Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda
Status:
Unknown status
Trial end date:
2012-12-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborator:
Association pour l'Etude des Fonctions Digestives (AEFD)