Back to resultsA Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Primary Purpose
Beta-Thalassemia, Hemosiderosis
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
deferasirox
Sponsored by
About this trial
This is an interventional treatment trial for Beta-Thalassemia focused on measuring Thalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiprone
Eligibility Criteria
Inclusion Criteria: Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone Exclusion Criteria: Means levels of ALT > 300 U/L Serum creatinine above upper limit of normal Active hepatitis C or chronic hepatitis B receiving specific treatment
Sites / Locations
- Novartis Investigative Site
- Ali Taher
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Deferasirox
Arm Description
Outcomes
Primary Outcome Measures
Liver Iron Concentration (as measured by biopsy)
Secondary Outcome Measures
Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)
Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation
Adverse events
hematology and biochemistry, urinalysis
ECG and Echocardiography
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00171171
Brief Title
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Official Title
A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Study Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
May 2004 (undefined)
Primary Completion Date
November 2006 (Actual)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
Novartis
4. Oversight
5. Study Description
Brief Summary
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-Thalassemia, Hemosiderosis
Keywords
Thalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiprone
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
252 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Deferasirox
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
deferasirox
Primary Outcome Measure Information:
Title
Liver Iron Concentration (as measured by biopsy)
Time Frame
at baseline and after 1 yeor of ICL670 treatment
Secondary Outcome Measure Information:
Title
Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)
Time Frame
after 1 year of ICL670 treatment
Title
Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation
Time Frame
monthly assessments for 1 year
Title
Adverse events
Time Frame
during 1 year ICL670 treatment
Title
hematology and biochemistry, urinalysis
Time Frame
monthly assessments for 1 year ICL670 treatment
Title
ECG and Echocardiography
Time Frame
6-monthly for 1 year ICL670 treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone
Exclusion Criteria:
Means levels of ALT > 300 U/L
Serum creatinine above upper limit of normal
Active hepatitis C or chronic hepatitis B receiving specific treatment
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ali Taher, Ass.Prof.
Organizational Affiliation
American University of Beirut Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Novartis Investigative Site
City
Cairo
Country
Egypt
Facility Name
Ali Taher
City
Beirut
ZIP/Postal Code
Riad El Solh 1107 2020
Country
Lebanon
Facility Name
Novartis Investigative Site
City
Muscat
Country
Oman
Facility Name
Novartis Investigative Site
City
Riyadh
Country
Saudi Arabia
Facility Name
Novartis Investigative Site
City
Damascus
Country
Syrian Arab Republic
12. IPD Sharing Statement
Citations:
PubMed Identifier
20424435
Citation
Taher A, Al Jefri A, Elalfy MS, Al Zir K, Daar S, Rofail D, Baladi JF, Habr D, Kriemler-Krahn U, El-Beshlawy A. Improved treatment satisfaction and convenience with deferasirox in iron-overloaded patients with beta-Thalassemia: Results from the ESCALATOR Trial. Acta Haematol. 2010;123(4):220-5. doi: 10.1159/000313447. Epub 2010 Apr 27.
Results Reference
result
Links:
URL
https://www.ncbi.nlm.nih.gov/pubmed/20424435
Description
Results for CICL670A2402 from the Novartis Clinical Trials website
Learn more about this trial
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
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