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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01818726
First received: March 5, 2013
Last updated: April 20, 2017
Last verified: April 2017
  Purpose
To evaluate 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 Intervention Phase
Aplastic Anemia Drug: ICL670A and standard immunosupressive therapy (Cyclosporine A) Drug: Immunosupressive therapy (Cyclosporine A) Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: No masking
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

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

Primary Outcome Measures:
  • Change of serum ferritin, iron transferrin saturation, serum total iron-binding capacity (TIBC) [ Time Frame: Screening, 4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]
    Change from baseline will be summarized descriptively for all on-treatment study visits.


Secondary Outcome Measures:
  • Adverse events [ Time Frame: 52 weeks ]
    Number of participants with adverse events as a measure of safety and tolerability

  • Change of proteinuria in urinalysis over a period of 1 year treatment. [ Time Frame: 1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]
  • Change from baseline in creatinine clearance over a period of 1 year treatment. [ Time Frame: 1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]
  • Change from baseline of serum creatinine, bilirubin, ALT, AST, glucose over a period of 1 year treatment. [ Time Frame: 1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]

Enrollment: 14
Actual Study Start Date: June 23, 2014
Study Completion Date: October 17, 2016
Primary Completion Date: October 17, 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Conventional treatment arm
10 Adult (aged above 18) transfusion-dependent patients with AA and serum ferritin < 1000 mg/L undergoing treatment programs of immunosuppressive treatment (Cyclosporine A)
Drug: Immunosupressive therapy (Cyclosporine A)
Comparative group of patients with aplastic anemia and transfusion-dependent iron overload is undergoing treatment programs of standard immunosuppressive treatment ( immunosupressant -Cyclosporine A)
Experimental: Exjade treatment arm
15 transfusion-dependent adult (aged above 18) patients with AA and serum ferritin ≥ 1000 mg/L undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) and Exjade
Drug: ICL670A and standard immunosupressive therapy (Cyclosporine A)
In the investigational arm all patients recieve a standard immunosuppressant (Cyclosporine A). The starting dose of Exjade will be 20 mg/kg/day with up- and down-titration steps of 5-10 mg/kg/day if necessary, depending on serum ferritin, tolerability and comorbidities. Doses above 30 mg/kg are discouraged as such experience is limited in aplastic anemia. Patients are to take Exjade once daily at least 30 minutes before meals, on the same time each day. A combination of 250 mg and 500 mg tablets may be used to provide a specific dose level. The duration of treatment in this study is 12 months. The investigator muct emphasize complaince and will instruct the patients to take the Exjade exactly as prescribe.
Other Name: Deferasirox and standard immunosupressive therapy

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01818726

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

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01818726     History of Changes
Other Study ID Numbers: CICL670ARU02
Study First Received: March 5, 2013
Last Updated: April 20, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
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


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, renal function

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Iron Overload
Hematologic Diseases
Bone Marrow Diseases
Iron Metabolism Disorders
Metabolic Diseases
Cyclosporins
Cyclosporine
Immunosuppressive Agents
Deferasirox
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors
Iron Chelating Agents
Chelating Agents
Sequestering Agents

ClinicalTrials.gov processed this record on July 24, 2017