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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03118674
Recruitment Status : Active, not recruiting
First Posted : April 18, 2017
Last Update Posted : May 25, 2020
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

Tracking Information
First Submitted Date  ICMJE April 13, 2017
First Posted Date  ICMJE April 18, 2017
Last Update Posted Date May 25, 2020
Actual Study Start Date  ICMJE August 10, 2017
Estimated Primary Completion Date August 30, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 13, 2017)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 13, 2017)
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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Harvoni Treatment Porphyria Cutanea Tarda
Official Title  ICMJE Newer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects With Chronic Hepatitis C
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Porphyria Cutanea Tarda
  • Hepatitis C
Intervention  ICMJE 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
Study Arms  ICMJE 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
Intervention: Drug: Harvoni
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: April 13, 2017)
49
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 30, 2024
Estimated Primary Completion Date August 30, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03118674
Other Study ID Numbers  ICMJE IRB00043341
U54DK083909 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Wake Forest University Health Sciences
Study Sponsor  ICMJE Wake Forest University Health Sciences
Collaborators  ICMJE
  • Gilead Sciences
  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • National Institutes of Health (NIH)
Investigators  ICMJE
Study Chair: Herbert L Bonkovsky, MD Wake Forest University Health Sciences
Study Director: Sean Rudnick, MD Wake Forest University Health Sciences
PRS Account Wake Forest University Health Sciences
Verification Date May 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP