Brief Title
Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients
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
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.
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Change in Serum Ferritin Values
Condition
Aplastic Anemia
Intervention
ICL670
Study Arms / Comparison Groups
Serum ferritin level ≥ 1,000 μg/l
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
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
15
Start Date
June 23, 2014
Completion Date
October 17, 2016
Primary Completion Date
October 17, 2016
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.
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Novartis Pharmaceuticals, ,
Location Countries
Russian Federation
Location Countries
Russian Federation
Administrative Informations
NCT ID
NCT01818726
Organization ID
CICL670ARU02
Responsible Party
Sponsor
Study Sponsor
Novartis Pharmaceuticals
Study Sponsor
Novartis Pharmaceuticals, Study Director, Novartis Pharmaceuticals
Verification Date
July 2019