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

NCT02720536 | Completed Phase 3 Results

• 1 other identifier: CICL670AIC04
• Study Type: interventional
• Target: 53
• Locations: 3 countries
• Sites: 14

Brief Summary

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

Conditions

Chronic Iron Overload

Keywords

adult; pediatric,; anemia.; Myelodysplastic Syndrome; Thalassemia; Film coated tablet; ICL670; MDS; Chelation; Deferasirox; Iron overload

Outcome Measures

Primary Outcomes (8)

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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 Outcomes (2)

• Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12

  Baseline, 6 and 12 months

• Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12

  Baseline, 6 and 12 months

Study Arms (1)

Deferasirox

EXPERIMENTAL

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.

Drug: Deferasirox

Interventions

Deferasirox DRUG

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

Deferasirox

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (14)

Novartis Investigative Site

Vienna, 1140, Austria

Location

Novartis Investigative Site

Goudi-Athens, GR, 115 27, Greece

Location

Novartis Investigative Site

Pátrai, 265 00, Greece

Location

Novartis Investigative Site

Thessaloniki, 54642, Greece

Location

Novartis Investigative Site

Catania, CT, 95125, Italy

Location

Novartis Investigative Site

Cona, FE, 44100, Italy

Location

Novartis Investigative Site

Genova, GE, 16128, Italy

Location

Novartis Investigative Site

Cagliari, ITA, 09121, Italy

Location

Novartis Investigative Site

Lecce, LE, 73100, Italy

Location

Novartis Investigative Site

Milan, MI, 20122, Italy

Location

Novartis Investigative Site

Palermo, PA, 90127, Italy

Location

Novartis Investigative Site

Palermo, PA, 90146, Italy

Location

Novartis Investigative Site

Verona, VR, 37126, Italy

Location

Novartis Investigative Site

Napoli, 80138, Italy

Location

Related Publications (1)

• 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.

  PMID: 32793403 DERIVED

MeSH Terms

Conditions

Anemia; Myelodysplastic Syndromes; Thalassemia; Iron Overload

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

888-778-8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 21, 2016
• First Posted: March 28, 2016
• Study Start: August 16, 2016
• Primary Completion: July 23, 2019
• Study Completion: July 23, 2019
• Last Updated: March 3, 2020
• Results First Posted: March 3, 2020

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will share

Locations

AU (1); IT (10); GR (3)