Transfusional Hemosiderosis MedDRA version: 20.1 Level: LLT Classification code 10019613 Term: Hemosiderosis System Organ Class: 100000004861
Conditions
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: o Patients =2 to 500 ng/mL, measured at screening visit 1 and requiring a DFX daily dose equivalent to FCT = 7mg/kg/day. o For 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
Exclusion criteria: o History of hypersensitivity to any of the study drug or excipients. o Serum creatinine > age adjusted ULN measured at any screening visit o Creatinine clearance below 90 mL/minute measured at any screening visit. o ALT and/or AST > 2.5 x ULN measured at screening visit 1. o Total bilirubin (TBIL) >1.5 x ULN measured at screening visit 1. o 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). o Patients unwilling or unable to comply with the protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: to assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal(GI) disorders in pediatric patients aged =2 to < 6 years with transfusional iron overload up to 24 weeks including 30 days safety follow-up;Secondary Objective: to evaluate adverse events (AEs) suspected to be related to the crushed deferasirox FCT during the study;Primary end point(s): number and percentage of patients with selected gastrointestinal disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea and vomiting) up to 24 weeks including 30 days safety follow-up;Timepoint(s) of evaluation of this end point: Week 2, week 3, week 4, week 8, week 12, week 16, week 20 and week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): o To assess the overall safety of crushed FCT of deferasirox o To assess the efficacy of deferasirox FCT treatment o To evaluate patient treatment satisfaction, palatability and gastrointestinal (GI) symptoms with Observer Reported Outcomes (ObsROs) ;Timepoint(s) of evaluation of this end point: Week 2, week 3, week 4, week 8, week 12, week 16, week 20 and week 24 | — |
Countries
Chile, Egypt, France, Italy, Lebanon, Malaysia, Oman, Russian Federation, Saudi Arabia, Thailand, Turkey, United Arab Emirates, United Kingdom, United States
Contacts
Public ContactMedica Information Services
Novartis Pharmaceuticals UK Limited
Outcome results
None listed