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Post Hematopoietic Stem Cell Transplantation

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ClinicalTrials.gov Identifier: NCT01610297
Recruitment Status : Completed
First Posted : June 4, 2012
Results First Posted : July 21, 2016
Last Update Posted : October 24, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Intervention Drug: ICL670
Enrollment 27
Recruitment Details  
Pre-assignment Details  
Arm/Group Title ICL670
Hide Arm/Group Description Oral dose of ICL670 at 10 mg/kg daily
Period Title: Overall Study
Started 27
Completed 26
Not Completed 1
Reason Not Completed
Lost to Follow-up             1
Arm/Group Title ICL670
Hide Arm/Group Description Oral dose of ICL670 at 10 mg/kg daily
Overall Number of Baseline Participants 27
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 27 participants
9.07  (3.81)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
Female
8
  29.6%
Male
19
  70.4%
1.Primary Outcome
Title Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set (SS) includes all included patients who were included in the study. All statistical analyses of safety and tolerability will be done in the SS.
Arm/Group Title ICL670
Hide Arm/Group Description:
Oral dose of ICL670 at 10 mg/kg daily
Overall Number of Participants Analyzed 27
Measure Type: Number
Unit of Measure: Participants
Adverse events 25
Serious adverse events 3
Death 0
2.Secondary Outcome
Title Change in Serum Ferritin Level.
Hide Description Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months.
Time Frame Baseline, 12 Months
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
Arm/Group Title ICL670
Hide Arm/Group Description:
Oral dose of ICL670 at 10 mg/kg daily
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: ng/mL
Baseline 1766.81  (599.64)
Month 12 903.56  (596.62)
3.Secondary Outcome
Title Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
Arm/Group Title ICL670
Hide Arm/Group Description:
Oral dose of ICL670 at 10 mg/kg daily
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: mg Fe/g dry liver weight
Baseline Liver MRI(n= 27) 12.07  (9.42)
Week 52 Liver MRI (n=25) 4.62  (2.85)
4.Secondary Outcome
Title The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
Arm/Group Title ICL670
Hide Arm/Group Description:
Oral dose of ICL670 at 10 mg/kg daily
Overall Number of Participants Analyzed 27
Measure Type: Number
Unit of Measure: Percentage of Patients
Week 28 (n=26) 7.7
Week 52 (n=27) 33.3
5.Secondary Outcome
Title Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS) comprises all patients in whom study treatment has been started and received at least one dose.
Arm/Group Title ICL670
Hide Arm/Group Description:
Oral dose of ICL670 at 10 mg/kg daily
Overall Number of Participants Analyzed 27
Mean (Standard Deviation)
Unit of Measure: ms
Baseline Cardiac MRI(n= 27) 26.48  (7.49)
Week 52 Cardiac MRI (n=24) 28.25  (5.53)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title ICL670
Hide Arm/Group Description Oral dose of ICL670 at 10 mg/kg daily
All-Cause Mortality
ICL670
Affected / at Risk (%)
Total   --/-- 
Hide Serious Adverse Events
ICL670
Affected / at Risk (%)
Total   3/27 (11.11%) 
General disorders   
Influenza like illness  1  1/27 (3.70%) 
Hepatobiliary disorders   
Hepatitis B  1  1/27 (3.70%) 
Investigations   
Neutrophil count decreased  1  2/27 (7.41%) 
Alanine aminotransferase increased  1  1/27 (3.70%) 
Aspartate aminotransferase increased  1  1/27 (3.70%) 
Surgical and medical procedures   
Office visit  1  1/27 (3.70%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 18.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
ICL670
Affected / at Risk (%)
Total   23/27 (85.19%) 
Blood and lymphatic system disorders   
Anemia  1  7/27 (25.93%) 
Gastrointestinal disorders   
Diarrhea  1  3/27 (11.11%) 
Vomiting  1  3/27 (11.11%) 
General disorders   
Pyrexia  1  7/27 (25.93%) 
Influenza like illness  1  2/27 (7.41%) 
Immune system disorders   
Hypersensitivity  1  2/27 (7.41%) 
Infections and infestations   
Pharyngitis  1  6/27 (22.22%) 
Haemophilus infection  1  2/27 (7.41%) 
Herpes zoster  1  2/27 (7.41%) 
Infection  1  2/27 (7.41%) 
Sinusitis  1  2/27 (7.41%) 
Investigations   
Aspartate aminotransferase increased  1  6/27 (22.22%) 
White blood cell count decreased  1  2/27 (7.41%) 
Alanine aminotransferase increased  1  7/27 (25.93%) 
Respiratory, thoracic and mediastinal disorders   
Cough  2  7/27 (25.93%) 
Influenza  1  5/27 (18.52%) 
Upper respiratory tract infection  1  2/27 (7.41%) 
Rhinorrhea  1  2/27 (7.41%) 
Skin and subcutaneous tissue disorders   
Eczema  1  2/27 (7.41%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 18.0
2
Term from vocabulary, MedDRA 18.2
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis
Phone: 862-778-8300
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01610297    
Other Study ID Numbers: CICL670ATR04
First Submitted: May 30, 2012
First Posted: June 4, 2012
Results First Submitted: April 21, 2016
Results First Posted: July 21, 2016
Last Update Posted: October 24, 2016