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

Primary Purpose

Chronic Iron Overload

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Deferasirox
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Iron Overload focused on measuring adult, pediatric,, anemia., Myelodysplastic Syndrome, Thalassemia, Film coated tablet, ICL670, MDS, Chelation, Deferasirox, Iron overload

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferasirox

Arm 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.

Outcomes

Primary Outcome Measures

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
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
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
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
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
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
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
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

Secondary Outcome Measures

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
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

Full Information

First Posted
March 21, 2016
Last Updated
February 18, 2020
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02720536
Brief Title
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

2. Study Status

Record Verification Date
February 2020
Overall Recruitment Status
Completed
Study Start Date
August 16, 2016 (Actual)
Primary Completion Date
July 23, 2019 (Actual)
Study Completion Date
July 23, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Iron Overload
Keywords
adult, pediatric,, anemia., Myelodysplastic Syndrome, Thalassemia, Film coated tablet, ICL670, MDS, Chelation, Deferasirox, Iron overload

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
53 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Deferasirox
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Intervention Description
Deferasirox FCT will be provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Primary Outcome Measure Information:
Title
Overview of Number of Participants With Adverse Events
Description
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
Time Frame
Baseline up to approximately 25 months
Title
Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12
Description
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
Time Frame
Baseline, 6 and 12 months
Title
Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12
Description
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
Time Frame
Baseline, 6 and 12 months
Title
Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12
Description
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
Time Frame
Baseline, 6 and 12 months
Title
Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12
Description
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
Time Frame
Baseline, 6 and 12 months
Title
Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12
Description
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
Time Frame
Baseline, 6 and 12 months
Title
Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12
Description
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
Time Frame
Baseline, 6 and 12 months
Title
Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12
Description
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
Time Frame
Baseline, 6 and 12 months
Secondary Outcome Measure Information:
Title
Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12
Description
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
Time Frame
Baseline, 6 and 12 months
Title
Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12
Description
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
Time Frame
Baseline, 6 and 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Novartis Investigative Site
City
Vienna
ZIP/Postal Code
1140
Country
Austria
Facility Name
Novartis Investigative Site
City
Goudi-Athens
State/Province
GR
ZIP/Postal Code
115 27
Country
Greece
Facility Name
Novartis Investigative Site
City
Patras
ZIP/Postal Code
265 00
Country
Greece
Facility Name
Novartis Investigative Site
City
Thessaloniki
ZIP/Postal Code
54642
Country
Greece
Facility Name
Novartis Investigative Site
City
Catania
State/Province
CT
ZIP/Postal Code
95125
Country
Italy
Facility Name
Novartis Investigative Site
City
Cona
State/Province
FE
ZIP/Postal Code
44100
Country
Italy
Facility Name
Novartis Investigative Site
City
Genova
State/Province
GE
ZIP/Postal Code
16128
Country
Italy
Facility Name
Novartis Investigative Site
City
Cagliari
State/Province
ITA
ZIP/Postal Code
09121
Country
Italy
Facility Name
Novartis Investigative Site
City
Lecce
State/Province
LE
ZIP/Postal Code
73100
Country
Italy
Facility Name
Novartis Investigative Site
City
Milano
State/Province
MI
ZIP/Postal Code
20122
Country
Italy
Facility Name
Novartis Investigative Site
City
Palermo
State/Province
PA
ZIP/Postal Code
90127
Country
Italy
Facility Name
Novartis Investigative Site
City
Palermo
State/Province
PA
ZIP/Postal Code
90146
Country
Italy
Facility Name
Novartis Investigative Site
City
Verona
State/Province
VR
ZIP/Postal Code
37126
Country
Italy
Facility Name
Novartis Investigative Site
City
Napoli
ZIP/Postal Code
80138
Country
Italy

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Citations:
PubMed Identifier
32793403
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.
Results Reference
derived

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

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