NCT03118674

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
23

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2017

Typical duration for phase_2

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 13, 2017

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 18, 2017

Completed
5 months until next milestone

Study Start

First participant enrolled

September 6, 2017

Completed
4.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 4, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 4, 2022

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

July 3, 2023

Completed
Last Updated

July 3, 2023

Status Verified

May 1, 2023

Enrollment Period

4.5 years

First QC Date

April 13, 2017

Results QC Date

May 5, 2023

Last Update Submit

June 14, 2023

Conditions

Porphyria Cutanea TardaHepatitis C

Keywords

Interventional, Open-label, PCT, Hepatitis C

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Resolution of Active PCT by 7 Months After Start of Therapy

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

    7 months

Secondary Outcomes (3)

  • Time to Resolution of Active PCT

    through study completion, an average of 1 year

  • Number of Participants With Complete Biochemical Remission of PCT

    12 Months

  • Number of Participants With Cure of CHC

    Up to 15 months

Study Arms (1)

Harvoni

EXPERIMENTAL

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

Interventions

HarvoniDRUG

One capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks

Also known as: ledipasvir, 90 mg + sofosbuvir, 400 mg
Harvoni

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Willing and able to give informed consent
  • ≥18 years of age
  • 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)
  • Clinical diagnosis of PCT established by a study PI
  • 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.
  • Women of child-bearing potential must be willing to avoid pregnancy and use an accepted and effective contraceptive method during treatment.

You may not qualify if:

  • Women who are pregnant or who are breast-feeding
  • 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
  • Patients who have already started another treatment regimen for CHC, or who have taken such treatment in the past 30 days
  • HIV infection with CD4 counts at baseline less than 350/µL or with evidence of any active AIDS-defining illnesses
  • Any ongoing active IV drug use
  • Patients who are taking amiodarone or who have taken amiodarone within 60 days prior to enrollment
  • 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
  • Uncontrolled diabetes (Hgb A1c \>9.5% within 60 days prior to enrollment)
  • Chronic hepatitis B
  • Autoimmune hepatic liver injury-autoimmune hepatitis, primary biliary cholangitis/sclerosing cholangitis or overlap syndrome
  • Alcoholic hepatitis
  • Other metabolic disorders of the liver, e.g. Alpha 1 antitrypsin deficiency with ZZ Pi type, Wilson's disease
  • Prior known or suspected drug-induced liver injury within 6 months of enrollment
  • Known or suspected hepatocellular carcinoma
  • On liver transplant list, or current MELD \>12
  • +5 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

University of Texas Medical Branch

Galveston, Texas, 77555, United States

Location

Related Publications (1)

  • Bonkovsky HL, Rudnick SP, Ma CD, Overbey JR, Wang K, Faust D, Hallberg C, Hedstrom K, Naik H, Moghe A, Anderson KE. Ledipasvir/Sofosbuvir Is Effective as Sole Treatment of Porphyria Cutanea Tarda with Chronic Hepatitis C. Dig Dis Sci. 2023 Jun;68(6):2738-2746. doi: 10.1007/s10620-023-07859-8. Epub 2023 Feb 22.

    PMID: 36811718DERIVED

Related Links

MeSH Terms

Conditions

Porphyria Cutanea TardaHepatitis C

Interventions

ledipasvir, sofosbuvir drug combinationledipasvirSofosbuvir

Condition Hierarchy (Ancestors)

Porphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesPorphyriasMetabolic DiseasesNutritional and Metabolic DiseasesBlood-Borne InfectionsCommunicable DiseasesInfectionsHepatitis, Viral, HumanVirus DiseasesFlaviviridae InfectionsRNA Virus InfectionsHepatitis

Intervention Hierarchy (Ancestors)

Uridine MonophosphateUracil NucleotidesPyrimidine NucleotidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleotidesNucleic Acids, Nucleotides, and NucleosidesRibonucleotides

Limitations and Caveats

We were not able to recruit as many subjects as we had originally planned and hoped to enroll \[original target n = 49\].

Results Point of Contact

Title
Herbert L. Bonkovsky, MD
Organization
Wake Forest School of Medicine
hbonkovs@wakehealth.edu
3367137341

Study Officials

  • Herbert L Bonkovsky, MD

    Wake Forest University Health Sciences

    STUDY CHAIR

  • Sean Rudnick, MD

    Wake Forest University Health Sciences

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 13, 2017

First Posted

April 18, 2017

Study Start

September 6, 2017

Primary Completion

March 4, 2022

Study Completion

March 4, 2022

Last Updated

July 3, 2023

Results First Posted

July 3, 2023

Record last verified: 2023-05

Data Sharing

IPD Sharing
Will not share

Locations

US(3)