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Post Hematopoietic Stem Cell Transplantation (MACS1553)

This study is not yet open for participant recruitment.
Verified February 2013 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01610297
First received: May 30, 2012
Last updated: February 21, 2013
Last verified: February 2013
History of Changes
  Purpose

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:

MedlinePlus related topics: Iron Thalassemia
Drug Information available for: Deferasirox
U.S. FDA Resources

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.


Estimated Enrollment: 30
Study Start Date: February 2013
Estimated Study Completion Date: February 2015
Estimated Primary Completion Date: February 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ICL670 Drug: ICL670

  Eligibility

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

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01610297

Contacts
Contact: Novartis Pharmaceuticals +41613241111
Contact: Novartis Pharmaceuticals

Locations
Turkey
Novartis Investigative Site Not yet recruiting
Ankara, Turkey, 06500
Novartis Investigative Site Not yet recruiting
Ankara, Turkey, 06100
Novartis Investigative Site Not yet recruiting
Antalya, Turkey, 07070
Novartis Investigative Site Not yet recruiting
Istanbul, Turkey, 34093
Novartis Investigative Site Not yet recruiting
Istanbul, Turkey
Novartis Investigative Site Not yet recruiting
Izmir, Turkey, 35040
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01610297     History of Changes
Other Study ID Numbers: CICL670ATR04
Study First Received: May 30, 2012
Last Updated: February 21, 2013
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:
Beta-Thalassemia
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
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 22, 2013