safety and efficacy of deferasirox film-coated tablets in patients completing study CICL670F2201

Open-label, multicenter, single arm, phase III study to collect additional safety and efficacy data with deferasirox film-coated tablets in patients completing study CICL670F2201

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-000186-23-AT

Enrollment

Registered

2016-05-20

Start date

2016-06-21

Completion date

Unknown

Last updated

2019-12-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

transfusion-dependent thalassemia or myelodysplastic syndrome at very low, low or intermediate (int-1) risk MedDRA version: 20.1 Level: LLT Classification code 10054658 Term: Thalassemia System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10028534 Term: Myelodysplastic syndrome NOS System Organ Class: 100000004864

Interventions

Trade Name: Exjade Product Name: deferasirox Product Code: ICL670 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: DEFERASIROX CAS Number: 201530-41-8 Current Sponsor code: ICL670 Concentr

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Have completed 24-weeks of study treatment as described in the core protocol (CICL670F2201). • Are deemed to be tolerating deferasirox treatment by the investigator. • Provided written informed consent/assent before any study-specific procedures are performed. For pediatric patients, consent will be obtained from parent(s) or legal patient’s representative. Investigators will also obtain assent of patients according to local, regional or national guidelines. Additional inclusion criteria as per main body of the protocol may apply Are the trial subjects under 18? yes Number of subjects for this age range: 6 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 52 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5

Exclusion criteria

Exclusion criteria: The exclusion criteria will follow 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 Additional exclusion criteria may apply.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the overall safety of deferasirox FCT formulation in patients with transfusion-dependent thalassemia or MDS at very low, low or intermediate risk;Secondary Objective: To evaluate efficacy of deferasirox FCT on serum ferritin levels (decrease or maintenance, according to the individual therapeutic goal);Primary end point(s): Number of Participants with Adverse Events as a Measure of Safety and Tolerability;Timepoint(s) of evaluation of this end point: Up to 24 months or 30 days from last dose.	—

Measure

Time frame

Main Objective: To evaluate the overall safety of deferasirox FCT formulation in patients with transfusion-dependent thalassemia or MDS at very low, low or intermediate risk;Secondary Objective: To evaluate efficacy of deferasirox FCT on serum ferritin levels (decrease or maintenance, according to the individual therapeutic goal);Primary end point(s): Number of Participants with Adverse Events as a Measure of Safety and Tolerability;Timepoint(s) of evaluation of this end point: Up to 24 months or 30 days from last dose.

—

Secondary

Measure	Time frame
Secondary end point(s): Absolute and relative change of serum ferritin level overtime;Timepoint(s) of evaluation of this end point: Baseline, up to 24 months or 7 days of last dose	—

Countries

Austria, Greece, Italy

Contacts

Public ContactDrug Regulatory Affairs

Novartis Pharma GmbH

austria.dra@novartis.com+43 1 86657 0

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026