Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study - Full Text View

Sponsor:	Novartis Pharmaceuticals
Information provided by (Responsible Party):	Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:	NCT00940602

Purpose

The primary purpose of this study is to prospectively assess the efficacy and safety of iron chelation therapy with deferasirox compared to placebo in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload.

Condition	Intervention	Phase
Myelodysplastic Syndromes	Drug: Deferasirox Drug: Deferasirox placebo	Phase III

Study Type:	Interventional
Study Design:	Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment
Official Title:	A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of Deferasirox in Patients With Myelodysplastic Syndromes (Low/Int-1 Risk) and Transfusional Iron Overload

Resource links provided by NLM:

MedlinePlus related topics: Iron Myelodysplastic Syndromes

Drug Information available for: Deferasirox

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

To compare deferasirox to placebo with regard to event-free survival (a composite primary endpoint including death and non-fatal events related to cardiac and liver function) in low and int-1 risk MDS patient with transfusional iron overload. [ Time Frame: 1 year - 5 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To compare overall survival Measure: Survival to date of death [ Time Frame: 1 year-5 years ] [ Designated as safety issue: No ]
To evaluate the change in endocrine function: thyroid Measure: TSH, Free T4 [ Time Frame: Annually ] [ Designated as safety issue: No ]
To evaluate the change in endocrine function: glucose metabolism Measure: Glucose Tolerance Testing [ Time Frame: Annually ] [ Designated as safety issue: No ]
To evaluate the change in MDS progression [ Time Frame: From date of Randomization Up to 5 years ] [ Designated as safety issue: No ]
To evaluate the change in hematological function expressed in frequency/total amount of blood transfusions during trial Measure: milliliters of blood [ Time Frame: From date of Randomization Up to 5 years ] [ Designated as safety issue: No ]

Estimated Enrollment:	630
Study Start Date:	November 2009
Estimated Study Completion Date:	January 2016
Estimated Primary Completion Date:	January 2016 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Placebo Comparator: Deferasirox, iron chelator	Drug: Deferasirox
Placebo Comparator: Placebo	Drug: Deferasirox Drug: Deferasirox placebo

Eligibility

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

Criteria

Inclusion Criteria:

Males or females ≥ 18 years of age MDS low -int-1 risk as determined by IPSS score and confirmed by bone marrow examination within 6 months prior to study entry
Ferritin> 1000 mcg/L and < 3500 mcg/L at screening
History of 20 to 75 PRBC transfusions
Chelation naïve patients
Anticipated to be transfused at least 8 times annually during the study

Exclusion Criteria:

More than 3 years since patient began receiving regular transfusions (2 units per 8 weeks or 4 units received in a 3 mont perio.
Significant proteinuria: urinary protein/creatinine ratio 0.5 mg/mg in a non first void urine sample
ECOG performance > 2
Left ventricular ejection fraction < 55% by ECHO
History of hospitalization for Congestive Heart Failure
Systemic disease that would prevent study treatment (uncontrolled hypertension, cardiovascular renal hepatic or metabolic disease)
Hepatitis B or C (HBsAg in the absences or HBsAB or HCV Ab positive with HCV RNA positive
History of HIV positivity by (ELISA or Western blot)
Treatment with systemic investigational drug within 4 weeks or topical investigational drug within 7 days of study start
ALT or AST > 2.5 x ULN at screening
Total bilirubin > ULN at screening
Diagnosis of liver cirrhosis
Patient participating in another clinical trial or receiving an investigational drug
History of non-compliance with medical regimen, or patients potentially unreliable and/or not cooperative
History of non-compliance with medical regimen, or patients potentially unreliable and/or not cooperative
Pregnant or intending to become pregnant or breast-feeding patents

Other protocol-defined inclusion/exclusion criteria may apply

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00940602

Contacts

Contact: Novartis Pharmaceuticals

800-340-6843

Show 83 Study Locations

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director:

Novartis Pharmaceuticals

More Information

No publications provided

Responsible Party:	Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:	NCT00940602 History of Changes
Other Study ID Numbers:	CICL670A2302, EudraCT: 2009-012418-38
Study First Received:	July 15, 2009
Last Updated:	November 3, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Novartis:

TELESTO
MDS Study
Myelodysplastic Syndromes
Myelodysplastic Syndromes (low-int-1 risk)

Additional relevant MeSH terms:

Myelodysplastic Syndromes
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms

Deferasirox
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on February 09, 2012