Phase IV safety study of crushed deferasirox film coated tablets in pediatric patients with transfusional hemosiderosis

Phase 1

• Conditions: Transfusional Hemosiderosis; MedDRA version: 20.1Level: LLTClassification code 10019613Term: HemosiderosisSystem Organ Class: 100000004861; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-003482-25-GB
• Lead Sponsor: ovartis Pharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-10-17
• Study Completion: 2019-12-05
• Last Update Posted: 13 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 44

Inclusion Criteria

oPatients =2 to <6 years old diagnosed with transfusional hemosiderosis
oDocumented history of red blood cell transfusions
oWritten informed consent/assent before any study-specific procedures.
oFor 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.
oFor patients on a prior chelator other than DFX (e.g. deferiprone or deferaxamine) or chelation naive: Serum ferritin (SF) >1000 ng/mL measured at screening visits 1 and 2
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

oHistory of hypersensitivity to any of the study drug or excipients.
oSerum creatinine > age adjusted ULN measured at any screening visit
oCreatinine clearance below 90 mL/minute measured at any screening visit.
oALT and/or AST > 2.5 x ULN measured at screening visit 1.
oTotal bilirubin (TBIL) >1.5 x ULN measured at screening visit 1.
oPatients 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).
oPatients unwilling or unable to comply with the protocol

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified