Porphyria Cutanea TardaPhase 2NCT01573754
Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
A Phase 2 study testing Hydroxychloroquine and Phlebotomy in people with Porphyria Cutanea Tarda, run by The University of Texas Medical Branch, Galveston at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Porphyria Cutanea Tarda
- What is being tested?
- Hydroxychloroquine and Phlebotomy
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Mar 2006to 6 Jul 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Documented porphyria cutanea tarda (PCT)
- Willing to give informed consent
- Age 18 or greater
Who cannot join
- Blistering skin lesions due to another condition
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas Medical Branch
Galveston, Texas, 77555
About this study
Official title: 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.
Read the 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):
- 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)
- Conditions
- Porphyria Cutanea Tarda
- Being tested
- Hydroxychloroquine, Phlebotomy
- Keywords
- Porphyria, rare disease, orphan disease, iron metabolism
- Medical subject headings
- Porphyria Cutanea Tarda, Porphyrias, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called Plaquenil
100 mg by mouth twice weekly
PhlebotomyProcedure or surgery
450 mL every 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| Hydroxychloroquine | Experimental | Low-dose hydroxychloroquine 100 mg by mouth twice weekly Receives: Drug: Hydroxychloroquine |
| Phlebotomy | Active comparator | Phlebotomy 450 mL biweekly Receives: Procedure: Phlebotomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Remission
Time to a decrease in plasma porphyrin concentration to less than 0.9 mcg/dL
Measured: To end of study, an average of 3 years
Secondary outcomes (3)
50% Reduction in Plasma Porphyrin Level
50% reduction in plasma porphyrin level during treatment
Measured: To end of study, an average of 3 years
75% Reduction in Plasma Porphyrin Level
Time to 75% reduction in plasma porphyrin level during treatment
Measured: To end of study, an average of 3 years
Number of Days With Normal Urinary Porphyrin Levels
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.
Measured: To end of study, an average of 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2012 (estimated)
- Study start21 Mar 2006
- Primary completion6 Jul 2021
- Study completion6 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Porphyria Cutanea TardaStudies of the same condition, open and under way.
- More trials run by The University of Texas Medical Branch, GalvestonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Karl E Anderson, MD, Principal investigator, University of Texas
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01573754
- Sponsor's protocol ID
- FDA-2604
- Other IDs
- R01FD002604
- Status last verified
- Feb 2023
- First submitted
- 5 Apr 2012
- Last updated
- 17 Feb 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Through a NIH data repository at some future time.
Publications
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.
PubMed 22985607
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435
- ClinicalTrials.gov ID
- NCT01573754
- Acronym / sponsor ID
- FDA-2604
- Conditions
- Porphyria Cutanea Tarda
- Keywords
- Porphyria, rare disease, orphan disease, iron metabolism
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- The University of Texas Medical Branch, GalvestonUniversity, hospital or other
- Study start
- 21 Mar 2006 (actual)
- Primary completion
- 6 Jul 2021 (actual)
- Study completion
- 6 Jul 2021 (actual)
- First posted
- 9 Apr 2012 (estimated)
- Results
- Posted 17 Feb 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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):
- 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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hydroxychloroquine | Experimental | Drug: HydroxychloroquineLow-dose hydroxychloroquine 100 mg by mouth twice weekly |
| Phlebotomy | Active comparator | Procedure: PhlebotomyPhlebotomy 450 mL biweekly |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Documented porphyria cutanea tarda (PCT)
- Willing to give informed consent
- Age 18 or greater
Key exclusion criteria (1 listed)
- Blistering skin lesions due to another condition
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxychloroquineAlso: Plaquenil100 mg by mouth twice weekly | Drug | Hydroxychloroquine |
| Phlebotomy450 mL every 2 weeks | Procedure or surgery | Phlebotomy |
Primary outcome
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
3 secondary outcomes
50% Reduction in Plasma Porphyrin Level
Time frame: To end of study, an average of 3 years
75% Reduction in Plasma Porphyrin Level
Time frame: To end of study, an average of 3 years
Number of Days With Normal Urinary Porphyrin Levels
Time frame: To end of study, an average of 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 22985607Singal 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.
- Individual participant dataSharing planned: No. Through a NIH data repository at some future time.
Related trials for Porphyria Cutanea Tarda
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Karl E Anderson, MD principal investigator
University of Texas
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxychloroquine and Phlebotomy in people with Porphyria Cutanea Tarda, run by The University of Texas Medical Branch, Galveston at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Feb 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Remission (time frame: To end of study, an average of 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01573754, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2023.