Back to resultsEfficacy and Safety of Oral Deferasirox (20 mg/kg/d) in Pts 3 to 6 Months After Allogeneic Hematopoietic Cell Transplantation Who Present With Iron Overload
Primary Purpose
Iron Overload
Status
Completed
Phase
Phase 4
Locations
Germany
Study Type
Interventional
Intervention
Deferasirox
Sponsored by
About this trial
This is an interventional treatment trial for Iron Overload focused on measuring Hematopoietic stem cell transplantation, HCT, iron overload, chelators, ICL670, deferasirox, serum ferritin, LIC, transfusional hemosiderosis, blood transfusions, adults, iron chelation, 3-6 months, allogeneic hematopoietic stem cell transplantation
Eligibility Criteria
Inclusion criteria:
- Transfusional iron overload three to six months after HCT with no evidence of active inflammation
- History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs).
- Patients of either gender and age ≥ 18 years.
- Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraception , or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
Exclusion criteria:
- Non-transfusion related iron overload
- Active malignancy
- Known active viral hepatitis or known HIV positiveness
- Mean levels of alanine aminotransferase (ALT) > 5x ULN
- Treatment with any iron chelator after transplantation
- Uncontrolled systemic hypertension
- Serum creatinine > 1.5 ULN and/or serum creatinine clearance < 60 ml/min
- History of nephrotic syndrome.
- Previous history of clinically relevant ocular or auditory toxicity related to iron chelation.
- Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
- Pregnant or breast feeding patients.
Other protocol-defined inclusion/exclusion criteria may apply
Sites / Locations
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
deferasirox
Arm Description
Outcomes
Primary Outcome Measures
To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox
Secondary Outcome Measures
To evaluate the relationship between serum ferritin, transferrin (TRF) and transferrin saturation during the whole study.
Full Information
NCT ID
NCT00654589
First Posted
April 2, 2008
Last Updated
November 16, 2016
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT00654589
Brief Title
Efficacy and Safety of Oral Deferasirox (20 mg/kg/d) in Pts 3 to 6 Months After Allogeneic Hematopoietic Cell Transplantation Who Present With Iron Overload
Official Title
A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/d) in Patients Three to Six Months After Allogeneic Hematopoietic Cell Transplantation in Whom Iron Overload is Present
Study Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
February 2008 (undefined)
Primary Completion Date
January 2012 (Actual)
Study Completion Date
January 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
5. Study Description
Brief Summary
The purpose of this study is to investigate the effects of iron chelation using deferasirox in patients who show signs of iron overload after an allogeneic stem cell transplantation The iron overload must be due to blood transfusions.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload
Keywords
Hematopoietic stem cell transplantation, HCT, iron overload, chelators, ICL670, deferasirox, serum ferritin, LIC, transfusional hemosiderosis, blood transfusions, adults, iron chelation, 3-6 months, allogeneic hematopoietic stem cell transplantation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
75 (Actual)
8. Arms, Groups, and Interventions
Arm Title
deferasirox
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Other Intervention Name(s)
ICL670
Primary Outcome Measure Information:
Title
To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
To evaluate the relationship between serum ferritin, transferrin (TRF) and transferrin saturation during the whole study.
Time Frame
52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria:
Transfusional iron overload three to six months after HCT with no evidence of active inflammation
History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs).
Patients of either gender and age ≥ 18 years.
Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraception , or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
Exclusion criteria:
Non-transfusion related iron overload
Active malignancy
Known active viral hepatitis or known HIV positiveness
Mean levels of alanine aminotransferase (ALT) > 5x ULN
Treatment with any iron chelator after transplantation
Uncontrolled systemic hypertension
Serum creatinine > 1.5 ULN and/or serum creatinine clearance < 60 ml/min
History of nephrotic syndrome.
Previous history of clinically relevant ocular or auditory toxicity related to iron chelation.
Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
Pregnant or breast feeding patients.
Other protocol-defined inclusion/exclusion criteria may apply
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmeceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Leipzig
Country
Germany
12. IPD Sharing Statement
Citations:
PubMed Identifier
26367238
Citation
Jaekel N, Lieder K, Albrecht S, Leismann O, Hubert K, Bug G, Kroger N, Platzbecker U, Stadler M, de Haas K, Altamura S, Muckenthaler MU, Niederwieser D, Al-Ali HK. Efficacy and safety of deferasirox in non-thalassemic patients with elevated ferritin levels after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2016 Jan;51(1):89-95. doi: 10.1038/bmt.2015.204. Epub 2015 Sep 14.
Results Reference
result
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Efficacy and Safety of Oral Deferasirox (20 mg/kg/d) in Pts 3 to 6 Months After Allogeneic Hematopoietic Cell Transplantation Who Present With Iron Overload
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