Chronic iron overload from blood transfusions in patients with congenital disorders of red blood cells
Conditions
Interventions
Product Name: EXJADE
Product Code: ICL670
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: deferasirox
CAS Number: 201530-41
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Of either sex and aged 2 years or older Documented congenital disorder of red blood cells requiring ongoing blood transfusions Patients that cannot be adequately treated with a locally approved iron chelator due to one of the following reasons: Documented non-compliance; Contraindications, unacceptable toxicities and/or documented poor response to locally approved iron chelators, despite proper compliance; History of at least 20 blood transfusions (equivalent to 100 mL/kg of PRBC) Serum ferritin value greater than or equal to 1000 µg/L Ability to comply with all study-related procedures, medications, and evaluations Written informed consent by the patient or for pediatric patients consent of the patient’s legal guardian. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Ongoing treatment with another iron chelator Eligibility for any other ongoing Novartis sponsored clinical study protocol with EXJADE® and geographic access to these sites Galactose intolerance, severe lactase deficiency or glucose-galactose malabsorption Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with EXJADE® Serum creatinine above the upper limit of normal within one week prior to baseline Significant proteinuria as indicated by a urinary protein/creatinine ratio= 0.5 (mg/mg) in a non-first void urine sample within one week prior to baseline, or alternatively a urinary protein/creatinine ratio = 0.5 (mg/mg) in two of three samples obtained ALT =500 U/L within one week prior to baseline Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline Pregnancy and patients who are breastfeeding Patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Provide expanded access of EXJADE® to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot be treated with other locally approved iron chelators due to documented non-compliance, contraindications, unacceptable toxicities and/or documented poor response. Further evaluate the safety profile of EXJADE® based upon drug administration and reporting of serious adverse events. ;Secondary Objective: None.;Primary end point(s): Safety (based on adverse event monitoring, monitoring and recording all serious adverse events, the regular monitoring of hematology and blood chemistry, and the regular monitoring of body weight, ocular and auditory conditions.) | — |
Countries
Belgium, Germany, Greece, Ireland, Italy, Spain, United Kingdom
Outcome results
None listed