Clinical trial NCT00481143: Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Myelodysplastic Syndromes
Transfusion Dependent Iron Overload
Keywords: 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)
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2007-06-01
Enrollment: 63
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox
timeFrame: 52 weeks
Secondary outcomes:
measure: Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs)
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmeceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Nolte F, Hochsmann B, Giagounidis A, Lubbert M, Platzbecker U, Haase D, Luck 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 the efficacy and safety of oral Deferasirox in patients diagnosed with low and int-1 risk myelodysplastic syndrome (MDS) and transfusion-dependent iron overload. Ann Hematol. 2013 Jan;92(2):191-8. doi: 10.1007/s00277-012-1594-z. Epub 2012 Oct 17.