Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda

NCT01284946 | Unknown Phase 2 DMC

• 1 other identifier: AEFD2010-01
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Porphyria Cutanea Tarda

Keywords

PCT sporadic or familial; Skin fragility and bullae lesions; non-transfusion iron overload

Outcome Measures

Primary Outcomes (2)

• The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT.

  6 months

• Related drug adverse events

  Incidence type and severity of drug related adverse events

  6 months

Secondary Outcomes (2)

• 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

  6 months

• Chage from baseline in serum ferritin, iron burden, improvement in clincal symptoms, porphyrin levels

  6 months

Study Arms (1)

Exjade

EXPERIMENTAL

Safety and efficacy

Drug: Exjade

Interventions

Exjade DRUG

Orodispersible Tablet, 10 mg/Kg/day ± 5 mg/Kg/day during 24 weeks Deferasirox should be taken daily 30 minutes before breakfast

Also known as: DEFERASIROX

Exjade

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female diagnosed with clinically overt Porphyria Cutanea Tarda, sporadic or familial as per the European Porphyria Network guidelines i.e. increased urinary and plasma porphyrins and faecal isocoproporphyrin detected by fluorescence emission spectroscopy,
• Skin fragility and bullae lesions,
• Age ≥ 18 years old,
• non-transfusion iron overload as depicted by a serum ferritin value ≥ 300 μg/L for men and ≥ 200 μg/L for women, and/or LIC ≥ 2 mg Fe/g dw for both men and women and with transferrin saturation ≥ 45%,
• Adequate liver function i.e. ALAT/ASAT and Alkaline Phosphatase ? 2.5 times ULN, bilirubin \< 1.5 times ULN,
• Signed informed consent prior to beginning the specific procedures of the protocol,
• Ability to comply with all study-related procedures, medications, and evaluations,
• Sexually active women must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months). Since hormonal therapy may cause PCT, oral contraceptives will not be started during the course of the study and patients already on oral contraceptives will be advised to speak to their physician about discontinuing them and will not be enrolled in the study.

You may not qualify if:

• Clinical evidence of active Hepatitis B (positive HBsAg with negative HBsAb) and/or hepatitis C (positive HCV antibody and detectable HCV RNA with ALT above the normal range)
• Patients with on going alcoholic dependency \> 60g/day
• Serum creatinine above the ULN
• Creatinine clearance \< 60 ml/min, estimated according to Cockcroft-Gault formula or MDRD formula for adults
• Significant proteinuria as indicated by a urine protein: urine creatinine ratio \> 0.5 mg/mg in a non-first void urine sample.
• Diabetes
• Iron overload due to hereditary hemochromatosis
• History of blood transfusion during the 6 months prior to study entry,
• Males with hemoglobin \<13 mg/dL, females with hemoglobin \<12 mg/dL
• Active peptic ulcus
• Treatment with phlebotomy within 2 weeks of screening visit
• Prior Desferal® treatment within 1 month of the screening visit
• Patients currently or previously treated with deferiprone or deferasirox
• Patients with a diagnosis of a clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation
• Patient with clinically significant decrease of hearing

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Association pour l'Etude des Fonctions Digestives (AEFD): collaborator

Study Sites (1)

Hopital Louis Mourier, GI unit,

Colombes, Île-de-France Region, 92700, France

RECRUITING

Related Publications (1)

• Puy H, Gouya L, Deybach JC. Porphyrias. Lancet. 2010 Mar 13;375(9718):924-37. doi: 10.1016/S0140-6736(09)61925-5.

  PMID: 20226990 RESULT

MeSH Terms

Conditions

Porphyria Cutanea Tarda

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Porphyrias; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Deybach Jean-Charles, Professor

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Benoit Coffin, Professor

CONTACT

33147606061; benoit.coffin@lmr.aphp.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: January 26, 2011
• First Posted: January 27, 2011
• Study Start: January 1, 2011
• Primary Completion: December 1, 2012
• Study Completion: December 1, 2012
• Last Updated: January 27, 2011

Record last verified: 2011-01

Locations

FR (1)