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

NCT ID: NCT06324396

Last Updated: 2026-01-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-01

Study Completion Date

2026-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Fontan Circulation

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

This is a single center, open-label, prospective, investigation to quantify the effects of liver congestion and fibrosis has on hepatic statin transport (SA1) and response (SA2) in children, adolescents, and young adults with Fontan circulation.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Assessment Arm

Assessment group will receive a single dose of oral sildenafil and oral pravastatin.

Group Type EXPERIMENTAL

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

Intervention Type DRUG

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

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

Intervention Type DRUG

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

Interventions

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

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

Intervention Type DRUG

Pravastatin 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.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* 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
Minimum Eligible Age

8 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Indiana Clinical and Translational Sciences Institute

OTHER

Sponsor Role collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role collaborator

Children's Mercy Hospital Kansas City

OTHER

Sponsor Role lead

Responsible Party

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Jon Wagner

Director, Division of Clinical Pharmacology, Toxicology, and Therapeutic Innovation

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Jonathan Wagner, DO

Role: PRINCIPAL_INVESTIGATOR

Children's Mercy

Locations

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Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Jonthan Wagner, DO

Role: CONTACT

816-731-7240

Facility Contacts

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Jonathan Wagner, DO

Role: primary

Other Identifiers

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5T32HD069038

Identifier Type: NIH

Identifier Source: secondary_id

View Link

STUDY00002974

Identifier Type: -

Identifier Source: org_study_id

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