Clinical trial to Evaluate Treatment Compliance, Efficacy and Safety of an Improved new formulation of Deferasirox in the form of Granules in children of the age 2 to 18 Years Old who have Iron overload.
- Conditions
- Health Condition 1: null- Patient with transfusion-dependent anemia associated with iron overload requiring iron chelationtherapy and with a history of transfusion and a treatmentgoal to reduce iron burden
- Registration Number
- CTRI/2017/10/010252
- Lead Sponsor
- ovartis Healthcare Private Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Closed to Recruitment of Participants
- Sex
- Not specified
- Target Recruitment
- 6
1. Written informed consent/assent before any study-specific procedures.
2. Consent will be obtained from parent(s) or legal guardians. Investigators will also
obtain assent of patients according to local guidelines.
3. Male and female children and adolescents aged between 2 and 18 years.
Applicable to core phase only. Once in the core phase patients can turn 18 years
and still be considered eligible, also for participation in the optional extension
phase.
4. 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 of reduction, not maintenance of iron burden as measured by
serum ferritin.
5. Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit
2 (the mean value will be used for eligibility criteria).
6. Completion of core phase per protocol (For the optional extension phase
criteria only).
7. Meeting inclusion criteria as defined in the core phase at Visit 17 except for age
requirement (For the optional extension phase criteria only).
1. Creatinine clearance below the contraindication limit in the locally approved
prescribing informationCreatinine clearance will be estimated from serum creatinine (using the Schwartz formula) at screening Visit 1 or screening Visit 2.
2. Serum creatinine > 1.5 xULN at screening Visit 1 or screening Visit 2.
3. ALT or AST greater than 3.0 x ULN at screening visit 1 or screening visit 2.
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 second morning 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).
7. Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive).
8. Patients with psychiatric or addictive disorders which prevent them from giving
their informed consent/assent or undergoing any of the treatment options or patients unwilling or unable to comply with the protocol (including use of electronic devices for ePRO).
9. Local access to new formulation (granules or FCT) is available for the patient
(For the optional extension phase criteria only).
10. Meeting any of the exclusion criteria as defined in the core phase (For the
optional extension phase criteria only).
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method