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Trial record 1 of 1 for:    MZ101
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Evaluation of Fontan-Associated Liver Disease (FALD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03430583
Recruitment Status : Active, not recruiting
First Posted : February 13, 2018
Last Update Posted : January 14, 2020
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Mezzion Pharma Co. Ltd

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

Condition or disease Intervention/treatment
Single Ventricle Heart Disease Drug: MZ101

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.

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Study Type : Observational
Estimated 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 : February 20, 2018
Estimated Primary Completion Date : July 30, 2020
Estimated Study Completion Date : December 31, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Diseases

Group/Cohort Intervention/treatment
MZ101
Dosing per treatment regimen
Drug: MZ101
Drug




Primary Outcome Measures :
  1. Range of Liver Stiffness in Adolescents with Single Ventricle Heart Disease [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 12 months ]
    Measurement of plasma levels of BNP

  3. Effect of Drug Therapy on n-Terminal BNP (NT-proBNP) [ Time Frame: 12 months ]
    Measurement of plasma levels of NT-proBNP

  4. Effect of Drug Therapy on MicroRNA Measures. [ Time Frame: 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 [ Time Frame: 12 months ]
    Measurement of ELF Score


Biospecimen Retention:   Samples With DNA
Plasma and serum


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Male and Female subjects 12 - 19 years old with meet the inclusion/exclusion criteria.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03430583


Locations
Show Show 17 study locations
Sponsors and Collaborators
Mezzion Pharma Co. Ltd
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
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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
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Responsible Party: Mezzion Pharma Co. Ltd
ClinicalTrials.gov Identifier: NCT03430583    
Other Study ID Numbers: PHN-Udenafil-04
U01HL068270 ( U.S. NIH Grant/Contract )
First Posted: February 13, 2018    Key Record Dates
Last Update Posted: January 14, 2020
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

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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:
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Heart Diseases
Cardiovascular Diseases