Post Hematopoietic Stem Cell Transplantation

This study has been completed.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: May 30, 2012
Last updated: November 4, 2015
Last verified: November 2015
This is a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study is 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: Allocation: Non-Randomized
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:
  • •Safety of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major [ 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 HSCT in patients with beta-thalassemia major in 12 months period

Secondary Outcome Measures:
  • • Efficacy of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major by 12th month. [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    • To evaluate change the serum ferritin level and change from baseline to 12th month • To evaluate change in the further parameters of iron overload (cardiac iron and liver iron concentration by MR examination) from baseline to 12th month • To determine the percentage of patients reaching serum ferritin levels lower than 500 µg/L at week 28 and at week 52.

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 Drug: ICL670


Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria

  • Patients who had HSCT for beta-thalassemia major
  • 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 have any contraindication for treatment with deferasirox according to the prescribing information

    •Patients who depend 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 are experiencing severe complication of HSCT (e.g. acute 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 identifier: NCT01610297

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
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals ) Identifier: NCT01610297     History of Changes
Other Study ID Numbers: CICL670ATR04
Study First Received: May 30, 2012
Last Updated: November 4, 2015
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Iron Overload
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Iron Metabolism Disorders
Metabolic Diseases processed this record on November 25, 2015