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| ID | Type | Description | Link |
|---|---|---|---|
| U01HL068270 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
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A study to evaluate the efficacy of MZ101 therapy in reducing liver stiffness.
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Udenafil Continuation (U+) | Experimental | Subjects who were receiving Udenafil 87.5 mg BID in FUEL study prior to enrolling to this study (FALD). |
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| Udenafil Naive (U-) | Experimental | Subjects who were receiving Placebo BID in FUEL study prior to enrolling to this study (FALD). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Udenafil | Drug | Open-label extension with Udenafil 87.5 mg BID treatment for 52 weeks. |
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| Measure | Description | Time Frame |
|---|---|---|
| Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Ultrasound Shear Wave Elastography | Liver stiffness was measured at baseline and 52 weeks using Ultrasound Shear Wave Elastography (USWE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness. | 52 weeks |
| Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Magnetic Resonance Elastography | Liver stiffness was measured at baseline and 52 weeks using Magnetic Resonance Elastography (MRE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness. | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Effect of Udenafil 87.5 mg BID on Enhanced Liver Fibrosis (ELF) Score After 52 Weeks of Treatment | Overall ELF score. ELF score is based on the serum levels of hyaluronic acid (HA), amino-terminal propeptide of type III collagen (PIIINP), and tissue inhibitor of metalloproteinase-1 (TIMP-1), and calculated using the following formula: ELF score= 2.494+0.846 In(CHA)+0.391 In(CPIIINP)+0.391 In(CTIMP-1). The levels of each component were as assessed individually and as part of the calculated ELF score as continuous variables. The normal range in healthy subjects ≤20 years of age is 6.9 - 8.8. Correlations between ELF and the degree of liver fibrosis have not been established in patients with Fontan circulation, however, a decrease in ELF score is associated with improved liver health in other disease states. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| 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 | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States | ||
| Nemours Cardiac Center/Alfred I. duPont Hospital for Children |
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| ID | Title | Description |
|---|---|---|
| FG000 | Udenafil Continuation (U+) | Treatment subgroup previously exposed to Udenafil |
| FG001 | Udenafil Naive (U-) | Treatment subgroup naive to Udenafil |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 11, 2017 |
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| 52 weeks |
| Effect of Udenafil 87.5 mg BID on Brain Natriuretic Peptide After 52 Weeks of Treatment | Brain Natriuretic Peptide (BNP) was measured at baseline and 52 weeks of treatment. BNP is a biomarker of overall cardiac function across a variety of disease states. A decrease in value is associated with improved cardiac function. | 52 weeks |
| Wilmington |
| Delaware |
| 19803 |
| United States |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20008 | United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | United States |
| Children's Mercy Hospital | Kansas City | Kansas | 64108 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Michigan Congenital Heart Center | Ann Arbor | Michigan | 48109-4204 | United States |
| Children's Mercy Hospital Kansas City | Kansas City | Missouri | 64108 | United States |
| University of Nebraska Children's Hospital and Medical Center | Omaha | Nebraska | 68114 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| Primary Children's Medical Hospital/Dept. of Pediatric Cardiology | Salt Lake City | Utah | 84113 | United States |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| The Hospital for Sick Children | Toronto | Ontario | ON M5G 1X8 | Canada |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Udenafil Continuation (U+) | Subset of subjects on Open-label extension study with Udenafil 87.5 mg BID treatment for 52 weeks, who received active Udenafil 87.5 mg BID treatment in FUEL study prior to enrolling to this study (FALD). |
| BG001 | Udenafil Naive (-) | Subset of subjects on Open-label extension study with Udenafil 87.5 mg BID treatment for 52 weeks, who received Placebo BID treatment in FUEL study prior to enrolling to this study (FALD). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants | No |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants | No |
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| Race (NIH/OMB) | Count of Participants | Participants | No |
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| Body Mass Index (BMI) | Mean | Standard Deviation | kilograms per meter squared |
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| Height | Mean | Standard Deviation | cm |
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| Weight | Mean | Standard Deviation | Kg |
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| Liver Stiffness by Ultrasound Shear Wave Elastography (USWE) | Baseline liver stiffness was measured in subjects at the start of the study by Ultrasound Shear Wave Elastography (USWE) (meters/second). | Baseline results presented for subjects who have data at 52 weeks (i.e., paired-data points). | Mean | Standard Deviation | meters/sec |
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| Liver Stiffness by Magnetic Resonance Elastography (MRE) | Baseline liver stiffness was measured in subjects at the start of the study by Magnetic Resonance Elastography (kilopascal; kPa). | Baseline results presented for subjects who have data at 52 weeks (i.e., paired-data points). | Mean | Standard Deviation | KiloPascal (kPa) |
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| Enhanced Liver Fibrosis (ELF) | ELF score is based on the serum levels of hyaluronic acid (HA), amino-terminal propeptide of type III collagen (PIIINP), and tissue inhibitor of metalloproteinase-1 (TIMP-1), and calculated using the following formula: ELF score= 2.494+0.846 In(CHA)+0.391 In(CPIIINP)+0.391 In(CTIMP-1). The normal range in healthy subjects ≤20 years of age is 6.9 - 8.8. Correlations between ELF and the degree of liver fibrosis have not been established in patients with Fontan circulation, however, a decrease in ELF score is associated with improved liver health in other disease states. | Baseline results presented for subjects who have data at 52 weeks (i.e., paired-data points). | Mean | Standard Deviation | score on a scale |
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| Brain Natriuretic Peptide (BNP) | Level of Brain Natriuretic Peptide (BNP) was measured in study subjects at baseline. | Baseline results presented for subjects who have data at 52 weeks (i.e., paired-data points). | Mean | Standard Deviation | pg/ml |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Ultrasound Shear Wave Elastography | Liver stiffness was measured at baseline and 52 weeks using Ultrasound Shear Wave Elastography (USWE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness. | Only participants with paired data points were included in the analysis population. | Posted | Mean | Standard Deviation | meters per second | 52 weeks |
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| Primary | Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Magnetic Resonance Elastography | Liver stiffness was measured at baseline and 52 weeks using Magnetic Resonance Elastography (MRE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness. | Only participants with paired data points were included in the analysis population. | Posted | Mean | Standard Deviation | kiloPascal (kPa) | 52 weeks |
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| Secondary | Effect of Udenafil 87.5 mg BID on Enhanced Liver Fibrosis (ELF) Score After 52 Weeks of Treatment | Overall ELF score. ELF score is based on the serum levels of hyaluronic acid (HA), amino-terminal propeptide of type III collagen (PIIINP), and tissue inhibitor of metalloproteinase-1 (TIMP-1), and calculated using the following formula: ELF score= 2.494+0.846 In(CHA)+0.391 In(CPIIINP)+0.391 In(CTIMP-1). The levels of each component were as assessed individually and as part of the calculated ELF score as continuous variables. The normal range in healthy subjects ≤20 years of age is 6.9 - 8.8. Correlations between ELF and the degree of liver fibrosis have not been established in patients with Fontan circulation, however, a decrease in ELF score is associated with improved liver health in other disease states. | Only participants with paired data points were included in the analysis population. | Posted | Mean | Standard Deviation | score on a scale | 52 weeks |
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| Secondary | Effect of Udenafil 87.5 mg BID on Brain Natriuretic Peptide After 52 Weeks of Treatment | Brain Natriuretic Peptide (BNP) was measured at baseline and 52 weeks of treatment. BNP is a biomarker of overall cardiac function across a variety of disease states. A decrease in value is associated with improved cardiac function. | Only participants with paired data points were included in the analysis population. | Posted | Mean | Standard Deviation | pg/ml | 52 weeks |
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Not applicable, as all safety information was collected in the Study PHN-Udenafil-03 (parent study) and will be reported as part of the results from that study. All-Cause Mortality, Serious, and Other (Not Including Serious) Adverse Events were not monitored/assessed as part of Study PHN-Udenafil-04.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Udenafil 87.5 mg BID | Open-label extension with Udenafil 87.5 mg BID treatment for 52 weeks. | 0 | 0 | 0 | 0 | 0 | 0 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Scott Oglesby | Mezzion Pharmaceuticals | 919-260-5782 | Oglesby@granzer.biz |
| May 18, 2025 |
| Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| C419664 | udenafil |
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