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
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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 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 |

Arm | Intervention/treatment |
---|---|
Experimental: ICL670
Oral dose of ICL670 at 10 mg/kg daily
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Drug: ICL670
Oral dose of ICL670 at 10 mg/kg daily |
- 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
- 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.
- 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
- 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.
- 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.

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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 |
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
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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

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
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 |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
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 |
hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload |
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 |