Clinical trial NCT02720536: Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Chronic Iron Overload
Keywords: adult
pediatric,
anemia.
Myelodysplastic Syndrome
Thalassemia
Film coated tablet
ICL670
MDS
Chelation
Deferasirox
Iron overload
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2016-03-28
Enrollment: 53
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility 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

Primary outcomes:
measure: Overview of Number of Participants With Adverse Events
timeFrame: Baseline up to approximately 25 months
measure: Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12
timeFrame: Baseline, 6 and 12 months
measure: Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12
timeFrame: Baseline, 6 and 12 months
measure: Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12
timeFrame: Baseline, 6 and 12 months
measure: Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12
timeFrame: Baseline, 6 and 12 months
measure: Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12
timeFrame: Baseline, 6 and 12 months
measure: Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12
timeFrame: Baseline, 6 and 12 months
measure: Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12
timeFrame: Baseline, 6 and 12 months
Secondary outcomes:
measure: Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12
timeFrame: Baseline, 6 and 12 months
measure: Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12
timeFrame: Baseline, 6 and 12 months
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Novartis Investigative Site
city: Goudi-Athens
country: Greece
facility: Novartis Investigative Site
state: GR
city: Patras
country: Greece
facility: Novartis Investigative Site
city: Thessaloniki
country: Greece
facility: Novartis Investigative Site
city: Catania
country: Italy
facility: Novartis Investigative Site
state: CT
city: Cona
country: Italy
facility: Novartis Investigative Site
state: FE
city: Genova
country: Italy
facility: Novartis Investigative Site
state: GE
city: Cagliari
country: Italy
facility: Novartis Investigative Site
state: ITA
city: Lecce
country: Italy
facility: Novartis Investigative Site
state: LE
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Palermo
country: Italy
facility: Novartis Investigative Site
state: PA
city: Palermo
country: Italy
facility: Novartis Investigative Site
state: PA
city: Verona
country: Italy
facility: Novartis Investigative Site
state: VR
city: Napoli
country: Italy
facility: Novartis Investigative Site
Study officials:
References:
citation: Tartaglione I, Origa R, Kattamis A, Pfeilstocker M, Gunes S, Crowe S, Fagan N, Vincenzi B, Ruffo GB. Two-year long safety and efficacy of deferasirox film-coated tablets in patients with thalassemia or lower/intermediate risk MDS: phase 3 results from a subset of patients previously treated with deferasirox in the ECLIPSE study. Exp Hematol Oncol. 2020 Aug 10;9:20. doi: 10.1186/s40164-020-00174-2. eCollection 2020.