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Harvoni Treatment Porphyria Cutanea Tarda

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03118674
Recruitment Status : Recruiting
First Posted : April 18, 2017
Last Update Posted : July 24, 2020
Gilead Sciences
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institutes of Health (NIH)
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Porphyria Cutanea Tarda Hepatitis C Drug: Harvoni Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 49 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Newer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects With Chronic Hepatitis C
Actual Study Start Date : September 6, 2017
Estimated Primary Completion Date : August 30, 2023
Estimated Study Completion Date : August 30, 2024

Arm Intervention/treatment
Experimental: Harvoni
1 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
Drug: Harvoni
One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Other Name: ledipasvir, 90 mg + sofosbuvir, 400 mg

Primary Outcome Measures :
  1. Resolution of active PCT by 7 months after start of therapy [ Time Frame: 7 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 :
  1. Time to resolution of active PCT [ Time Frame: 12 months ]
    Time to resolution of active PCT, defined as cessation of any new blisters or bullae and normalization of plasma porphyrins

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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 identifier (NCT number): NCT03118674

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Contact: Hetanshi Naik, MS 212-241-7699
Contact: Dee Faust 336-713-1442

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United States, Alabama
McGuire, Brendan M. MD Withdrawn
Birmingham, Alabama, United States, 35294
United States, California
University of California, San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Yuvraaj Kapoor, MS    415-476-8405   
Principal Investigator: D. Montgomery Bissell, MD         
United States, Florida
University of Miami Liver center Withdrawn
Miami, Florida, United States, 33136
United States, New York
Icahn School of Medicine at Mount Sinai Withdrawn
New York, New York, United States, 10029
United States, North Carolina
Wake Forest University Health Sciences Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Dee Faust    336-713-1442   
Principal Investigator: Herbert L Bonkovsky, MD         
Sub-Investigator: Sean Rudnick, MD         
United States, Pennsylvania
Kimmel School of Medicine of T Jefferson University Withdrawn
Philadelphia, Pennsylvania, United States, 19085
United States, Texas
University of Texas Medical Branch Recruiting
Galveston, Texas, United States, 77555
Contact: Csilla Hallberg, MD    409-772-6287   
Principal Investigator: Kar Anderson, MD         
United States, Utah
University of Utah Withdrawn
Salt Lake City, Utah, United States, 84132
Sponsors and Collaborators
Wake Forest University Health Sciences
Gilead Sciences
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institutes of Health (NIH)
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Study Chair: Herbert L Bonkovsky, MD Wake Forest University Health Sciences
Study Director: Sean Rudnick, MD Wake Forest University Health Sciences
Additional Information:
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Responsible Party: Wake Forest University Health Sciences Identifier: NCT03118674    
Other Study ID Numbers: IRB00043341
U54DK083909 ( U.S. NIH Grant/Contract )
First Posted: April 18, 2017    Key Record Dates
Last Update Posted: July 24, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Wake Forest University Health Sciences:
Interventional, Open-label, PCT, Hepatitis C
Additional relevant MeSH terms:
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Hepatitis A
Hepatitis C
Porphyria Cutanea Tarda
Porphyrias, Hepatic
Porphyria, Erythropoietic
Liver Diseases
Digestive System Diseases
Hepatitis, Viral, Human
Virus Diseases
Enterovirus Infections
Picornaviridae Infections
RNA Virus Infections
Flaviviridae Infections
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Ledipasvir, sofosbuvir drug combination
Antiviral Agents
Anti-Infective Agents