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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01284946
• Date of registration: 26/01/2011
• Prospective Registration: No
• Primary sponsor: Assistance Publique - Hôpitaux de Paris
• Public title: Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda
• Scientific 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
• Date of first enrolment: January 2011
• Target sample size: 45
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT01284946
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

France

Contacts

• Name: Benoit Coffin, Professor

• Name: Benoit Coffin, Professor

• Name: Deybach Jean-Charles, Professor
• Affiliation: Assistance Publique - Hôpitaux de Paris

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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

- Pregnant or lactating women or women of childbearing potential not using
adequate contraception (pregnancy test mandatory and negative for patient with
childbearing potential)

- Known hypersensitivity to the active ingredient of deferasirox or any excipients

- Contraindication to the administration of deferasirox as outlined in the
approved prescribing information.

- Presence of a surgical or medical condition that might significantly alter the
absorption, distribution, metabolism or excretion of deferasirox

- Presence of a non-controlled severe disease affected vital organs as cardiac
and/or pulmonary disease

- Patients with a known diagnosis of cirrhosis (confirmed by biopsy)

- Patients with active inflammatory diseases that may interfere with the accurate
measurement of serum ferritin

- Patients treated with systemic investigational drug within 4 weeks prior or with
topical investigational drug within 7 days prior to the screening visit



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Porphyria Cutanea Tarda

Intervention(s)

Drug: Exjade

Primary Outcome(s)

The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT. [Time Frame: 6 months]

Related drug adverse events [Time Frame: 6 months]

Secondary Outcome(s)

Chage from baseline in serum ferritin, iron burden, improvement in clincal symptoms, porphyrin levels [Time Frame: 6 months]

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]

Secondary ID(s)

AEFD2010-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Association pour l'Etude des Fonctions Digestives (AEFD)