Post Hematopoietic Stem Cell Transplantation

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01610297
First received: May 30, 2012
Last updated: June 9, 2016
Last verified: June 2016
  Purpose
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 Intervention Phase
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Drug: ICL670
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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.

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    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:
  • Change in Serum Ferritin Level. [ Time Frame: Baseline, 12 Months ] [ Designated as safety issue: No ]
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the serum ferritin level.

  • Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination) [ Time Frame: Baseline, 12 month ] [ Designated as safety issue: No ]
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the R2*MRI (Liver MRI).

  • The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L [ Time Frame: Week 28 and Week 52 ] [ Designated as safety issue: No ]
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the serum ferritin level.

  • Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination) [ Time Frame: Baseline, 12 month ] [ Designated as safety issue: No ]
    Friedman test and Wilcoxon Signed Rank test were conducted to test whether there is a significant change in the T2*MRI (Cardiac MRI),


Enrollment: 27
Study Start Date: September 2013
Study Completion Date: October 2015
Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ICL670
Oral dose of ICL670 at 10 mg/kg daily
Drug: ICL670
Oral dose of ICL670 at 10 mg/kg daily

  Eligibility

Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
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
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: NCT01610297

Locations
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
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01610297     History of Changes
Other Study ID Numbers: CICL670ATR04 
Study First Received: May 30, 2012
Results First Received: April 21, 2016
Last Updated: June 9, 2016
Health Authority: Turkey: Ministry of Health

Keywords provided by Novartis:
hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload

Additional relevant MeSH terms:
Thalassemia
Iron Overload
Beta-Thalassemia
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

ClinicalTrials.gov processed this record on August 24, 2016