NCT01573754

Brief Summary

Porphyria cutanea tarda (PCT) is an iron-related disorder that responds to treatment by phlebotomy or low-dose hydroxychloroquine, but comparative data on these treatments are limited. The hypothesis is that hydroxychloroquine is noninferior to phlebotomy in terms of time to remission. Patients with well documented PCT are assigned to treatment by randomization if specific criteria are met. All patients are followed until remission - defined as achieving a normal plasma porphyrin concentration.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
48

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Mar 2006

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

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

Study Start

First participant enrolled

March 21, 2006

Completed
6 years until next milestone

First Submitted

Initial submission to the registry

April 5, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

April 9, 2012

Completed
9.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 6, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 6, 2021

Completed
1.6 years until next milestone

Results Posted

Study results publicly available

February 17, 2023

Completed
Last Updated

February 17, 2023

Status Verified

February 1, 2023

Enrollment Period

15.3 years

First QC Date

April 5, 2012

Results QC Date

February 7, 2022

Last Update Submit

February 15, 2023

Conditions

Porphyria Cutanea Tarda

Keywords

Porphyria, rare disease, orphan disease, iron metabolism

Outcome Measures

Primary Outcomes (1)

  • Remission

    Time to a decrease in plasma porphyrin concentration to less than 0.9 mcg/dL

    To end of study, an average of 3 years

Secondary Outcomes (3)

  • 50% Reduction in Plasma Porphyrin Level

    To end of study, an average of 3 years

  • 75% Reduction in Plasma Porphyrin Level

    To end of study, an average of 3 years

  • Number of Days With Normal Urinary Porphyrin Levels

    To end of study, an average of 3 years

Study Arms (2)

Hydroxychloroquine

EXPERIMENTAL

Low-dose hydroxychloroquine 100 mg by mouth twice weekly

Drug: Hydroxychloroquine

Phlebotomy

ACTIVE COMPARATOR

Phlebotomy 450 mL biweekly

Procedure: Phlebotomy

Interventions

HydroxychloroquineDRUG

100 mg by mouth twice weekly

Also known as: Plaquenil
Hydroxychloroquine
PhlebotomyPROCEDURE

450 mL every 2 weeks

Phlebotomy

Eligibility Criteria

Age18 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Documented porphyria cutanea tarda (PCT)
  • Willing to give informed consent
  • Age 18 or greater

You may not qualify if:

  • Blistering skin lesions due to another condition

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Texas Medical Branch

Galveston, Texas, 77555, United States

Location

Related Publications (1)

  • Singal AK, Kormos-Hallberg C, Lee C, Sadagoparamanujam VM, Grady JJ, Freeman DH Jr, Anderson KE. Low-dose hydroxychloroquine is as effective as phlebotomy in treatment of patients with porphyria cutanea tarda. Clin Gastroenterol Hepatol. 2012 Dec;10(12):1402-9. doi: 10.1016/j.cgh.2012.08.038. Epub 2012 Sep 14.

    PMID: 22985607DERIVED

MeSH Terms

Conditions

Porphyria Cutanea TardaPorphyriasRare Diseases

Interventions

HydroxychloroquinePhlebotomy

Condition Hierarchy (Ancestors)

Porphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

ChloroquineAminoquinolinesQuinolinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsBlood Specimen CollectionSpecimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesTherapeuticsSurgical Procedures, OperativeInvestigative Techniques

Results Point of Contact

Title
Dr. Karl Anderson
Organization
University of Texas Medical Branch Galveston
kanderso@utmb.edu
409-772-4661

Study Officials

  • Karl E Anderson, MD

    University of Texas

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 5, 2012

First Posted

April 9, 2012

Study Start

March 21, 2006

Primary Completion

July 6, 2021

Study Completion

July 6, 2021

Last Updated

February 17, 2023

Results First Posted

February 17, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share

Through a NIH data repository at some future time.

Locations

US(1)