Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation

Novartis

Status and phase: Completed; Phase 3

Conditions: Chronic Iron Overload

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT02720536

CICL670AIC04

Details and patient eligibility

About

Extend evaluation of deferasirox film-coated tablet (FCT) formulation

Full description

Collection of additional safety and efficacy data with deferasirox film-coated tablet (FCT) in patients who had completed study CICL670F2201

• Provide patients who completed 24 weeks of treatment in the core study, CICL670F2201, the possibility to have additional treatment with the deferasirox FCT.
• Collect additional longer term data on the safety and the tolerability of the deferasirox FCT.
• Collect efficacy data on the deferasirox FCT in reduction or maintenance of iron burden as measured by serum ferritin level.

Enrollment: 53 patients

Sex: All

Ages: 10+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria for subjects:

• Completed 24-weeks of study treatment as described in the core protocol (CICL670F2201).
• Were deemed to have tolerated deferasirox treatment by the investigator.
• Provided written informed consent/assent before any study-specific procedures were performed. For pediatric patients, consent was obtained from parent(s) or legal patient's representative. Investigators were to have also obtained assent of patients according to local, regional or national guidelines.

Key Exclusion for subjects:

The exclusion criteria followed those described for the core protocol CICl670F2201, which were as follows:

• Creatinine clearance below the contraindication limit in the locally approved prescribing information.
• Serum creatinine > 1.5 × upper limit of normal range (ULN) at Screening
• Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) > 5 × ULN,
• Significant proteinuria
• Patients with significant impaired gastrointestinal function or gastrointestinal disease
• Clinical or laboratory evidence of active Hepatitis B or Hepatitis C
• Patients with psychiatric or addictive disorders
• Patients with a known history of HIV seropositivity (Elisa or Western blot).
• History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there was an evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin.
• Patients with a history of hypersensitivity to any of the study drug or excipients.
• Patients with significant medical condition that could interfere with the ability to participate in this study
• Patients who were participating in another clinical trial or receiving an investigational drug.
• Patients using prohibited medication,
• Patients with liver disease with severity of Child-Pugh Class B or C.
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they were using effective methods of contraception during dosing of study treatment

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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53 participants in 1 patient group

Deferasirox

Experimental group

Description:

Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient's actual body weight.

Treatment:

Drug: Deferasirox