Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
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|ClinicalTrials.gov Identifier: NCT01573754|
Recruitment Status : Recruiting
First Posted : April 9, 2012
Last Update Posted : July 25, 2018
|Condition or disease||Intervention/treatment||Phase|
|Porphyria Cutanea Tarda||Drug: Hydroxychloroquine Procedure: Phlebotomy||Phase 2|
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:
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.
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 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)
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435|
|Study Start Date :||April 2003|
|Estimated Primary Completion Date :||August 2020|
|Estimated Study Completion Date :||August 2022|
Low-dose hydroxychloroquine 100 mg by mouth twice weekly
100 mg by mouth twice weekly
Other Name: Plaquenil
Active Comparator: Phlebotomy
Phlebotomy 450 mL biweekly
450 mL every 2 weeks
- Plasma porphyrin concentration [ Time Frame: 6 months ]Upper normal limit - 9 mcg/dL or less
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01573754
|Contact: Karl E Anderson, MDfirstname.lastname@example.org|
|Contact: Csilla Kormos Hallberg, MD||409-772-4661||ckhallbe@UTMB.EDU|
|United States, Texas|
|University of Texas Medical Branch||Recruiting|
|Galveston, Texas, United States, 77555|
|Contact: Karl E Anderson, MD 409-772-4661 email@example.com|
|Contact: Csilla K Hallberg, MD 409-772.4661 firstname.lastname@example.org|
|Principal Investigator: Karl E Anderson, MD|
|Principal Investigator:||Karl E Anderson, MD||University of Texas|