Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda
Recruitment status was Recruiting
While clinical phlebotomy is current standard practice for alleviating non-transfusion iron overload in patients with PCT, it may not be suitable for all patients. For example, some patients are unwilling to be adequately phlebotomized because of inconvenience, as phlebotomy can be cumbersome, especially during the induction treatment phase requiring frequent clinic visits (twice a month, for at least 6 months) or because of venous access difficulties. Other patients are unable to undergo phlebotomy due to medical reasons such as anemia or cardiopulmonary disorders. It is postulated such patients with PCT who have non-transfusion iron overload could benefit from treatment with deferasirox (Exjade®), a once daily oral iron chelator licensed in several countries, including the EU, for treating transfusion iron overload in adult and pediatric patients. Although there is some data on the efficacy and safety of deferasirox in patients with HH, who, like those with PCT, have non-transfusional iron overload, there is a need to evaluate the safety and efficacy of deferasirox treatment of non-transfusion iron overload in patients with PCT.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II, Open Label Clinical Trial Exploring the Safety and the Efficacy of Oral Deferasirox in Patients Newly Diagnosed With Porphyria Cutanea Tarda (PCT) and Non-transfusion Iron Overload|
- The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
- Related drug adverse events [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]Incidence type and severity of drug related adverse events
- The change from baseline in serum ferritin after 12 and 24 weeks of treatment,The change from baseline in iron burden after 24 weeks of treatment measured by liver MRI T2,The evolution of clinical symptoms [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
- Chage from baseline in serum ferritin, iron burden, improvement in clincal symptoms, porphyrin levels [ Time Frame: 6 months ] [ Designated as safety issue: No ]
|Study Start Date:||January 2011|
|Estimated Study Completion Date:||December 2012|
|Estimated Primary Completion Date:||December 2012 (Final data collection date for primary outcome measure)|
Safety and efficacy
Orodispersible Tablet, 10 mg/Kg/day ± 5 mg/Kg/day during 24 weeks Deferasirox should be taken daily 30 minutes before breakfast
Other Name: DEFERASIROX
The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT.
The secondary objective is to assess the effectiveness of deferasirox treatment :
After 3 and 6 months to:
•Lower serum ferritin from abnormal to normal standard ranges specified for males and females in this patient population.
After 6 months to :
•Lower liver iron content after 24 weeks of treatment measured by liver MRI T2
After 3 and 6 months to :
- Improve clinical symptoms, i.e. improvement in skin lesions (reduction or no new bullae formation), and skin fragility (photographs will be used).
- Reduce porphyrin levels.
|Contact: Benoit Coffin, Professorfirstname.lastname@example.org|
|Hopital Louis Mourier, GI unit,||Recruiting|
|Colombes, Ile de France, France, 92700|
|Contact: Benoit Coffin, Professor 33147606061 email@example.com|
|Principal Investigator: Deybach Jean-Charles, Professor|
|Principal Investigator:||Deybach Jean-Charles, Professor||Assistance Publique - Hôpitaux de Paris|