Porphyria Cutanea Tarda
Conditions
Keywords
Exjade PCT Study, PCT Study, Porphyria Cutanea Tarda
Brief 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.
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.
Interventions
DRUGDeferasirox
250 mg of deferasirox once daily for 6 months
Sponsors
Novartis Pharmaceuticals
University of Texas Southwestern Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Showing Reduction or Elimination of Skin Blistering | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level | 6 months | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Deferasirox Deferasirox : 250 mg of deferasirox once daily for 6 months with an increase to 500 mg/d after 2 months if new blisters continued to develop. | 10 |
| Total | 10 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Noncompliant | 1 |
Baseline characteristics
| Characteristic | Deferasirox |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants |
| Age, Continuous | 53.7 years STANDARD_DEVIATION 5.907622195 |
| Region of Enrollment United States | 10 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 1 / 10 |
| serious Total, serious adverse events | 0 / 10 |
Outcome results
Primary
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.
Time frame: Within 6 months of treatment.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Deferasirox | Number of Participants Showing Reduction or Elimination of Skin Blistering | Reduction | 8 participants |
| Deferasirox | Number of Participants Showing Reduction or Elimination of Skin Blistering | Elimination | 0 participants |
Secondary
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.
Time frame: 6 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Deferasirox | Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level | Decrease in ferritin level | 8 participants |
| Deferasirox | Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level | Decrease in urinary porphyrin level | 6 participants |