Clinical trial NCT01818726: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Aplastic Anemia
Keywords: Aplastic anemia
deferasirox
ICL670
renal function
transfusion-dependent iron overload
immunosuppressive treatment
Cyclosporine A
unosuppressive treatment without chelation therapy
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-03-26
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Change in Serum Ferritin Values
timeFrame: 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
measure: Change in Transferrin Saturation With Iron (TSI) Values
timeFrame: 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
measure: Change in Serum Total Iron-binding Capacity (TIBC)
timeFrame: 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
Secondary outcomes:
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: