Study to evaluate treatment compliance, efficacy and safety of an improved deferasirox formulation (granules) in pediatric patients (2-<18 years old) with iron overload
- Conditions
- transfusion-dependent anemia requiring chelation therapy due to iron overloadMedDRA version: 19.0Level: LLTClassification code 10074295Term: Transfusion dependent anemiaSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2013-004739-55-DK
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 240
1. Written informed consent/assent before any study-specific procedures. Consent will be obtained from parent(s) or legal guardians. Investigators will also obtain assent of patients according to local guidelines.
2. Male and female children and adolescents aged = 2 and < 18 years.
3. Any transfusion-dependent anemia associated with iron overload requiring iron chelation therapy and with a history of transfusion of approximately 20 PRBC units and a treatment goal to reduce iron burden (300mL PRBC = 1 unit in adults whereas 4 ml/kg PRBC is considered 1 unit for children).
4. Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
Are the trial subjects under 18? yes
Number of subjects for this age range: 240
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
1. Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine (using the Schwartz formula) at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
2. Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
3. ALT and/or AST > 3.0 x ULN.
4. Liver disease with severity of Child-Pugh class B or C.
5. Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
6. Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
Other exclusion criteria as per full protocol may apply.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: 1. To evaluate both formulations on patient compliance, using stick pack/tablet count in ICT naive patients<br>2. To evaluate the change from baseline in serum ferritin at 48 weeks of treatment for both formulations in ICT naive patients;Secondary Objective: 1.to evaluate both formulations on serum ferritin in ICT naive and pre-treated patients<br>2. To evaluate both formulations on patient satisfaction and palatability using Patient / Observer Reported Outcomes<br>(PRO/ObsRO) questionnaires<br>3. To evaluate both formulations on overall safety<br>4. To evaluate compliance using a daily PRO/ObsRO questionnaire<br>5. To evaluate pre-dose PK data to support the assessment of compliance<br>6. Post-dose data to be analyzed along with pre-dose PK data;Primary end point(s): 1. Compliance measured by stick pack/tablet count<br>2. Change from baseline in serum ferritin at 48 weeks of treatment;Timepoint(s) of evaluation of this end point: 1. 48 weeks<br>2. baseline and 48 weeks
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. Change from baseline in serum ferritin at 48 weeks of treatment<br>2. Domain scores of treatment satisfaction and palatability over time<br>3. Frequency of Adverse Events (AEs) as a measure of overall safety<br>4. Severity of Adverse Events (AEs) as a measure of overall safety<br>4. Rate of dosing instructions deviations<br>5. Pre-dose deferasirox concentrations in all patients<br>6. Post-dose deferasirox concentrations between 2 and 4 hours post-dose at Weeks 5 and 9 (2 samples);Timepoint(s) of evaluation of this end point: 1. 48 weeks<br>2. 48 weeks<br>3. Baseline and 48 weeks<br>4. Baseline and 48 weeks<br>5. Weeks 1, 3, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45<br>6. Week 5 and 9 <br>