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NCT Number: NCT01573754

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

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.

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Key information

Conditions

Porphyria Cutanea Tarda Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Disease Attributes Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Porphyrias Porphyrias, Hepatic Rare Diseases Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Texas Medical Branch

Galveston, Texas, 77555, United States

About this study

Study Design: Pragmatic Interventional study

Primary Study Objective: To determine and compare time to remission with treatment with low-dose hydroxychloroquine or repeated phlebotomy in participants with PCT.

Secondary Study Objective(s):

  • To assess the effects of susceptibility factors on responses to treatment of PCT by these methods.
  • To determine and compare rates of recurrence of PCT after treatment with low-dose hydroxychloroquine or phlebotomy.

Study Population and Main Eligibility/ Exclusion Criteria:

Treatment:

Hydroxychloroquine 100 mg twice weekly for up to 24 months by mouth vs. phlebotomy 450 mL biweekly until target serum ferritin reached, or up to 24 months.

Safety Issues- 1. Side effects of phlebotomy or hydroxychloroquine, which are the same as in clinical practice.

Primary Outcome Measures:

  • Time to achievement of a normal plasma total porphyrin level.
  • Tolerability and safety of both treatments

Secondary Outcome Measures:

  • Time to 50% reduction in plasma porphyrin levels. 2. Time to 75% reduction in plasma porphyrin levels. 3. Time to normal urinary porphyrin levels
  • Time to disappearance of a plasma fluorescence peak at neutral pH.
  • Time to normalization of urinary total porphyrins.
  • Time to normalization of the urinary total porphyrin pattern by HPLC
  • Effects of susceptibility factors such as hepatitis C, inherited UROD deficiency, etc. on efficacy and safety of the two treatment methods.
  • Rates of recurrence after each type of treatment and the effects of susceptibility factors on recurrence rates.

Statistical Considerations (sample size and analysis plan): Time to achieving biochemical endpoints will be determined from individual subject data. Outcome measures such as time to remission will be compared using Cox proportional models to study the effects of susceptibility factors on the hazard ratio to compare the two treatments. Additional modeling will assess factors affecting the frequency of recurrence and seasonality effects using logistic regression modeling and log-rank testing, respectively.

Sponsors: National Institutes of Health (NIH)

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • Blistering skin lesions due to another condition

Treatment and study plan

Hydroxychloroquine

Drug

100 mg by mouth twice weekly

Other names: Plaquenil

Phlebotomy

Procedure

450 mL every 2 weeks

Primary outcomes

  1. Remission

    Time frame: To end of study, an average of 3 years

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

Secondary outcomes

  1. 50% Reduction in Plasma Porphyrin Level

    Time frame: To end of study, an average of 3 years

    50% reduction in plasma porphyrin level during treatment

  2. 75% Reduction in Plasma Porphyrin Level

    Time frame: To end of study, an average of 3 years

    Time to 75% reduction in plasma porphyrin level during treatment

  3. Number of Days With Normal Urinary Porphyrin Levels

    Time frame: To end of study, an average of 3 years

    Number of days to normal urinary porphyrin levels for participants treated for Porphyria Cutanea Tarda (PCT). Days are summed together for all participants for a single value in each Arm.

Sponsors and collaborators

Lead sponsor

The University of Texas Medical Branch, Galveston

Other

Registry information

Official study title

A Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435

Important dates

Study start
2006
Primary completion
2021
Study completion
2021
First posted
Apr 9, 2012
Registry last updated
Feb 17, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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