of normal for aspartate aminotransferase (AST) and alanine aminotransferase (ALT), total and conjugated bilirubin
  • Diarrhea in the last 24 hours *History of solid organ transplantation

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Interventions

DRUGSildenafil 10mg oral tablet (participants <20kg), or 20mg oral tablet (participants ≥20kg)

A single oral dose of sildenafil will be administered to all study subjects.

DRUGPravastatin 20 mg oral tablet (ages <13 years), or 40 mg oral tablet (≥14 years)

A single oral dose of pravastatin will be administered to all study subjects.


Locations(1)

Children's Mercy Hospital

Kansas City, Missouri, United States

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NCT06324396


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