Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00110266
First received: May 4, 2005
Last updated: July 12, 2012
Last verified: July 2012
  Purpose

The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.


Condition Intervention Phase
Myelodysplastic Syndrome
Iron Overload
Drug: Deferasirox
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-risk and INT-1, Myelodysplastic Patients Using Serum Ferritin Monitoring

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 12 month safety in patients with myelodysplastic syndrome (MDS) [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Changes in serum ferritin from baseline to 3, 6, 9 and 12 months after initiating treatment [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
  • Effect of non-transferrin bound iron (NTBI), serum iron, transferrin and transferrin saturation on the safe administration of deferasirox [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
  • Changes in transfusion requirements, serum erythropoietin levels, and estimated frequency of hematologic improvement in patients who are not receiving growth factors or chemotherapy for their underlying MDS. [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
  • Trough pharmacokinetic parameters of deferasirox in patients with MDS [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
  • Drug accountability [ Time Frame: through out the study ] [ Designated as safety issue: No ]

Enrollment: 176
Study Start Date: May 2005
Study Completion Date: March 2010
Primary Completion Date: March 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ICL670
Evaluate the safety and tolerability of deferasirox 20 mg/kg/day over one year in patients with MDS
Drug: Deferasirox
20 mg/kg/day over one year in patients with MDS
Other Names:
  • ICL670A
  • chelator
  • desferal
  • iron chelation

Detailed Description:

Study entry requires a diagnosis of low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria and serum ferritin ≥ 1000 ng/mL. Patients must have had at least 30 prior red blood cell transfusions. Deferasirox will be administered at an initial dose of 20 mg/kg orally once per day. Patient transfusion history and at least three complete blood count (CBC) values must be available for the 12 weeks prior to study registration for patients with MDS and chronic iron overload from blood transfusions.

  Eligibility

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

Inclusion Criteria:

  • Male or female patients with low or intermediate (INT-1) risk MDS
  • Patients can be EITHER naïve to iron chelation OR have had prior treatment with deferoxamine (DFO).
  • Age greater than or equal to 18 years
  • Availability of transfusion records for the 12 weeks prior to registration
  • A lifetime minimum of 30 previous packed red blood cell transfusions
  • Availability of at least three CBC values (pretransfusion) during the 12 weeks prior to registration
  • Serum Ferritin:

For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two occasions, at least two weeks apart, during the prior year.

Serum ferritin ≥ 1000 ng/mL at screening via the central lab.

  • Life expectancy ≥ 6 months
  • Sexually active women must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months)
  • Able to provide written informed consent

Exclusion Criteria:

  • Serum creatinine above the upper limit of normal
  • ALT > 500 U/L during screening
  • Clinical or laboratory evidence of active Hepatitis B or C
  • Urinary protein/creatinine ratio > 0.5 mg/mg
  • History of HIV positive test result (ELISA or Western blot)
  • ECOG Performance Status > 2
  • Patients with uncontrolled systemic hypertension
  • Unstable cardiac disease not controlled by standard medical therapy
  • Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
  • Pregnancy or breast feeding
  • Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
  • Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
  • History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
  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: NCT00110266

  Show 48 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Additional Information:
No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00110266     History of Changes
Obsolete Identifiers: NCT00343837
Other Study ID Numbers: CICL670AUS03
Study First Received: May 4, 2005
Last Updated: July 12, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
ICL670
Deferasirox
Iron chelation
Chelator
Desferal

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Iron Overload
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Iron Metabolism Disorders
Metabolic Diseases
Chelating Agents
Deferasirox
Iron
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Trace Elements
Micronutrients
Growth Substances
Physiological Effects of Drugs
Iron Chelating Agents

ClinicalTrials.gov processed this record on July 24, 2014