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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


Phase 4
18 Years
N/A
Not Enrolling
Both
Myelodysplastic Syndromes, Transfusion Dependent Iron Overload

Thank you

Trial Information

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


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.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox

Outcome Time Frame:

52 weeks

Safety Issue:

No

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmeceuticals

Authority:

Germany: Federal Institute for Drugs and Medical Devices

Study ID:

CICL670ADE03

NCT ID:

NCT00481143

Start Date:

May 2007

Completion Date:

Related Keywords:

  • Myelodysplastic Syndromes
  • Transfusion Dependent Iron Overload
  • Myelodysplastic Syndrome
  • MDS
  • iron overload
  • chelation
  • chelators
  • ICL670
  • Deferasirox
  • serum ferritin
  • LIC
  • transfusional hemosiderosis
  • Refractory Anemia
  • RA
  • Refractory Anemia with Ringed Sideroblasts
  • RARS
  • blood transfusions
  • Mydelodysplastic Syndrome(s)
  • Myelodysplastic Syndromes
  • Preleukemia
  • Iron Overload

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