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Harvoni Treatment Porphyria Cutanea Tarda

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ClinicalTrials.gov Identifier: NCT03118674
Recruitment Status : Recruiting
First Posted : April 18, 2017
Last Update Posted : June 26, 2018
Sponsor:
Collaborators:
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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 49 participants
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 : August 10, 2017
Estimated Primary Completion Date : August 30, 2022
Estimated Study Completion Date : August 30, 2022


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



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 > 800 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. Subjects who are enrolled in PC7206
  5. HIV infection with CD4 counts at baseline less than 350/µL or with evidence of any active AIDS-defining illnesses
  6. 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)
  7. Any ongoing active IV drug use
  8. Patients who are taking amiodarone or who have taken amiodarone within 60 days prior to enrollment
  9. 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
  10. Uncontrolled diabetes (HbG A1c >9.5% within 60 days prior to enrollment)
  11. Chronic hepatitis B
  12. Autoimmune hepatic liver injury—autoimmune hepatitis, primary biliary cholangitis/sclerosing cholangitis or overlap syndrome
  13. Alcoholic hepatitis
  14. Other metabolic disorders of the liver, e.g. Alpha 1 antitrypsin deficiency with ZZ Pi type, Wilson's disease
  15. Prior known or suspected drug-induced liver injury within 6 months of enrollment
  16. Known or suspected hepatocellular carcinoma
  17. On liver transplant list, or current MELD >12
  18. History of liver transplant
  19. Estimated GFR (Creatinine clearance) <30 mL/min (per Sofosbuvir being cleared by the kidney)
  20. Serum ALT or AST >10x normal
  21. Serum bilirubin >2 mg/dL (excluding patients with known or suspected Gilbert's syndrome)
  22. 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 ClinicalTrials.gov identifier (NCT number): NCT03118674


Contacts
Contact: Hetanshi Naik, MS 212-241-7699 hetanshi.naik@mssm.edu
Contact: Dee Faust 336-713-1442 delannin@wakehealth.edu

Locations
United States, Alabama
University of Alabama, Birmingham Recruiting
Birmingham, Alabama, United States, 35294
Contact: Angelia Johnson    205-934-0498    angeliagjohnson@uabmc.edu   
Principal Investigator: Ashwani Singal, MD,MS         
United States, California
University of California, San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Yuvraaj Kapoor, MS    415-476-8405    yuvraaj.kapoor@ucsf.edu   
Principal Investigator: D. Montgomery Bissell, MD         
United States, New York
Icahn School of Medicine at Mount Sinai Recruiting
New York, New York, United States, 10029
Contact: Karli Hedstrom, MPH    212-659-1450    karli.hedstrom@mssm.edu   
Contact: Tanushree Laud    212-659-1418    tanushree.laud@mssm.edu   
Principal Investigator: Behnam Saberi, MD         
United States, North Carolina
Wake Forest University Health Sciences Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Dee Faust    336-713-1442    delannin@wakehealth.edu   
Principal Investigator: Herbert L Bonkovsky, MD         
United States, Texas
University of Texas Medical Branch Recruiting
Galveston, Texas, United States, 77555
Contact: Csilla Hallberg, MD    409-772-6287    challberg@utmb.edu   
Principal Investigator: Kar Anderson, MD         
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Sharada Dixit    801-587-7525    sharada.dixit@hsc.utah.edu   
Principal Investigator: John Phillips, PhD         
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)
Investigators
Study Chair: Herbert L Bonkovsky, MD Wake Forest University Health Sciences

Additional Information:
Responsible Party: Wake Forest University Health Sciences
ClinicalTrials.gov Identifier: NCT03118674     History of Changes
Other Study ID Numbers: IRB00043341
U54DK083909 ( U.S. NIH Grant/Contract )
First Posted: April 18, 2017    Key Record Dates
Last Update Posted: June 26, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
Porphyrias
Porphyria, Erythropoietic
Porphyria Cutanea Tarda
Porphyrias, Hepatic
Hepatitis
Hepatitis A
Hepatitis C
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
Sofosbuvir
Ledipasvir
Ledipasvir, sofosbuvir drug combination
Antiviral Agents
Anti-Infective Agents