Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
| Status: | Recruiting |
|---|---|
| Conditions: | Gastrointestinal, Hematology |
| Therapuetic Areas: | Gastroenterology, Hematology |
| Healthy: | No |
| Age Range: | 18 - 100 |
| Updated: | 7/27/2018 |
| Start Date: | April 2003 |
| End Date: | August 2022 |
| Contact: | Karl E Anderson, MD |
| Email: | kanderso@utmb.edu |
| Phone: | 409-772-4661 |
A Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435
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.
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.
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:
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:
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)
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:
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:
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)
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
We found this trial at
1
site
301 University Blvd
Galveston, Texas 77555
Galveston, Texas 77555
(409) 772-1011
Principal Investigator: Karl E Anderson, MD
Phone: 409-772-4661
University of Texas Medical Branch Established in 1891 as the University of Texas Medical Department,...
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