Porphyria Cutanea TardaPhase 3NCT00599326
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
A Phase 3 study testing Deferasirox in people with Porphyria Cutanea Tarda, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
To determine the efficacy and tolerability of deferasirox in the treatment of Porphyria Cutanea Tarda. Primary objective - the elimination of all blistering within 6 months of treatment. Secondary objective - decrease in total body iron levels. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Porphyria Cutanea Tarda
- What is being tested?
- Deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2008to Apr 2010
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosis of Porphyria Cutanea Tarda based on clinical exam and 24-hour urine porphyrin levels
- have Porphyria Cutanea Tarda for at least 3 months prior to enrollment with active blistering (3 blisters or erosions per month)
- women of childbearing potential must use an effective method of contraception during the study, however this cannot include hormonal contraception (oral contraceptives, hormone patches, Depo-Provera injections, NUVA Ring, etc.)
- treatment naive patients or patients unresponsive or intolerant of phlebotomy
- Ferritin level is greater than or equal to 25ng/mL
Who cannot join
- patients with serum creatinine above the upper limit of normal
- patients receiving phlebotomy who are controlled on this therapy
- pregnant or breast feeding females
- patients with liver transaminases more than 5 times the upper limit of normal
- patients with a history of hypersensitivity to deferasirox
- patients with a history of pre-existing renal condition, or receiving medication that depresses renal function
- patients on other chelators
- history of non-compliance to medical regimens.
Trial locations and contacts
1 site in 1 country.
United States1 site
UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
Dallas, Texas, 75390-8802
About this study
To determine the efficacy and tolerability of deferasirox in the treatment of Porphyria Cutanea Tarda.
Primary objective - the elimination of all blistering within 6 months of treatment.
Secondary objective - decrease in total body iron levels.
Read the detailed description
Phlebotomy is the standard therapy for Porphyria Cutanea Tarda (PCT), but it can be inconvenient and cause anemia in some patients.
Deferasirox is a new class of tridentate iron chelators with high affinity and selectivity for iron. The medication is administered orally, which if effective for PCT would make it a more convenient and possibly more tolerable option for patients.
- Conditions
- Porphyria Cutanea Tarda
- Being tested
- Deferasirox
- Keywords
- Exjade PCT Study, PCT Study, Porphyria Cutanea Tarda
- Medical subject headings
- Porphyria Cutanea Tarda
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
Also called Exjade
250 mg of deferasirox once daily for 6 months
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Showing Reduction or Elimination of Skin Blistering
The present trial was undertaken to determine if oral deferasirox could be useful in the treatment of PCT. Monthly clinic visits with a physical examination was conducted to assess the skin for blisters.
Measured: Within 6 months of treatment.
Secondary outcomes (1)
Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level
Patients with PCT usually have normal or elevated serum iron and ferritin levels as well as increased iron absorption. Phlebotomy is conducted to analyzes the ferritin levels. Urine collection is performed and samples of the urine are analyzed for porphyrin levels.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2008 (estimated)
- Study startJan 2008
- Primary completionApr 2010
- Study completionApr 2010
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 University of Texas Southwestern Medical CenterThe 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Responsible party
- Principal investigator, Amit Pandya, University of Texas Southwestern Medical Center
- Investigators
- Amit Pandya, M.D., Principal investigator, UT Southwestern Medical Center at Dallas - Department of Dermatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00599326
- Sponsor's protocol ID
- CICL670A US17
- Other IDs
- IRB File Number 062007-047
- Status last verified
- Jan 2014
- First submitted
- 10 Jan 2008
- Last updated
- 10 Feb 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Pandya AG, Nezafati KA, Ashe-Randolph M, Yalamanchili R. Deferasirox for porphyria cutanea tarda: a pilot study. Arch Dermatol. 2012 Aug;148(8):898-901. doi: 10.1001/archdermatol.2012.807.
PubMed 22911183
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
- ClinicalTrials.gov ID
- NCT00599326
- Acronym / sponsor ID
- CICL670A US17
- Conditions
- Porphyria Cutanea Tarda
- Keywords
- Exjade PCT Study, PCT Study, Porphyria Cutanea Tarda
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Study start
- Jan 2008
- Primary completion
- Apr 2010 (actual)
- Study completion
- Apr 2010 (actual)
- First posted
- 23 Jan 2008 (estimated)
- Results
- Posted 10 Feb 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To determine the efficacy and tolerability of deferasirox in the treatment of Porphyria Cutanea Tarda.
Primary objective - the elimination of all blistering within 6 months of treatment.
Secondary objective - decrease in total body iron levels.
Detailed description
Phlebotomy is the standard therapy for Porphyria Cutanea Tarda (PCT), but it can be inconvenient and cause anemia in some patients.
Deferasirox is a new class of tridentate iron chelators with high affinity and selectivity for iron. The medication is administered orally, which if effective for PCT would make it a more convenient and possibly more tolerable option for patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: Deferasirox |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- diagnosis of Porphyria Cutanea Tarda based on clinical exam and 24-hour urine porphyrin levels
- have Porphyria Cutanea Tarda for at least 3 months prior to enrollment with active blistering (3 blisters or erosions per month)
- women of childbearing potential must use an effective method of contraception during the study, however this cannot include hormonal contraception (oral contraceptives, hormone patches, Depo-Provera injections, NUVA Ring, etc.)
- treatment naive patients or patients unresponsive or intolerant of phlebotomy
- Ferritin level is greater than or equal to 25ng/mL
Key exclusion criteria (8 listed)
- patients with serum creatinine above the upper limit of normal
- patients receiving phlebotomy who are controlled on this therapy
- pregnant or breast feeding females
- patients with liver transaminases more than 5 times the upper limit of normal
- patients with a history of hypersensitivity to deferasirox
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 |
|---|---|---|
| DeferasiroxAlso: Exjade250 mg of deferasirox once daily for 6 months | Drug | A |
Primary outcome
Number of Participants Showing Reduction or Elimination of Skin Blistering
Time frame: Within 6 months of treatment.
The present trial was undertaken to determine if oral deferasirox could be useful in the treatment of PCT. Monthly clinic visits with a physical examination was conducted to assess the skin for blisters.
1 secondary outcome
Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 22911183Pandya AG, Nezafati KA, Ashe-Randolph M, Yalamanchili R. Deferasirox for porphyria cutanea tarda: a pilot study. Arch Dermatol. 2012 Aug;148(8):898-901. doi: 10.1001/archdermatol.2012.807.
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.
Amit Pandya, M.D. principal investigator
UT Southwestern Medical Center at Dallas - Department of Dermatology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Deferasirox in people with Porphyria Cutanea Tarda, run by University of Texas Southwestern Medical Center 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 5 inclusion and 8 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 10 Feb 2014. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 10 Feb 2014). 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?
Number of Participants Showing Reduction or Elimination of Skin Blistering (time frame: Within 6 months of treatment.). 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 NCT00599326, 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 10 Feb 2014.