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Post Hematopoietic Stem Cell Transplantation

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ClinicalTrials.gov Identifier: NCT01610297
Recruitment Status : Completed
First Posted : June 4, 2012
Results First Posted : July 21, 2016
Last Update Posted : October 24, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief Summary:
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.

Condition or disease Intervention/treatment Phase
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major Drug: ICL670 Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation.
Study Start Date : September 2013
Actual Primary Completion Date : October 2015
Actual Study Completion Date : October 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia
Drug Information available for: Deferasirox

Arms and Interventions
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Arm Intervention/treatment
Experimental: ICL670
Oral dose of ICL670 at 10 mg/kg daily
 Drug: ICL670
Oral dose of ICL670 at 10 mg/kg daily


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability [ Time Frame: 12 months ]
    To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period


Secondary Outcome Measures :
  1. Change in Serum Ferritin Level. [ Time Frame: Baseline, 12 Months ]
    Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months.

  2. Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination) [ Time Frame: Baseline, 12 month ]
    Liver Iron Concentration (LIC) values between 3 and 7 mg Fe / g dry weight are indicative of mild iron deposition, while values between 7 and 15 mg Fe / g dry weight are indicative of moderate iron deposition which have been associated with liver disease. Values >15 mg Fe/g dry weight are indicative of severe iron deposition which is associated with progressive liver fibrosis, increased morbidity and mortality

  3. The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L [ Time Frame: Week 28 and Week 52 ]
    Serum Ferritin values between 1000-2500 μg/L are indicative of mild to moderate iron overload while values >2500 μg/L are indicative of severe iron overload and levels constantly higher than 2500 μg/L has been shown to to increase the risk of cardiac complications and endocrine disease. Maintaining levels <1000 μg/L is associated with increased survival and less morbidity.

  4. Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination) [ Time Frame: Baseline, 12 month ]
    Cardiac MRI values between 10 to 20 milliseconds (ms) are indicative of moderate cardiac iron deposition associated with declining left ventricular ejection fraction and arrhythmias while values <10 ms are indicative of deposition sufficient to risk cardiac decompensation and associated with overt heart failure and mortality.


Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  • Patients who had hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major
  • hematopoietic stem cell transplantation (HSCT) was performed minimum 6 months and maximum 2 years ago
  • The washout period after the immunosuppressive therapy should be at least 3 months.
  • Signifacant IOL should be present including:

A. Serum ferritin >1000 μg/L or B. cardiac MRI <20 ms or C. liver iron concentration ≥ 5 mg/g dry weight measured by R2* MRI

Exclusion Criteria

  • Patients who had any contraindication for treatment with deferasirox according to the prescribing information

    •Patients who depended on transfusion

  • Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
  • Patients who were experiencing severe complication of HSCT e.g. acute Graft-versus host disease (GVHD)
  • Significant proteinuria / Increase in serum creatinine
Contacts and Locations
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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): NCT01610297


Locations
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Turkey
Novartis Investigative Site
Ankara, Turkey, 06100
Novartis Investigative Site
Ankara, Turkey, 06500
Novartis Investigative Site
Antalya, Turkey, 07000
Novartis Investigative Site
Antalya, Turkey, 07070
Novartis Investigative Site
Istanbul, Turkey
Novartis Investigative Site
Izmir, Turkey, 35040
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01610297    
Other Study ID Numbers: CICL670ATR04
First Posted: June 4, 2012    Key Record Dates
Results First Posted: July 21, 2016
Last Update Posted: October 24, 2016
Last Verified: September 2016
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
 Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action