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Trial details
Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis (MIMAS)
Current status:
Approved
|
Date registered:
17/12/2019
Trial version(s)
History: 28/02/2019
History: 28/02/2019
History: 28/02/2019
History: 28/02/2019
Current: 28/02/2019
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Main Information
Primary registry identifying number
LBCTR2019030206
Protocol number
CICL670F2429
MOH registration number
32772/2018
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
01/08/2018
Primary sponsor
Novartis Pharma Services Inc.
Primary sponsor: Country of origin
Novartis Pharmaceuticals
Public title
Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis (MIMAS)
Acronym
Scientific title
A Single-arm Interventional Phase IV, Post-authorisation Study Evaluating the Safety of Pediatric Patients With Transfusional Hemosiderosis Treated With Deferasirox Crushed Film Coated Tablets
Acronym
Brief summary of the study: English
The study employs an interventional, prospective, single arm, open label, global, multicenter,non-randomized trial design to monitor and assess the safety profile of the crushed deferasirox FCT in pediatric patients between age ≥2 to <6 with transfusional hemosiderosis over 24 weeks. This study will aim to enroll at least 40 patients.
Brief summary of the study: Arabic
دراسة تدخلية وحيدة المجموعة في المرحلة الرابعة بعد الترخيص لتقييم سلامة المرضى الاطفال المصابين بالحدد الدموي (الهيموسيدروسز) ذي الصلة بنقل الدم والمعالجين بأقراص ديفيرازيروكس المسحوقة المغلفة بطبقة رقيقة
Health conditions/problem studied: Specify
Transfusional Hemosiderosis
Interventions: Specify
Drug: Deferasirox Deferosirox is provided in tablet forms of 90, 180 and 360mg. Tablets must be crushed. Other Name: ICL670
Key inclusion and exclusion criteria: Inclusion criteria
1.Patients ≥2 to <6 years old diagnosed with transfusional hemosiderosis 2.Documented history of red blood cell transfusions 3.Written informed consent/assent before any study-specific procedures. The consent will be obtained from caregiver(s) or patient's legal representative. Investigators will also obtain assent of patients according to local, regional, or national regulations. 4.For patients on prior DFX: Serum ferritin (SF) >500 ng/mL, measured at screening visit 1 and requiring a DFX daily dose equivalent to FCT ≥ 7mg/kg/day. 5.For patients on a prior chelator other than DFX (e.g. deferiprone or deferoxamine) or chelation naive: Serum ferritin (SF) >1000 ng/mL measured at screening visits 1 and 2.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
2
Key inclusion and exclusion criteria: Age maximum
6
Key inclusion and exclusion criteria: Exclusion criteria
1.Patients that receive more than one iron chelator at the same time as current iron chelation treatment. (Patients who have received combination therapy in their medical history but are currently being treated with a single ICT agent are eligible.) 2.Patients continuing on deferoxamine or deferiprone in addition to study treatment. (Patients switching to or continuing on deferasirox are eligible). 3.Unresolved adverse events if the patient was previously treated with deferiprone or deferoxamine or deferasirox. 4.Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void sample urine measured at screening visit 1. 5.Serum creatinine > age adjusted ULN measured at any screening visit 6.Creatinine clearance below 90 mL/minute measured at any screening visit. Creatinine clearance using the Schwartz formula will be estimated from serum creatinine measured at each respective visit. 7.ALT and/or AST > 2.5 x ULN measured at screening visit 1. 8.Total bilirubin (TBIL) >1.5 x ULN measured at screening visit 1. 9.Patients with significant impaired GI function or GI disease that may significantly alter the absorption of oral deferasirox FCT (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection). 10.History of and/or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive. 11.Liver disease with severity of Child-Pugh Class B or C. 12.History of hypersensitivity to any of the study drug or excipients. 13.Patients participating in another clinical trial or receiving an investigational drug. 14.Patients with a known history of HIV seropositivity. 15.Patients unwilling or unable to comply with the protocol. 16.History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin. 17.Significant medical condition interfering with the ability to partake in this study (e.g. uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease: cardiovascular, renal, hepatic, etc.). 18.Female patients who reach menarche and they or their caregivers refuse pregnancy testing and/or if there is a positive pregnancy test result.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
N/A: Single arm study
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
4
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
Yes, Lebanon and Worldwide
IMP has market authorization: Specify the countries
USA, UK, France, Germany, Netherlands, Switzerland, Sweden, Italy...
Name of IMP
Deferasirox Film Coated Tablets "Jadenu"
Year of authorization
2017
Month of authorization
10
Type of IMP
Others
Type of IMP: Specify
Tridentate iron chelators
Pharmaceutical class
Non-chiral, Tridentate ligand iron chelator
Therapeutic indication
Iron Over Load
Therapeutic benefit
Treatment of Iron Overload symptoms
Biospecimen retention
None retained
Biospecimen description
Local Lab tests at site
Target sample size
3
Actual enrollment target size
3
Date of first enrollment: Type
Actual
Date of first enrollment: Date
10/10/2018
Date of study closure: Type
Actual
Date of study closure: Date
11/03/2020
Recruitment status
Complete
Date of completion
17/05/2019
IPD sharing statement plan
No
IPD sharing statement description
undecided
Additional data URL
https://clinicaltrials.gov/ct2/show/record/NCT03372083?id=CICL670F2429&rank=1&view=record
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Clinical Trials. gov
NCT03372083
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
009613755669
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
Chronic Care Center
26/07/2018
Michele Abi saad
cccmas@chroniccare.org.lb
+961 3 664 310
American University of Beirut Medical Center
05/07/2018
Fuad Ziyadeh
fz05@aub.edu.lb
+961 (0) 1 350 000 ext:5445
Countries of Recruitment
Name
Egypt
Lebanon
Italy
Oman
Russian Federation
Saudi Arabia
Thailand
United Arab Emirates
Health Conditions or Problems Studied
Condition
Code
Keyword
Transfusional Hemosiderosis
Thalassaemia, unspecified (D56.9)
Transfusional Hemosiderosis
Interventions
Intervention
Description
Keyword
Physical examination, Vitals, Height, Weight, Hematology, Chemistry, urinalisis, ECG, Ocular assessment, Auditory assessment
Physical examination, Vitals, Height, Weight, Hematology, Chemistry, urinalisis, ECG, Ocular assessment, Auditory assessment
ICF, IMP, Lab tests , diary completion
Primary Outcomes
Name
Time points
Measure
Percentage of patients with selected gastrointestinal disorders
24 weeks
24 wks
To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders
through out the study
through out the study
Key Secondary Outcomes
Name
Time points
Measure
•Percentage of patients who experienced AEs suspected to be related to study drug
24 weeks
24 wks
•Change from baseline ECGs up
24 weeks
24 weeks
•Change from baseline serum ferritin (SF)
24 weeks
24 weeks
•Absolute change for serum creatinine
24 weeks
24 weeks
•Absolute change for creatinine clearance UPCR
24 weeks
24 weeks
•Palatability Questionnaire Score
24 weeks
24 weeks
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
Updated recruitment status to : completed. Also attached as per yearly process the SUSARS, PDs, and IB
15/12/2019
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