Clincosm LogoSign in Get a demo

FALD: Evaluation of Fontan-Associated Liver Disease

Sponsor
Mezzion Pharma Co. Ltd (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03430583
Collaborator
National Heart, Lung, and Blood Institute (NHLBI) (NIH)
100
Enrollment
17
Locations
42.3
Anticipated Duration (Months)
5.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

A study to evaluate the efficacy of MZ101 therapy in reducing liver stiffness.

Condition or Disease Intervention/Treatment Phase

Detailed Description

This is open-label, long-term prospective study in a cohort of Fontan subjects taking drug daily for one year. The study will determine the scope of hepatic stiffness in the cohort by ultrasound and magnetic resonance imaging elastography and evaluate MZ101 pharmacotherapy as a potentially efficacious treatment to reduce liver stiffness.

Study Design

Study Type:
Observational
Anticipated Enrollment :
100 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Imaging and Biomarker Evaluation of Hepatic Stiffness in Children Enrolled in the Fontan Udenafil Exercise Longitudinal Study
Actual Study Start Date :
Feb 20, 2018
Anticipated Primary Completion Date :
Jun 30, 2021
Anticipated Study Completion Date :
Aug 31, 2021

Arms and Interventions

Arm Intervention/Treatment
MZ101

Dosing per treatment regimen

Drug: MZ101
Drug

Outcome Measures

Primary Outcome Measures

  1. Range of Liver Stiffness in Adolescents with Single Ventricle Heart Disease [12 months]

    Liver Stiffness as measured by Shear Wave Ultrasound Elastography (meters/second) or Magnetic Resonance Ultrasound (kilopascals).

Secondary Outcome Measures

  1. Effect of Drug Therapy on Liver Stiffness [12 months]

    Liver Stiffness as measured by Shear Wave Ultrasound Elastography (meters/second) or Magnetic Resonance Ultrasound (kilopascals).

  2. Effect of Drug Therapy on Brain Type Natriuretic Peptide (BNP) Levels [12 months]

    Measurement of plasma levels of BNP

  3. Effect of Drug Therapy on n-Terminal BNP (NT-proBNP) [12 months]

    Measurement of plasma levels of NT-proBNP

  4. Effect of Drug Therapy on MicroRNA Measures. [12 months]

    Measurement of MicroRNA by miRNA qRT-PCR analysis of total RNA from plasma

  5. Effect of Drug Therapy on Enhanced Liver Fibrosis (ELF) Score [12 months]

    Measurement of ELF Score

Eligibility Criteria

Criteria

Ages Eligible for Study:
12 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  1. Enrollment in on-going Phase 3 Open-Label Safety Study

  2. Informed assent from subject, informed consent from parent/legal guardian as appropriate

Exclusion Criteria:

  1. Non-enrollment in the on-going Phase 3 Open-Label Study

  2. Subjects with contra-indications for MRI (these subjects will be excluded from the MRI component of this study)

  3. Other exclusionary criteria will match those used for the Open-Label Safety Study

Contacts and Locations

Locations

Site City State Country Postal Code
1 Children's Hospital Colorado Aurora Colorado United States 80045
2 Nemours Cardiac Center/Alfred I. duPont Hospital for Children Wilmington Delaware United States 19803
3 Children's National Medical Center Washington District of Columbia United States 20008
4 Children's Healthcare of Atlanta Atlanta Georgia United States 30322
5 Children's Mercy Hospital Kansas City Kansas United States 64108
6 Boston Children's Hospital Boston Massachusetts United States 02115
7 University of Michigan Congenital Heart Center Ann Arbor Michigan United States 48109-4204
8 Children's Mercy Hospital Kansas City Kansas City Missouri United States 64108
9 University of Nebraska Children's Hospital and Medical Center Omaha Nebraska United States 68114
10 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
11 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
12 Medical University of South Carolina Charleston South Carolina United States 29425
13 Vanderbilt University Medical Center Nashville Tennessee United States 37232
14 Texas Children's Hospital Houston Texas United States 77030
15 Primary Children's Medical Hospital/Dept. of Pediatric Cardiology Salt Lake City Utah United States 84113
16 Children's Hospital of Wisconsin Milwaukee Wisconsin United States 53226
17 The Hospital for Sick Children Toronto Ontario Canada ON M5G 1X8

Sponsors and Collaborators

  • Mezzion Pharma Co. Ltd
  • National Heart, Lung, and Blood Institute (NHLBI)

Investigators

  • Principal Investigator: David J Goldberg, MD, Children's Hospital of Philadelphia
  • Principal Investigator: Kurt R Schumacher, MD, University of Michigan Congenital Heart Center/C.S. Mott Children's Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Mezzion Pharma Co. Ltd
ClinicalTrials.gov Identifier:
NCT03430583
Other Study ID Numbers:
  • PHN-Udenafil-04
  • U01HL068270
First Posted:
Feb 13, 2018
Last Update Posted:
Nov 24, 2020
Last Verified:
Nov 1, 2020
Individual Participant Data (IPD) Sharing Statement:
Yes
Plan to Share IPD:
Yes
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Mezzion Pharma Co. Ltd
Fontan
Ultrasound
Magnetic Resonance Imaging Elastography
Additional relevant MeSH terms:
Heart Diseases
Univentricular Heart

Study Results

No Results Posted as of Nov 24, 2020