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CTRI Number  CTRI/2017/10/010252 [Registered on: 30/10/2017] Trial Registered Prospectively
Last Modified On: 27/08/2024
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Randomized, Parallel Group Trial 
Public Title of Study   Clinical trial to Evaluate Treatment Compliance, Efficacy and Safety of an Improved new formulation of Deferasirox in the form of Granules in children of the age 2 to 18 Years Old who have Iron overload. 
Scientific Title of Study   randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance, efficacy and safety of an improved deferasirox formulation (granules) in pediatric patients with iron overload 
Trial Acronym   
Secondary IDs if Any
Modification(s)  
Secondary ID  Identifier 
CICL670F2202 version 3  Protocol Number 
CICL670F2202 version 4  Protocol Number 
CICL670F2202 Version 5  Protocol Number 
NCT02435212  ClinicalTrials.gov 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
Modification(s)  
Name  Dr Manish Mistry 
Designation  Medical Director Oncology 
Affiliation  Novartis India Limited 
Address  Novartis Healthcare Pvt Ltd GDO Trial Monitoring, India 6 & 7 floor , Inspire BKC G Block, BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai Mumbai

Mumbai
MAHARASHTRA
400051
India 
Phone    
Fax    
Email  manish.mistry@novartis.com  
 
Details of Contact Person
Scientific Query
Modification(s)  
Name  Dr Manish Mistry 
Designation  Medical Director Oncology 
Affiliation  Novartis India Limited 
Address  Novartis Healthcare Pvt Ltd GDO Trial Monitoring, India 6 & 7 floor , Inspire BKC G Block, BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai Mumbai

Mumbai
MAHARASHTRA
400051
India 
Phone    
Fax    
Email  manish.mistry@novartis.com  
 
Details of Contact Person
Public Query
Modification(s)  
Name  Dr Manish Mistry 
Designation  Medical Director Oncology 
Affiliation  Novartis India Limited 
Address  Novartis Healthcare Pvt Ltd GDO Trial Monitoring, India 6 & 7 floor , Inspire BKC G Block, BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai

Mumbai
MAHARASHTRA
400051
India 
Phone    
Fax    
Email  manish.mistry@novartis.com  
 
Source of Monetary or Material Support
Modification(s)  
Novartis Healthcare Pvt Ltd GDO Trial Monitoring, India 6 & 7 floor , Inspire BKC G Block, BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai Mumbai MAHARASHTRA 400051 India 
 
Primary Sponsor
Modification(s)  
Name  Novartis Healthcare Private Limited 
Address  Novartis Healthcare Pvt Ltd GDO Trial Monitoring, India 6 & 7 floor , Inspire BKC G Block, BKC Main Road Bandra Kurla Complex Bandra (East) Mumbai Mumbai MAHARASHTRA 400051 India 
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     Australia
Belgium
Bulgaria
China
Colombia
Denmark
Egypt
France
Hungary
India
Italy
Lebanon
Malaysia
Mexico
Oman
Panama
Philippines
Russian Federation
Spain
Sri Lanka
Taiwan
Thailand
Tunisia
Turkey
United Arab Emirates
United States of America  
Sites of Study  
No of Sites = 2  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Apurba Ghosh  Institute of Child Health  Institute of Child Health (ICH), 11, Dr Biresh Guha Street, Kolkata – 700017, West Bengal, India
Kolkata
WEST BENGAL 		 09007716516

apurbaghosh@yahoo.com 
Dr Prantar Chakrabarti  Nil Ratan Sircar Medical College anmd Hospital  Department of Hematology, Nil Ratan Sircar Medical College and Hospital, 138-AJC Road, Kolkata - 700014
Kolkata
WEST BENGAL 		 9433018899

prantar@gmail.com 
 
Details of Ethics Committee  
No of Ethics Committees= 1  
Name of Committee  Approval Status 
Institutional Ethics Committee - Institute of Child Health  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  Patient with transfusion-dependent anemia associated with iron overload requiring iron chelation therapy and with a history of transfusion and a treatment goal to reduce iron burden,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Comparator Agent  Deferasirox DT  Patients will be randomized to either Deferasirox granules or Deferasirox DT (Exjade) in the core phase. All patients who choose to continue in the extension phase will receive deferasirox granules in the extension phase. 
Intervention  Deferasirox granules   Patients will be randomized to either Deferasirox granules or Deferasirox DT (Exjade) in the core phase. All patients who choose to continue in the extension phase will receive deferasirox granules in the extension phase. 
 
Inclusion Criteria  
Age From  2.00 Year(s)
Age To  18.00 Year(s)
Gender  Both 
Details  1. Written informed consent/assent before any study-specific procedures.
2. Consent will be obtained from parent(s) or legal guardians. Investigators will also
obtain assent of patients according to local guidelines.
3. Male and female children and adolescents aged between 2 and 18 years.
Applicable to core phase only. Once in the core phase patients can turn 18 years
and still be considered eligible, also for participation in the optional extension
phase.
4. Any transfusion-dependent anemia associated with iron overload requiring iron
chelation therapy and with a history of transfusion of approximately 20 PRBC units,
and a treatment goal of reduction, not maintenance of iron burden as measured by
serum ferritin.
5. Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit
2 (the mean value will be used for eligibility criteria).
6. Completion of core phase per protocol (For the optional extension phase
criteria only).
7. Meeting inclusion criteria as defined in the core phase at Visit 17 except for age
requirement (For the optional extension phase criteria only). 
 
ExclusionCriteria 
Details  1. Creatinine clearance below the contraindication limit in the locally approved
prescribing informationCreatinine clearance will be estimated from serum creatinine (using the Schwartz formula) at screening Visit 1 or screening Visit 2.
2. Serum creatinine > 1.5 xULN at screening Visit 1 or screening Visit 2.
3. ALT or AST greater than 3.0 x ULN at screening visit 1 or screening visit 2.
4. Liver disease with severity of Child-Pugh class B or C.
5. Significant proteinuria as indicated by a urinary protein/creatinine ratio >0.5 mg/mg in a second morning urine sample at screening Visit 1 or screening Visit 2.
6. Patients with significant impaired gastrointestinal (GI) function or GI disease
that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
7. Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive).
8. Patients with psychiatric or addictive disorders which prevent them from giving
their informed consent/assent or undergoing any of the treatment options or patients unwilling or unable to comply with the protocol (including use of electronic devices for ePRO).
9. Local access to new formulation (granules or FCT) is available for the patient
(For the optional extension phase criteria only).
10. Meeting any of the exclusion criteria as defined in the core phase (For the
optional extension phase criteria only). 
 
Method of Generating Random Sequence   Computer generated randomization 
Method of Concealment   On-site computer system 
Blinding/Masking   Open Label 
Primary Outcome  
Outcome  TimePoints 
1. To evaluate both formulations on patient compliance, using stick pack/tablet count in ICT naïve
patients during core phase
2. To evaluate the change from baseline in serum
ferritin at 48 weeks of treatment for both formulations
in ICT naïve patients 		 1. To evaluate both formulations on patient
compliance, using stick pack/tablet count in ICT naïve
patients during core phase
2. To evaluate the change from baseline in serum
ferritin at 48 weeks of treatment for both formulations
in ICT naïve patients 
 
Secondary Outcome  
Outcome  TimePoints 
To evaluate both formulations on change in serum
ferritin in ICT naïve and pre-treated patients 		 Change from baseline in serum ferritin at 48 weeks of
treatment 
To evaluate both formulations on patient satisfaction
and palatability using Patient / Observer Reported
Outcomes (PRO/ObsRO) questionnaires 		 Domain scores of treatment satisfaction and
palatability over time 
To evaluate both formulations on overall safety  Overall safety, as measured by frequency and severity
of adverse events (including active monitoring for renal
toxicity; including renal failure, hepatic toxicity;
including hepatic failure, and gastrointestinal
hemorrhage), and changes in laboratory values from
baseline (serum creatinine, creatinine clearance, ALT,
AST, RBC and WBC). In addition, vital signs, physical,
ophthalmological, audiometric, cardiac, and growth
and development evaluations will be assessed. 
To evaluate compliance using a daily PRO/ObsRO
questionnaire 		 Rate of dosing instructions deviations (doses missed /
not taken at the same time every day) 
To evaluate pre-dose PK data to support the
assessment of compliance 		 Pre-dose deferasirox concentrations in all patients
at Weeks 1, 3, 5, 9, 13, 17, 21, 25, 29,
33, 37, 41, and 45 (13 samples) 
Post-dose data to be analyzed along with pre-dose PK
data 		 Post-dose deferasirox concentrations between 2 and 4
hours post-dose at Weeks 5 and 9 (2 samples) 
To explore exposure-response relationships for
measures of safety and effectiveness 		 Serum creatinine change from baseline, notable
serum creatinine values, serum creatinine clearance
change from baseline and notable serum creatinine
clearance categories, serum ferritin change from
baseline, in relationship to pre- and post-dose
deferasirox concentrations. 
(For optional extension phase) To assess additional safety data about new
formulation (granules) in pediatric population 		 Overall safety, as measured by frequency and severity
of adverse events (including active monitoring for renal
toxicity; including renal failure, hepatic toxicity;
including hepatic failure, and gastrointestinal
hemorrhage), and changes in laboratory values from
baseline (serum creatinine, creatinine clearance, ALT,
AST, RBC and WBC). In addition, vital signs, physical,
ophthalmological, audiometric, and growth and
development evaluations will be assessed. 
 
Target Sample Size   Total Sample Size="240"
Sample Size from India="20" 
Final Enrollment numbers achieved (Total)= "224"
Final Enrollment numbers achieved (India)="76" 
Phase of Trial   Phase 2 
Date of First Enrollment (India)   15/11/2017 
Date of Study Completion (India) 17/06/2020 
Date of First Enrollment (Global)  21/10/2015 
Date of Study Completion (Global) 15/01/2024 
Estimated Duration of Trial   Years="5"
Months="0"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Completed 
Recruitment Status of Trial (India)  Completed 
Publication Details   Novartis assures that the key design elements of this protocol will be posted in a publicly accessible database such as clinicaltrials.gov. In addition, upon study completion and finalization of the study report the results of this study will be either submitted for publication and/or posted in a publicly accessible database of clinical study results.  
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Brief Summary  

This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin over time of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged ≥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, and having a treatment goal to reduce iron burden as measured by serum ferritin. Primary objective of the study is to evaluate patient compliance (using stick pack or tablet counts) and change in serum ferritin over time for both formulations of deferasirox, i.e., granules and dispersible tablet (DT) in iron chelation therapy (ICT) naïve patients in the core phase. Male and female children and adolescents aged ≥ 2 and < 18 years.

Written informed consent/assent before any study-specific procedures. Consent will be obtained from patients, parent(s) or legal guardians. Investigators will also obtain consent/assent of patients according to local guidelines.

Patients will be randomized to either Deferasirox granules or Deferasirox DT(Exjade) in the core phase.All patients who choose to continue in the extension phase will receive deferasirox granules in the extension phase.

The co-primary efficacy endpoints assessed during the core phase will be Compliance measured by stick pack/tablet count.and Change from baseline in serum ferritin at 48 weeks of treatment.

Compliance will be measured by stick pack/tablet count: it will be performed by study personnel every 4 weeks  during the core phase based on the amount of medication dispensed, returned and reported as lost/wasted by the patient / caregiver.

Serum ferritin test will be performed at Screening Visits 1 and 2 and every 4 weeks from week 5 till end of treatment visit.























 
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