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

Chronic Iron Overload Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02720536
• Other study ID #: CICL670AIC04
• Status: Completed
• Phase: Phase 3
• Start date: August 16, 2016
• Est. completion date: July 23, 2019

Study information

• Verified date: February 2020
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 53
• Est. completion date: July 23, 2019
• Est. primary completion date: July 23, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Chronic Iron Overload
• Iron Overload

Intervention

Drug:
• Deferasirox
Deferasirox FCT will be provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.

Locations

Country	Name	City	State
Austria	Novartis Investigative Site	Vienna
Greece	Novartis Investigative Site	Goudi-Athens	GR
Greece	Novartis Investigative Site	Patras
Greece	Novartis Investigative Site	Thessaloniki
Italy	Novartis Investigative Site	Cagliari	ITA
Italy	Novartis Investigative Site	Catania	CT
Italy	Novartis Investigative Site	Cona	FE
Italy	Novartis Investigative Site	Genova	GE
Italy	Novartis Investigative Site	Lecce	LE
Italy	Novartis Investigative Site	Milano	MI
Italy	Novartis Investigative Site	Napoli
Italy	Novartis Investigative Site	Palermo	PA
Italy	Novartis Investigative Site	Palermo	PA
Italy	Novartis Investigative Site	Verona	VR

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

Austria,  Greece,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overview of Number of Participants With Adverse Events	Numbers represent counts of participants within the categories. An adverse event (AE) was defined as treatment emergent if its onset date is on or after (=) the first administration of study treatment within this study or events present prior to start of study treatment but increased in severity on or after (=) the first administration of study treatment within this study but not later than 30 days after the last study treatment in this study	Baseline up to approximately 25 months
Primary	Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline	Baseline, 6 and 12 months
Primary	Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline	Baseline, 6 and 12 months
Primary	Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline	Baseline, 6 and 12 months
Primary	Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline	Baseline, 6 and 12 months
Primary	Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline	Baseline, 6 and 12 months
Primary	Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline	Baseline, 6 and 12 months
Primary	Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline	Baseline, 6 and 12 months
Secondary	Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12	The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline. A negative change from baseline is regarded as an improvement in this study	Baseline, 6 and 12 months
Secondary	Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12	The percentage relative change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point. A negative percentage relative change from baseline is regarded as an improvement in this study	Baseline, 6 and 12 months