Harvoni Treatment Porphyria Cutanea Tarda
Condition: Porphyria Cutanea Tarda; Hepatitis C
MeSH terms: Hepatitis A; Hepatitis C; Hepatitis; Porphyria Cutanea Tarda; Porphyrias, Hepatic; Porphyria, Erythropoietic; Porphyrias
Intervention: Harvoni
Intervention description: One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Intervention MeSH terms: Sofosbuvir; Ledipasvir, sofosbuvir drug combination; Ledipasvir
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 23.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Interventional, Open-label, PCT, Hepatitis C

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.
    

Eligibility criteria:

        Inclusion Criteria

          1. Willing and able to give informed consent

          2. ≥18 years of age

          3. Symptoms and signs consistent with PCT and well documented biochemical diagnosis
             (urinary total porphyrin excretion > 500 mcg/g Creatinine with HPLC pattern typical of
             PCT-predominance of 8- and 7-carboxyl porphyrins)

          4. Clinical diagnosis of PCT established by a study PI

          5. Chronic hepatitis C: HCV RNA positive and quantifiable in serum detected within 90
             days of enrollment, and documented HCV genotypes 1,4, 5, or 6 for which Harvoni is an
             approved therapy.

          6. Women of child-bearing potential must be willing to avoid pregnancy and use an
             accepted and effective contraceptive method during treatment.

        Exclusion Criteria

          1. Women who are pregnant or who are breast-feeding

          2. Patients who have already started treatment of PCT with phlebotomy or low dose
             hydroxychloroquine or chloroquine, or who have been in such treatment in the past 30
             days

          3. Patients who have already started another treatment regimen for CHC, or who have taken
             such treatment in the past 30 days

          4. HIV infection with CD4 counts at baseline less than 350/µL or with evidence of any
             active AIDS-defining illnesses

          5. Ongoing active alcohol abuse, defined as a history of drinking more than 25 drinks of
             alcohol per week during most weeks in the prior 4 months (History of prior, but not
             current alcohol abuse will NOT be grounds for exclusion because we seek to treat
             subjects with PCT and CHC of the type typically seen in clinical practice)

          6. Any ongoing active IV drug use

          7. Patients who are taking amiodarone or who have taken amiodarone within 60 days prior
             to enrollment

          8. Patients who are taking, or within the prior 28 days have taken, rifampicin or St
             John's wort (Hypericum perforatum), both of which are P-gp inducers, which may
             significantly reduce the drug levels and therapeutic effects of Harvoni

          9. Uncontrolled diabetes (Hgb A1c >9.5% within 60 days prior to enrollment)

         10. Chronic hepatitis B

         11. Autoimmune hepatic liver injury-autoimmune hepatitis, primary biliary
             cholangitis/sclerosing cholangitis or overlap syndrome

         12. Alcoholic hepatitis

         13. Other metabolic disorders of the liver, e.g. Alpha 1 antitrypsin deficiency with ZZ Pi
             type, Wilson's disease

         14. Prior known or suspected drug-induced liver injury within 6 months of enrollment

         15. Known or suspected hepatocellular carcinoma

         16. On liver transplant list, or current MELD >12

         17. History of liver transplant

         18. Estimated GFR (Creatinine clearance) <30 mL/min (per Sofosbuvir being cleared by the
             kidney)

         19. Serum ALT or AST >10x normal

         20. Serum bilirubin >2 mg/dL (excluding patients with known or suspected Gilbert's
             syndrome)

         21. Any other comorbid condition, which, in the opinion of the investigator, precludes
             participation