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Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients
This study has been completed.
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00110266
First received: May 4, 2005
Last updated: November 16, 2016
Last verified: November 2016
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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: | 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 ( Novartis Pharmaceuticals ):
Primary Outcome Measures:
- 12 month safety in patients with myelodysplastic syndrome (MDS) [ Time Frame: throughout the study ]
Secondary Outcome Measures:
- Changes in serum ferritin from baseline to 3, 6, 9 and 12 months after initiating treatment [ Time Frame: throughout the study ]
- Effect of non-transferrin bound iron (NTBI), serum iron, transferrin and transferrin saturation on the safe administration of deferasirox [ Time Frame: throughout the study ]
- 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 ]
- Trough pharmacokinetic parameters of deferasirox in patients with MDS [ Time Frame: throughout the study ]
- Drug accountability [ Time Frame: through out the study ]
| 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:
|
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 (Adult, Senior) |
| Sexes Eligible for Study: | All |
| 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
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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
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:
Publications:
| Responsible Party: | 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: | November 16, 2016 |
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
|
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 Iron Chelating Agents |
Deferasirox Deferoxamine Trace Elements Micronutrients Growth Substances Physiological Effects of Drugs Sequestering Agents Molecular Mechanisms of Pharmacological Action Iron Chelating Agents Siderophores |
ClinicalTrials.gov processed this record on June 23, 2017