Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload

Myelodysplastic Syndromes Clinical Trial

Official title:

A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00481143
• Other study ID #: CICL670ADE03
• Status: Completed
• Phase: Phase 4
• First received: May 31, 2007
• Last updated: May 30, 2017
• Start date: May 2007

Study information

• Verified date: November 2016
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to investigate the effects of iron chelation using deferasirox in low and INT-1 risk (referring to the international prognostic scoring system, IPSS) MDS patients who show signs of iron overload due to repeated blood transfusions.

This trial is not recruiting patients in the United States.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. primary completion date: November 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload

• Patients of either gender and age = 18 years

• History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs)

• Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1)

• Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.

Exclusion Criteria:

• Non-transfusion related iron overload

• Treatment with deferasirox (ICL670) before study start

• Patients with a concomitant malignant disease

• Patients with out of range lab values

• History of nephrotic syndrome

• 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 treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days

• Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug.

• Patients with active uncontrolled infectious disease

• Pregnancy or breast feeding

Other protocol-defined inclusion/exclusion criteria may apply.

Related Conditions & MeSH terms

• Iron Overload
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome
• Transfusion Dependent Iron Overload

Intervention

Drug:
• ICL670/Deferasirox

Locations

Country	Name	City	State
Germany	Novartis Investigative Site	Berlin

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Germany, 

References & Publications (1)

Nolte F, Höchsmann B, Giagounidis A, Lübbert M, Platzbecker U, Haase D, Lück A, Gattermann N, Taupitz M, Baier M, Leismann O, Junkes A, Schumann C, Hofmann WK, Schrezenmeier H. Results from a 1-year, open-label, single arm, multi-center trial evaluating t — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox		52 weeks
Secondary	Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs)		52 weeks

External Links

•   Clinical Trial Results for CICL670ADE03 at Novartis Clinical Trials.com