Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients

Novartis

Status and phase: Terminated; Phase 4

Conditions: Aplastic Anemia

Treatments: Drug: Chelation; Drug: ICL670; Drug: No chelation

Study type: Interventional

Funder types: Industry

Identifiers

NCT01818726

CICL670ARU02

Details and patient eligibility

About

Evaluated Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) without chelation therapy.

Full description

The secondary endpoints that were originally planned for this study were not analyzed as the study ended prematurely.

Enrollment: 15 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Main diagnosis: aplastic anemia
• Absence of severe and/or uncontrolled comorbidities
• Confirmed iron overload (serum ferritin ≥ 1000 mkg/L)
• Serum creatinine is not higher than the upper limit of normal for the given age
• Absence of severe proteinuria. Protein/Creatinine ratio should be < 0.5 mg/mg
• Liver enzymes are < 5 ULN
• Completion of a scheduled cycle of immunosuppressive treatment program, with no severe infectious or generalized hemorrhagic complications
• WHO (ECOG) performance status ≤ 2

Exclusion criteria

• No signed informed consent form
• Patient is under 18 years old
• Severe concomitant condition
• Severe infectious and generalized haemorrhagic complication following regular planned cycle of programmed immune suppressive treatment.
• History of increased sensitivity to active substance and any other ingredient of the medicinal product.
• Creatinine clearance (CC) < 60 ml/min and/or creatinine concentration in blood serum is 2 or more times higher than upper limit of age normal by results of 2 tests at Visits 1 and 2.
• Severe liver disorders (class C by Child-Pugh scale).
• Patients with aplastic anaemia in which chelator treatment will be ineffective due to rapid progression of the disease.
• Significant proteinuria basing on protein creatinine ratio > 1.0 mg/ml in urine sample from second urination at Visits 1 and 2 (or as an alternative in 2 of 3 urine samples at screening);
• Rare hereditary disorders related to galactose intolerance, severe deficit of lactase or glucose-galactose malabsorption;
• Pregnancy, lactation;
• Level of liver enzymes higher than 5 upper limits of age normal at Visits 1 and 2.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

15 participants in 2 patient groups

Serum ferritin level ≥ 1,000 μg/l

Experimental group

Description:

Transfusion-dependent adult patients with AA and serum ferritin ≥ 1000 mg/L on programmed immune suppressive treatment with cyclosporine A who were receiving chelation with Exjade (deferasirox) during the study

Treatment:

Drug: Chelation

Drug: ICL670

Serum ferritin level < 1,000 μg/l

Experimental group

Description:

Transfusion-dependent adult patients with AA and serum ferritin \< 1,000 mg/L on programmed immune suppressive treatment with cyclosporine A who were not receiving the investigational product

Treatment:

Drug: No chelation