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Post Hematopoietic Stem Cell Transplantation

Primary Purpose

Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major

Status
Completed
Phase
Phase 4
Locations
Turkey
Study Type
Interventional
Intervention
ICL670
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major focused on measuring hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ICL670

Arm Description

Oral dose of ICL670 at 10 mg/kg daily

Outcomes

Primary Outcome Measures

Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
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.
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)
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
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)
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.

Full Information

First Posted
May 30, 2012
Last Updated
September 12, 2016
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01610297
Brief Title
Post Hematopoietic Stem Cell Transplantation
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 Type
Interventional

2. Study Status

Record Verification Date
September 2016
Overall Recruitment Status
Completed
Study Start Date
September 2013 (undefined)
Primary Completion Date
October 2015 (Actual)
Study Completion Date
October 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Keywords
hematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overload

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ICL670
Arm Type
Experimental
Arm Description
Oral dose of ICL670 at 10 mg/kg daily
Intervention Type
Drug
Intervention Name(s)
ICL670
Intervention Description
Oral dose of ICL670 at 10 mg/kg daily
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
Description
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
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Change in Serum Ferritin Level.
Description
Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months.
Time Frame
Baseline, 12 Months
Title
Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Description
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
Time Frame
Baseline, 12 month
Title
The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L
Description
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.
Time Frame
Week 28 and Week 52
Title
Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Description
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.
Time Frame
Baseline, 12 month

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Facility Name
Novartis Investigative Site
City
Ankara
ZIP/Postal Code
06500
Country
Turkey
Facility Name
Novartis Investigative Site
City
Antalya
ZIP/Postal Code
07000
Country
Turkey
Facility Name
Novartis Investigative Site
City
Antalya
ZIP/Postal Code
07070
Country
Turkey
Facility Name
Novartis Investigative Site
City
Istanbul
Country
Turkey
Facility Name
Novartis Investigative Site
City
Izmir
ZIP/Postal Code
35040
Country
Turkey

12. IPD Sharing Statement

Citations:
PubMed Identifier
29155313
Citation
Yesilipek MA, Karasu G, Kaya Z, Kuskonmaz BB, Uygun V, Dag I, Ozudogru O, Ertem M. A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with beta-Thalassemia Major. Biol Blood Marrow Transplant. 2018 Mar;24(3):613-618. doi: 10.1016/j.bbmt.2017.11.006. Epub 2017 Nov 16.
Results Reference
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Post Hematopoietic Stem Cell Transplantation

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