Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis (MIMAS)

• Register: LBCTR
• Last refreshed on: 30 August 2021
• Main ID: LBCTR2019030206
• Date of registration: 06/02/2021
• Prospective Registration: No
• Primary sponsor: Novartis Pharma Services Inc.
• Public title: Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis (MIMAS)
• 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
• Date of first enrolment: 10/10/2018
• Target sample size: 3
• Recruitment status: Complete
• URL: https://lbctr.moph.gov.lb/Trials/Details/4743
• Study type: Interventional
• Study design: Allocation: N/A: Single arm study;Masking: Open (masking not used);Control: N/A;Assignment: Single;Purpose: Treatment
• Phase: 4

Countries of recruitment

Egypt, Italy, Lebanon, Oman, Russian Federation, Saudi Arabia, Thailand, United Arab Emirates

Contacts

• Name: Ali Taher
• Affiliation: Chronic Care Center

• Name: Hind Khairallah
• Affiliation: Khalil Fattal et Fils s.a.l.

Key inclusion & 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.

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.


Age minimum: 2
Age maximum: 6
Gender: Both

Health Condition(s) or Problem(s) studied

D56.9

Transfusional Hemosiderosis

Transfusional Hemosiderosis
Transfusional Hemosiderosis

Intervention(s)

Drug: Deferasirox
Deferosirox is provided in tablet forms of 90, 180 and 360mg. Tablets must be crushed.

Other Name: ICL670

ICF, IMP, Lab tests , diary completion

Physical examination, Vitals, Height, Weight, Hematology, Chemistry, urinalisis, ECG, Ocular assessment, Auditory assessment

Primary Outcome(s)

Name: Percentage of patients with selected gastrointestinal disorders ;Timepoints: 24 weeks;Measure: 24 wks

Name: To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders ;Timepoints: through out the study ;Measure: through out the study

Secondary Outcome(s)

Name: •Absolute change for creatinine clearance UPCR ;Timepoints: 24 weeks;Measure: 24 weeks

Name: •Absolute change for serum creatinine ;Timepoints: 24 weeks;Measure: 24 weeks

Name: •Change from baseline ECGs up ;Timepoints: 24 weeks;Measure: 24 weeks

Name: •Change from baseline serum ferritin (SF);Timepoints: 24 weeks;Measure: 24 weeks

Name: •Palatability Questionnaire Score;Timepoints: 24 weeks;Measure: 24 weeks

Name: •Percentage of patients who experienced AEs suspected to be related to study drug ;Timepoints: 24 weeks;Measure: 24 wks

Secondary ID(s)

NCT03372083

Source(s) of Monetary Support

Novartis Pharma Services Inc.

Ethics review

Status: Approved
Approval date: 05/07/2018
Contact:

[e-mail]

American University of Beirut Medical Center

[phone] ext:5445

[e-mail]

Status: Approved
Approval date: 26/07/2018
Contact:

[e-mail]

Chronic Care Center

[phone]

[e-mail]

Results

• Date Completed: 17/05/2019