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

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ClinicalTrials.gov Identifier: NCT01818726
Recruitment Status : Terminated (Failure to meet the schedule of patient recruitment)
First Posted : March 26, 2013
Results First Posted : August 16, 2019
Last Update Posted : August 16, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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.

Condition or disease Intervention/treatment Phase
Aplastic Anemia Drug: ICL670 Drug: Chelation Drug: No chelation Phase 4

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : June 23, 2014
Actual Primary Completion Date : October 17, 2016
Actual Study Completion Date : October 17, 2016


Arm Intervention/treatment
Experimental: 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
Drug: ICL670
ICL670 was supplied in registered packages as 250mg or 500mg dispersible tablets.
Other Name: Deferasirox

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).

Experimental: 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
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)




Primary Outcome Measures :
  1. Change in Serum Ferritin Values [ Time Frame: 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 ]

    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.


  2. Change in Transferrin Saturation With Iron (TSI) Values [ Time Frame: 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 ]
    Mean percentage change from baseline in transferrin saturation with iron was summarized descriptively for all on-treatment study visits.

  3. Change in Serum Total Iron-binding Capacity (TIBC) [ Time Frame: 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 ]
    Mean change from baseline in serum total iron-binding capacity was summarized descriptively for all on-treatment study visits.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01818726


Locations
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Russian Federation
Novartis Investigative Site
Moscow, Russian Federation, 125167
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01818726     History of Changes
Other Study ID Numbers: CICL670ARU02
First Posted: March 26, 2013    Key Record Dates
Results First Posted: August 16, 2019
Last Update Posted: August 16, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com


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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Aplastic anemia
deferasirox
ICL670
renal function
transfusion-dependent iron overload
immunosuppressive treatment
Cyclosporine A
unosuppressive treatment without chelation therapy
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Iron Overload
Hematologic Diseases
Bone Marrow Diseases
Iron Metabolism Disorders
Metabolic Diseases
Iron
Cyclosporine
Cyclosporins
Immunosuppressive Agents
Deferasirox
Trace Elements
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors
Iron Chelating Agents
Chelating Agents
Sequestering Agents