A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload

February 8, 2020 updated by: Novartis Pharmaceuticals

A One Year, Open-label, Single-arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/Day) in Patients Diagnosed With Transfusion-dependent Iron Overload

This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

1784

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Adelaide, Australia
        • Novartis Investigative Site
      • Camperdown, Australia
        • Novartis Investigative Site
      • Clayton, Australia
        • Novartis Investigative Site
      • Melbourne, Australia
        • Novartis Investigative Site
      • Perth, Australia
        • Novartis Investigative Site
      • South Brisbane, Australia
        • Novartis Investigative Site
      • Westmead, Australia
        • Novartis Investigative Site
  • Austria
      • Graz, Austria
        • Novartis Investigative Site
      • Linz, Austria
        • Novartis Investigative Site
      • Wien, Austria
        • Novartis Investigative Site
  • Belgium
      • Brussels, Belgium
        • Novartis Investigative Site
      • Gent, Belgium
        • Novartis Investigative Site
      • Godinne, Belgium
        • Novartis Investigative Site
      • La Louviere, Belgium
        • Novartis Investigative Site
      • Leuven, Belgium
        • Novartis Investigative Site
  • China
      • GuangZhou, China
        • Novartis Investigative Site
      • Nanjing, China
        • Novartis Investigative Site
      • Shanghai, China
        • Novartis Investigative Site
  • Denmark
      • Arhus, Denmark
        • Novartis Investigative Site
      • Copenhagen, Denmark
        • Novartis Investigative Site
      • Herlev, Denmark
        • Novartis Investigative Site
      • Hillerod, Denmark
        • Novartis Investigative Site
  • Egypt
      • Cairo, Egypt
        • Novartis Investigative Site
  • France
      • Angers, France
        • Novartis Investigative Site
      • Avignon, France
        • Novartis Investigative Site
      • Bobigny, France
        • Novartis Investigative Site
      • Creteil, France
        • Novartis Investigative Site
      • Lille Cedex, France
        • Novartis Investigative Site
      • Lyon, France
        • Novartis Investigative Site
      • Nice, France
        • Novartis Investigative Site
      • Paris, France
        • Novartis Investigative Site
      • Paris Cedex 14, France
        • Novartis Investigative Site
      • Pessac Cedex, France
        • Novartis Investigative Site
      • Rennes, France
        • Novartis Investigative Site
      • Toulouse Cedex, France
        • Novartis Investigative Site
      • Vandoeuvre Les Nancy, France
        • Novartis Investigative Site
  • Germany
      • Augsburg, Germany
        • Novartis Investigative Site
      • Braunschweig, Germany
        • Novartis Investigative Site
      • Dresden, Germany
        • Novartis Investigative Site
      • Dusseldorf, Germany
        • Novartis Investigative Site
      • Frankfurt, Germany
        • Novartis Investigative Site
      • Frankfurt/Main, Germany
        • Novartis Investigative Site
      • Frieburg, Germany
        • Novartis Investigative Site
      • Gottingen, Germany
        • Novartis Investigative Site
      • Greifswald, Germany
        • Novartis Investigative Site
      • Hannover, Germany
        • Novartis Investigative Site
      • Mainz, Germany
        • Novartis Investigative Site
      • Muenchen, Germany
        • Novartis Investigative Site
      • Ulm, Germany
        • Novartis Investigative Site
  • Greece
      • Athens, Greece
        • Novartis Investigative Site
      • Larissa, Greece
        • Novartis Investigative Site
      • Patras, Greece
        • Novartis Investigative Site
      • Thessaloniki, Greece
        • Novartis Investigative Site
  • Hong Kong
      • Hong Kong, Hong Kong
        • Novartis Investigative Site
  • Israel
      • Afula, Israel
        • Novartis Investigative Site
      • Jerusalem, Israel
        • Novartis Investigative Site
      • Petach-Tikva, Israel
        • Novartis Investigative Site
  • Italy
      • Bologna, Italy
        • Novartis Investigative Site
      • Brindisi, Italy
        • Novartis Investigative Site
      • Cagliari, Italy
        • Novartis Investigative Site
      • Cona, Italy
        • Novartis Investigative Site
      • Genova, Italy
        • Novartis Investigative Site
      • Milano, Italy
        • Novartis Investigative Site
      • Napoli, Italy
        • Novartis Investigative Site
      • Orbassano, Italy
        • Novartis Investigative Site
      • Palermo, Italy
        • Novartis Investigative Site
      • Pavia, Italy
        • Novartis Investigative Site
      • Pisa, Italy
        • Novartis Investigative Site
      • Reggio Calabria, Italy
        • Novartis Investigative Site
      • Roma, Italy
        • Novartis Investigative Site
      • Sassari, Italy
        • Novartis Investigative Site
  • Korea, Republic of
      • Seoul, Korea, Republic of
        • Novartis Investigative Site
  • Lebanon
      • Hazmiyeh, Lebanon
        • Novartis Investigative Site
  • Malaysia
      • Kota Bahru, Malaysia
        • Novartis Investigative Site
      • Kuala Lumpur, Malaysia
        • Novartis Investigative Site
  • Netherlands
      • Nijmegen, Netherlands
        • Novartis Investigative Site
  • South Africa
      • Johannesburg, South Africa
        • Novartis Investigative Site
      • Parktown, South Africa
        • Novartis Investigative Site
  • Spain
      • Baracaldo, Spain
        • Novartis Investigative Site
      • Barcelona, Spain
        • Novartis Investigative Site
      • Madrid, Spain
        • Novartis Investigative Site
      • Sevilla, Spain
        • Novartis Investigative Site
      • Valencia, Spain
        • Novartis Investigative Site
  • Switzerland
      • Geneve, Switzerland
        • Novartis Investigative Site
      • Zurich, Switzerland
        • Novartis Investigative Site
  • Taiwan
      • Taichung, Taiwan
        • Novartis Investigative Site
      • Taipei, Taiwan
        • Novartis Investigative Site
  • Thailand
      • Bangkok, Thailand
        • Novartis Investigative Site
      • Chaingmai, Thailand
        • Novartis Investigative Site
  • Turkey
      • Adana, Turkey
        • Novartis Investigative Site
      • Ankara, Turkey
        • Novartis Investigative Site
      • Istanbul, Turkey
        • Novartis Investigative Site
      • Izmir, Turkey
        • Novartis Investigative Site
  • United Kingdom
      • Leeds, United Kingdom
        • Novartis Investigative Site
      • Leicester, United Kingdom
        • Novartis Investigative Site
      • London, United Kingdom
        • Novartis Investigative Site
      • Manchester, United Kingdom
        • Novartis Investigative Site
      • Sheffield, United Kingdom
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patients presenting with transfusion-dependent anemias (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of ≥ 1000 ng/ml
  • Patients of either gender and aged ≥ 2 years
  • Female patients who have reached menarche and who are sexually active must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.

Additional Inclusion Criteria for Adult Patients:

  • Written informed consent by the patient

Additional Inclusion Criteria for Pediatric Patients:

  • The definition of the term "pediatric" will be in accordance with local legislation. Parents or legal guardians will be fully informed by the investigator as to the requirements of the study. The pediatric patients themselves will be informed according to their capabilities in a language and terms that they are able to understand. Written informed consent will be obtained from their parents or legal guardians on the patient's behalf in accordance with the national legislation. If capable, all patients should also personally sign their written informed assent.

Exclusion Criteria:

  • Non-transfusional hemosiderosis
  • Patients with clinical evidence supporting the need for intensive chelation, based on the investigator's judgment
  • Patients with mean levels of alanine aminotransferase (ALT) > 300 U/l
  • Patients with uncontrolled systemic hypertension
  • Patients with serum creatinine above the upper limit of normal (ULN)
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 (mg/mg) in second-voiding urine samples taken at both visits 1 and 2. A third sample is to be taken from patients in whom one ratio is > 0.5 (mg/mg) and one is ≤ 0.5 (mg/mg) and patients in whom the urinary protein/creatinine ratio is > 0.5 (mg/mg) in two of the three determinations are also to be excluded.
  • History of nephrotic syndrome
  • Patients with 3rd atrioventricular (A-V) block, clinically relevant Q-T interval prolongation as well as patients requiring treatment with digoxin and similar compounds or drugs which may induce prolongation of the Q-T interval
  • Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
  • Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
  • Pregnant or breast feeding patients
  • Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drugs within the past 7 days

  • Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:

    • history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
    • history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
    • history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase;
    • history or presence of impaired renal function as indicated by creatinine or blood urea nitrogen (BUN) values equal or above ULN;
    • history of urinary obstruction or difficulty in voiding.
  • History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
  • History of drug or alcohol abuse within the 12 months prior to dosing or evidence of such abuse as indicated by the laboratory assays conducted during the run-in period
  • Patients with positive test to HIV
  • Life expectancy of < 1 year

Exclusion Criteria for Pediatric Patients:

  • Patient body weight which prevents the use of the smallest tablet strength (i.e. 125 mg) for proper dosing

Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: ICL670 (Deferasirox)
Drug: Deferasirox
Other Names:
  • ICL670

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To evaluate if fixed starting doses of ICL670, based on transfusion history and subsequent dose titration can provide clinically acceptable chelation as measured by serum ferritin
Time Frame: at baseline and at 52 weeks
at baseline and at 52 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
To evaluate the safety and tolerability profile of in patients treated for up to 52 weeks
Time Frame: Monthly
Monthly
Evaluate efficacy, tolerabilty and safety in the subgroup of patients with baseline LIC < 7 mg Fe/g dw
Time Frame: Monthly
Monthly
Evaluate the relationship between serum ferritin and potential surrogate markers
Time Frame: Monthly
Monthly

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2005

Primary Completion (ACTUAL)

May 1, 2009

Study Completion (ACTUAL)

July 1, 2010

Study Registration Dates

First Submitted

September 13, 2005

First Submitted That Met QC Criteria

September 13, 2005

First Posted (ESTIMATE)

September 15, 2005

Study Record Updates

Last Update Posted (ACTUAL)

February 11, 2020

Last Update Submitted That Met QC Criteria

February 8, 2020

Last Verified

June 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CICL670A2409
  • 2004-003953-16 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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