Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT01033747
First received: October 16, 2009
Last updated: July 18, 2011
Last verified: July 2011
  Purpose

The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.


Condition Intervention Phase
Liver Iron Overload
Drug: Deferasirox
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20 mg/kg/Day of Deferasirox in Comparison With 40 mg/kg/Day Deferoxamine in Patients With Transfusion-dependent Iron Overload

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox [ Time Frame: Baseline to 7 Years ] [ Designated as safety issue: No ]
    The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.


Secondary Outcome Measures:
  • Relative Change in Serum Ferritin From Baseline to 3.5 Years [ Time Frame: Baseline to 3.5 years ] [ Designated as safety issue: No ]
    The mean percentage change in serum ferritin was evaluated by comparing the serum ferritin level at the start of Deferasirox treatment to the serum ferritin level collected 18 months following the start of the extension study. Serum ferritin is measured in micrograms per Liter. Relative Change = 1- (Change in ferritin level from Baseline/Baseline level) x 100.


Enrollment: 70
Study Start Date: February 2003
Study Completion Date: January 2008
Primary Completion Date: January 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox
Deferasirox group consists of all participants who were initially randomized to 10 and 20 mg/kg/day deferasirox orally daily in the main study and remained on the same treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative study
Drug: Deferasirox
10 mg/kg or 30 mg/kg orally daily
Experimental: Deferasirox Crossover
Deferasirox Crossover group consists of participants who were initially randomized to 40 mg/kg/day deferoxamine (DFO)subcutaneously in the main study and comparative prolongation study and crossed over to 5mg/kg/day to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study
Drug: Deferasirox
5 mg/kg or 30 mg/kg orally daily

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients Currently participating in the 9-month comparative prolongation of extension phase of the original study.
  • Patients currently participating in the food-effect sub-study, according to amendment 3.
  • Ability to provide written informed consent prior to participation in this non-comparative extension study.
  • Female patients sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
  • Body weight of at least 35 kg.

Exclusion Criteria:

  • Pregnant or breastfeeding patients.
  • History of non-compliance to medical regimens and patients who are considered potentially unreliable.
  • Proteinuria > 300 mg/L second void morning urine.
  • Patients with serum creatinine above the upper limit normal.

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: NCT01033747

Locations
Italy
Novartis Investigative Site
Cagliari, Italy
Novartis Investigative Site
Genova, Italy
Novartis Investigative Site
Milan, Italy
Novartis Investigative Site
Torino, Italy
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01033747     History of Changes
Other Study ID Numbers: CICL670A0105E2
Study First Received: October 16, 2009
Results First Received: December 21, 2010
Last Updated: July 18, 2011
Health Authority: United States: Food and Drug Administration
Italy: Ministry of Health

Keywords provided by Novartis:
iron overload
iron chelation therapy
B-thalassemia

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

ClinicalTrials.gov processed this record on July 23, 2014