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Clinical Trial 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.


Clinical Trial Description

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. ;


Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT01284946
Study type Interventional
Source Assistance Publique - Hôpitaux de Paris
Contact Benoit Coffin, Professor
Phone 33147606061
Email benoit.coffin@lmr.aphp.fr
Status Recruiting
Phase Phase 2
Start date January 2011
Completion date December 2012

See also
  Status Clinical Trial Phase
Completed NCT03118674 - Harvoni Treatment Porphyria Cutanea Tarda Phase 2
Completed NCT00005103 - Study of the Pathogenesis of Porphyria Cutanea Tarda N/A
Completed NCT00213772 - Risk Factors of Porphyria Cutanea Tarda (PCT) N/A
Completed NCT01573754 - Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda Phase 2
Completed NCT00599326 - Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda Phase 3