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

NCT ID: NCT01818726

Last Updated: 2019-08-16

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-06-23
• Study Completion Date: 2016-10-17

Brief Summary

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.

Detailed Description

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

Conditions

Aplastic Anemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Serum ferritin level ≥ 1,000 μg/l

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

Group Type: EXPERIMENTAL

ICL670

Intervention Type: DRUG

ICL670 was supplied in registered packages as 250mg or 500mg dispersible tablets.

Chelation

Intervention Type: DRUG

Main group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment (immunosupressant - Cyclosporine A) and received chelation with ICL670 (deferasirox).

Serum ferritin level < 1,000 μg/l

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

Group Type: EXPERIMENTAL

No chelation

Intervention Type: DRUG

Comparative group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment ( immunosupressant -Cyclosporine A)

Interventions

ICL670

ICL670 was supplied in registered packages as 250mg or 500mg dispersible tablets.

Intervention Type: DRUG

Chelation

Main group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment (immunosupressant - Cyclosporine A) and received chelation with ICL670 (deferasirox).

Intervention Type: DRUG

No chelation

Comparative group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment ( immunosupressant -Cyclosporine A)

Intervention Type: DRUG

Other Intervention Names

Deferasirox

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Moscow, , Russia

Countries

Russia

Other Identifiers

CICL670ARU02

Identifier Type: -

Identifier Source: org_study_id