Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload

A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients With Iron Overload

ClinicalTrials.gov Identifier: NCT02435212

Novartis Reference Number: CICL670F2202

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged ≥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden.

Randomization will be stratified by age groups (2 to <10 years, 10 to <18 years) and prior iron chelation therapy (Yes/ No). There will be two study phases which include a 1 year core phase where patients will be randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all patients will receive the granules up to 5 years. Patients who demonstrated benefit to granules or DT in the core phase, and/or express the wish to continue in the optional extension phase on granules, will be offered this possibility until there is local access to the new formulation (granules or FCT) or up to 5 years, whichever occurs first.

Condition 
Transfusion-dependent Anemia
Phase 
Phase 2
Overall status 
Active, not recruiting
Enrollment count 
224 participants
Start date 
Oct 21, 2015
Completion date 
Dec 19, 2023
Gender 
All
Age(s)
2 Years - 17 Years (Child)

Interventions

Drug
Deferasirox granule formulation
Deferasirox granules will be provided as stick packs containing 90 mg, 180 mg and 360 mg granules for oral use.
Drug
Deferasirox DT formulation
Deferasirox DT will be provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use

Eligibility Criteria

Inclusion Criteria:

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.
Male and female children and adolescents aged ≥ 2 and < 18 years. [France: Male and female children and adolescent aged ≥ 2 and < 18 years old, however children aged ≥ 2 and ≤ 6years can be enrolled only when deferoxamine treatment is contraindicated or inadequate in these patients as per investigator decision. 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.
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).
Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
Patient has to have participated and completed the 48 weeks core phase treatment as per protocol (For optional extension phase eligibility only).

Exclusion Criteria:

Creatinine clearance below the contraindication limit in the locally approved prescribing information (using Schwartz formula) at screening visit 1 or screening visit 2.
Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and or screening Visit 2
ALT and/or AST > 3.0 x ULN at screening visit 1 or screening visit 2.
(Criterion no longer applicable, removed as part of Amendment 1): Prior iron chelation therapy.
Liver disease with severity of Child-Pugh class B or C.
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.
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 protocol-defined Inclusion/Exclusion may apply.

Study Locations

United States
Children's Hospital Oakland Onc Dept
-
Oakland, 94609
California
United States
Childrens Healthcare of Atlanta Onc Dept
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Atlanta, 30342
Georgia
United States
Lurie Children's Hospital of Chicago Onc Dept
-
Chicago, 60611
Illinois
United States
Children's Hospital at Montefiore
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Bronx, 10467
New York
United States
Weill Cornell Medical College SC -
-
New York, 10021
New York
United States
Childrens Hospital of Philadelphia Onc. Dept
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Philadelphia, 19104 4399
Pennsylvania
United States
Medical University of South Carolina Medical Uni of South Carolina
-
Charleston, 29425
South Carolina
United States
St. Jude Children's Research Hospital Memphis St Jude
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Memphis, 38105
Tennessee
United States
Belgium
Novartis Investigative Site
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Edegem, 2650
Antwerpen
Belgium
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Brussel, 1200
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Belgium
Bulgaria
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Plovdiv, 4002
-
Bulgaria
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Sofia, 1527
-
Bulgaria
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Varna, 9010
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Bulgaria
Egypt
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Alexandria, 21131
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Egypt
France
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Creteil, 94000
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France
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Paris, 75015
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France
Hungary
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Debrecen, 4032
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Hungary
India
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Kolkata, 700017
West Bengal
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Italy
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Genova, 16128
GE
Italy
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Palermo, 90127
PA
Italy
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Palermo, 90146
PA
Italy
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Napoli, 80132
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Italy
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Hazmiyeh, PO BOX 213
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Russian Federation
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Bangkok noi, 10700
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Adana, 01330
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Turkey
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Ankara, 06100
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Turkey
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Izmir, 35040
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Turkey

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