Study to evaluate treatment compliance, efficacy and safety of an improved deferasirox formulation (granules) in pediatric patients (2-<18 years old) with iron overload

Phase 1

• Conditions: transfusion-dependent anemia requiring chelation therapy due to iron overload; MedDRA version: 20.0Level: LLTClassification code 10074295Term: Transfusion dependent anemiaSystem Organ Class: 100000012844; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2013-004739-55-HU
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-07-25
• Last Update Posted: 19 February 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 240

Inclusion Criteria

1. Written informed consent/assent before any study-specific procedures. Consent will be obtained from parent(s) or legal guardians. Investigators will also obtain consent/assent of patients according to local guidelines.
2. 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
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 for inclusion in the core phase or Visit 16 and Visit 777
for inclusion in the extension phase (the mean value will be used for eligibility criteria).
5. Patient has to have participated and completed the 48 weeks core phase treatment as per protocol (For optional extension phase eligibility only).
6. Patient needs to meet all the eligibility criteria (inclusion and exclusion) as defined in the core phase at Visit 17 (For optional extension phase eligibility only).

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

Exclusion Criteria

1. Creatinine clearance below the contraindication limit in the locally approved prescribing information (using the Schwartz formula) at screening Visit 1 , screening Visit 2, Visit 16 or Visit 777
2. Serum creatinine > 1.5 xULN at screening Visit 1 , screening Visit 2, Visit 16 or Visit 777
3. ALT and/or AST > 3.0 x ULN at screening Visit 1 , screening Visit 2, Visit 16 or Visit 777
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, screening Visit 2, Visit 16 or Visit 777
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