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

Aplastic Anemia Clinical Trial

Official title:

Open-label Study of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Undergoing Treatment Programs in Comparison With Control Group

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01818726
• Other study ID #: CICL670ARU02
• Status: Terminated
• Phase: Phase 4
• Start date: June 23, 2014
• Est. completion date: October 17, 2016

Study information

• Verified date: July 2019
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: October 17, 2016
• Est. primary completion date: October 17, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia
• Iron Overload

Intervention

Drug:
• ICL670
ICL670 was supplied in registered packages as 250mg or 500mg dispersible tablets.
• 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).
• No chelation
Comparative group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment ( immunosupressant -Cyclosporine A)

Locations

Country	Name	City	State
Russian Federation	Novartis Investigative Site	Moscow

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Serum Ferritin Values	Change from baseline was be summarized descriptively for all on-treatment study visits.; Changes to the planned statistical analysis were related to significant withdrawal of patients from the Per-Protocol Analysis Set due to a large number of patients who discontinued the study (lack of assessments of iron exchange parameters at visits) and deviations from the Protocol affecting the assessment of efficacy parameters. Because of that, the additional efficacy analysis in the Per-Protocol Analysis Set was not performed.	Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52
Primary	Change in Transferrin Saturation With Iron (TSI) Values	Mean percentage change from baseline in transferrin saturation with iron was summarized descriptively for all on-treatment study visits.	Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52
Primary	Change in Serum Total Iron-binding Capacity (TIBC)	Mean change from baseline in serum total iron-binding capacity was summarized descriptively for all on-treatment study visits.	Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52