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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00171821
First received: September 13, 2005
Last updated: June 5, 2013
Last verified: June 2013
  Purpose

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.


Condition Intervention Phase
Transfusion-dependent Iron Overload
Drug: Deferasirox
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

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

Enrollment: 1784
Study Start Date: April 2005
Study Completion Date: July 2010
Primary Completion Date: May 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ICL670 (Deferasirox) Drug: Deferasirox
Other Name: ICL670

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00171821

  Show 98 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided by Novartis

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00171821     History of Changes
Obsolete Identifiers: NCT00565578
Other Study ID Numbers: CICL670A2409, 2004-003953-16
Study First Received: September 13, 2005
Last Updated: June 5, 2013
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Austria: Ethikkommission
Belgium: Federal Agency for Medicinal Products and Health Products
China: Ministry of Health
Denmark: Danish Medicines Agency
Egypt: Ministry of Health and Population
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Greece: Ministry of Health and Welfare
Hong Kong: Department of Health
Israel: Ministry of Health
Italy: Ministry of Health
Lebanon: Ministry of Public Health
Malaysia: Ministry of Health
Netherlands: Medicines Evaluation Board
South Africa: Department of Health
South Korea: Korea Food and Drug Administration
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Switzerland: Swissmedic
Taiwan : Food and Drug Administration
Thailand: Food and Drug Administration
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Novartis:
Thalassemia
Myelodysplastic Syndrome
MDS
Sickle cell
Diamond-Blackfan anemia
Transfusion
Anemia
Fanconi
Chelation
Deferasirox

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 26, 2014