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Trial details
Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload ( CALYPSO)
Current status:
Approved
|
Date registered:
07/05/2019
Trial version(s)
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
Current: 20/02/2019
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Main Information
Primary registry identifying number
LBCTR2019020197
Protocol number
ICL670F2202
MOH registration number
6428/ص
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Retrospective
Type of registration: Justify
LCTR was recently initiated, original file was previously submitted by Paper
Date of registration in national regulatory agency
15/07/2015
Primary sponsor
Novartis Pharma Services Inc.
Primary sponsor: Country of origin
Novartis Pharmaceuticals
Public title
Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload ( CALYPSO)
Acronym
CALYPSO
Scientific title
A 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
Acronym
Brief summary of the study: English
This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin 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, to demonstrate the effect of improved compliance on iron burden. Randomization will be stratified by age groups (2 to <10 years, 10 to <18 years) and prior iron chelation therapy (Yes/ No). There will be two study phases which include a 1 year core phase where patients will be randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all patients will receive the granules up to 5 years. Patients who demonstrated benefit to granules or DT in the core phase, and/or express the wish to continue in the optional extension phase on granules, will be offered this possibility until there is local access to the new formulation (granules or FCT) or up to 5 years, whichever occurs first.
Brief summary of the study: Arabic
دراسة عشوائيّة التوزيع، مفتوحة اللصاقة، متعددة المراكز، ذات مجموعتين، في المرحلة الثانية لتقييم الامتثال للعلاج بصيغة ديفيرازيروكس محسّنة (حُبيبات) وفعاليّتها وسلامتها لدى الأطفال المرضى الذين يعانون من الحديد الزائد
Health conditions/problem studied: Specify
Pediatric Patients (2-<18 Years Old) With Iron Overload
Interventions: Specify
•Drug: Deferasirox granule formulation Deferasirox granules will be provided as stick packs containing 90 mg, 180 mg and 360 mg granules for oral use. Other Name: ICL670 •Drug: Deferasirox DT formulation Deferasirox DT will be provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use Other Name: ICL670
Key inclusion and exclusion criteria: Inclusion criteria
•Written informed consent/assent before any study-specific procedures. Consent will be obtained from parent(s) or legal guardians. Investigators will also obtain assent of patients according to local guidelines. •Male and female children and adolescents aged ≥ 2 and < 18 years. [France: Male and female children and adolescent aged ≥ 2 and < 18 years old, however children aged ≥ 2 and ≤ 6years can be enrolled only when deferoxamine treatment is contraindicated or inadequate in these patients as per investigator decision. 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. •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 to reduce iron burden (300mL PRBC = 1 unit in adults whereas 4 ml/kg PRBC is considered 1 unit for children). •Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria). •Patient has to have participated and completed the 48 weeks core phase treatment as per protocol (For optional extension phase eligibility only).
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
2
Key inclusion and exclusion criteria: Age maximum
18
Key inclusion and exclusion criteria: Exclusion criteria
•Creatinine clearance below the contraindication limit in the locally approved prescribing information (using Schwartz formula) at screening visit 1 or screening visit 2. •Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and or screening Visit 2 •ALT and/or AST > 3.0 x ULN at screening visit 1 or screening visit 2. •(Criterion no longer applicable, removed as part of Amendment 1): Prior iron chelation therapy. •Liver disease with severity of Child-Pugh class B or C. •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. •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). Other protocol-defined Inclusion/Exclusion may apply.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
Randomized controlled trial
Study design: Masking
Open (masking not used)
Study design: Control
Active
Study phase
2
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
Yes, Lebanon and Worldwide
IMP has market authorization: Specify the countries
Worldwide
Name of IMP
Jadenu ( ICL670 ) / Deferasirox
Year of authorization
2017
Month of authorization
10
Type of IMP
Others
Type of IMP: Specify
tridentate iron chelators
Pharmaceutical class
Deferasirox is an N-substituted bis-hydroxyphenyl-triazole, a class of tridentate iron chelators.
Therapeutic indication
Patients with Iron Overload/ Transfusion Dependent Anemia
Therapeutic benefit
- Change in serum ferritin in ICT naïve patients. -The comparison of means between the two treatment arms of change from baseline to week 24 of treatment in serum ferritin in pediatric ICT naïve patients with iron overload.
Biospecimen retention
Samples with DNA**
Biospecimen description
MCHC, MCV, Platelets, Red blood cells, White blood cells(WBC) count with differential, RBC Morphology with Differential (Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils) Biochemistry Albumin, Alkaline phosphatase, ALT, AST, Bicarbonate, Calcium, Chloride, Creatinine, Creatine kinase, Direct (conjugated) Bilirubin, Indirect Bilirubin, Total Bilirubin, Total Cholesterol, LDL, HDL, Lactate Dehydrogenase (LDH), Total Protein, Triglycerides, Blood Urea Nitrogen (BUN) or Urea, Uric Acid, C Reactive Protein (CRP), Urinalysis Microscopic Panel: Red Blood Cells, White Blood Cells, Casts, Crystals, Bacteria, Epithelial cells Macroscopic Panel (Dipstick): Color, Bilirubin, Blood, Glucose, Ketones, Leukocytes esterase, Nitrite, pH, Protein, Specific Gravity, Urobilinogen Hepatitis markers HbsAg, HbsAb, HbcAb, HCV RNA, Anti-HCV Additional tests Serum ferritin, creatinine clearance, urine protein/creatinine ratio, serum pregnancy test
Target sample size
23
Actual enrollment target size
23
Date of first enrollment: Type
Actual
Date of first enrollment: Date
13/10/2016
Date of study closure: Type
Actual
Date of study closure: Date
31/12/2019
Recruitment status
Complete
Date of completion
21/12/2017
IPD sharing statement plan
No
IPD sharing statement description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Additional data URL
https://clinicaltrials.gov/ct2/show/NCT02435212?term=2013-004739-55&rank=1
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Clinical Trials. gov
NCT02435212
Sources of Monetary or Material Support
Name
Novartis Pharma Services Inc.
Secondary Sponsors
Name
NA
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Ali
Taher
Beirut
Lebanon
01-350000 ext 5392
ataher@aub.edu.lb
Chronic Care Center
Scientific
Hind
Khairallah
Sin El Fil
Lebanon
+961 1 512002 Ext. 271
Hind.Khairallah@fattal.com.lb
Khalil Fattal et Fils s.a.l.
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Chronic Care Center
Dr Ali Taher
Hematology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
American University of Beirut Medical Center
15/06/2016
Fouad Ziyadeh
fz05@aub.edu.lb
+961 (0) 1 350 000 ext:5445
Chronic Care Center
11/07/2016
Michele Abi saad
cccmas@chroniccare.org.lb
+961 3 664 310
Countries of Recruitment
Name
Lebanon
Belgium
Bulgaria
Egypt
Oman
United States of America
India
Italy
France
Tunisia
Turkey
Health Conditions or Problems Studied
Condition
Code
Keyword
Patients with Iron Overload/ Transfusion Dependent Anemia
Anaemia, unspecified (D64.9)
Transfusion Dependent Anemia
Interventions
Intervention
Description
Keyword
Physical examination, height, weight, Hematology, Chemistry, Ferritin, Creatinine, Cleatinine Clearance, Hepatitis, Pregnancy Test, Urine Dipstick, Microscopic Urinalysis, Proteinuria, Urine Pregnancy Test, Liver function test, Ocular exam, audiometry, ECG, Electrocardiogram, PK sampling, vital signs, Growth and development
ICF, IMP, Lab tests and ECG , diary completion
ICF, IMP, Lab tests and ECG , diary completion
Primary Outcomes
Name
Time points
Measure
•Compliance (using stick/pack tablet count).
24 weeks
24 wks
•Change in serum ferritin in ICT naive patients
baseline, 24 wks
baseline, 24 wks
Key Secondary Outcomes
Name
Time points
Measure
•Compliance (using stick/pack tablet count)
48 weeks
48 wks
•Change in serum ferritin in ICT naive patients
baseline, 24 wks, 48 wks
baseline, 24 wks, 48 wks
•Overall safety, as measured by frequency and severity of adverse
from baseline to 48 wks
from baseline to 48 wks
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial