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Trial record 3 of 28 for:    "porphyria" | ( Map: United States )

Hydroxychloroquine and Phlebotomy for Treating 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01573754
Recruitment Status : Recruiting
First Posted : April 9, 2012
Last Update Posted : August 16, 2019
Information provided by (Responsible Party):
The University of Texas Medical Branch, Galveston

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.

Condition or disease Intervention/treatment Phase
Porphyria Cutanea Tarda Drug: Hydroxychloroquine Procedure: Phlebotomy Phase 2

Detailed Description:

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):

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

Study Population and Main Eligibility/ Exclusion Criteria:


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:

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

Secondary Outcome Measures:

  1. Time to disappearance of a plasma fluorescence peak at neutral pH.
  2. Time to normalization of urinary total porphyrins.
  3. Time to normalization of the urinary total porphyrin pattern by HPLC
  4. Effects of susceptibility factors such as hepatitis C, inherited UROD deficiency, etc. on efficacy and safety of the two treatment methods.
  5. 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)

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435
Actual Study Start Date : March 21, 2006
Estimated Primary Completion Date : August 2020
Estimated Study Completion Date : August 2022

Arm Intervention/treatment
Experimental: Hydroxychloroquine
Low-dose hydroxychloroquine 100 mg by mouth twice weekly
Drug: Hydroxychloroquine
100 mg by mouth twice weekly
Other Name: Plaquenil

Active Comparator: Phlebotomy
Phlebotomy 450 mL biweekly
Procedure: Phlebotomy
450 mL every 2 weeks

Primary Outcome Measures :
  1. Plasma porphyrin concentration [ Time Frame: 6 months ]
    Upper normal limit - 9 mcg/dL or less

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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 identifier (NCT number): NCT01573754

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Contact: Karl E Anderson, MD 409-772-4661
Contact: Csilla Kormos Hallberg, MD 409-772-4661 ckhallbe@UTMB.EDU

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United States, Texas
University of Texas Medical Branch Recruiting
Galveston, Texas, United States, 77555
Contact: Karl E Anderson, MD    409-772-4661   
Contact: Csilla K Hallberg, MD    409-772.4661   
Principal Investigator: Karl E Anderson, MD         
Sponsors and Collaborators
The University of Texas Medical Branch, Galveston
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Principal Investigator: Karl E Anderson, MD University of Texas

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: The University of Texas Medical Branch, Galveston Identifier: NCT01573754     History of Changes
Other Study ID Numbers: FDA-2604
R01FD002604 ( U.S. FDA Grant/Contract )
First Posted: April 9, 2012    Key Record Dates
Last Update Posted: August 16, 2019
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: Through a NIH data repository at some future time.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by The University of Texas Medical Branch, Galveston:
Porphyria, rare disease, orphan disease, iron metabolism

Additional relevant MeSH terms:
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Porphyria Cutanea Tarda
Porphyrias, Hepatic
Porphyria, Erythropoietic
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antirheumatic Agents