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Harvoni Treatment Porphyria Cutanea Tarda

Phase 2
Completed
Conditions
Hepatitis C
Porphyria Cutanea Tarda
Interventions
Registration Number
NCT03118674
Lead Sponsor
Wake Forest University Health Sciences
Brief Summary

In the medical literature there case reports that Harvoni improves symptoms in patients with PCT. However, this has never been systematically tested. Therefore, the purpose of this study is to assess whether Harvoni alone is an effective therapy of active PCT in patients with Chronic Hepatitis C.

Detailed Description

This is a clinical trial, which means its purpose is to study an intervention or treatment. In this study all patients with PCT will be given a standard dose of Harvoni and monitored for two years. Currently there are two standard therapies for PCT, phlebotomies (removing certain amounts of blood at specific intervals), or low dose hydroxychloroquine (an oral pill). These treatments are used for patients with PCT whether or not they also have HCV. For patients with HCV however, we do not know whether treating the HCV first will also resolve the PCT symptoms. There will be an initial visit to determine whether participants are eligible to be in the study. If a participant is found to be eligible, he/she will be asked come to the study site once every month over the course of one year, and then once every 3 months for an additional year. There will be approximately 17 visits over the course of the whole study. At these visits the study doctors will check in with the participant and some blood and urine samples will be taken. Participants will not be charged for any of the lab tests that are being done as a part of this study alone. All participants in this study will receive the Harvoni pills at no cost to them.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
23
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
HarvoniHarvoni1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis
Primary Outcome Measures
NameTimeMethod
Number of Participants With Resolution of Active PCT by 7 Months After Start of Therapy7 months

Resolution of active PCT, defined as normalization of plasma porphyrins (less than 0.9 mcg/dL) by 7 months after start of therapy

Secondary Outcome Measures
NameTimeMethod
Number of Participants With Cure of CHCUp to 15 months

Defined as no detectable HCV RNA at end of treatment and persisting for at least 12 weeks after end of treatment.

Number of Participants With Complete Biochemical Remission of PCT12 Months

Defined as a decrease of the sum of urinary uro- and hepta-carboxyl porphyrins to less than 100 mcg/g creatinine and a normal urine porphyrin HPLC pattern defined as the total of highly carboxylated porphyrins (uro- and heptacarboxyl-porphyrins) being less than that of coproporphyrins, and the absence of a plasma fluorescence peak by fluorescence scanning

Time to Resolution of Active PCTthrough study completion, an average of 1 year

Time to resolution of active PCT, defined as cessation of any new blisters or bullae and normalization of plasma porphyrins

Trial Locations

Locations (3)

University of Texas Medical Branch

🇺🇸

Galveston, Texas, United States

Wake Forest University Health Sciences

🇺🇸

Winston-Salem, North Carolina, United States

University of California, San Francisco

🇺🇸

San Francisco, California, United States

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