Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study

Condition(s) targeted: Myelodysplastic Syndromes

Intervention: Deferasirox (Drug); Deferasirox placebo (Drug)

Phase: Phase 3

Status: Recruiting

Sponsored by: Novartis Pharmaceuticals

Official(s) and/or principal investigator(s):
Novartis Pharmaceuticals, Study Director, Affiliation: Novartis Pharmaceuticals

Overall contact:
Novartis Pharmaceuticals, Phone: 800-340-6843

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.

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

Study design: Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Primary outcome: 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.

Secondary outcome:

To compare overall survival Measure: Survival to date of death

To evaluate the change in endocrine function: thyroid Measure: TSH, Free T4

To evaluate the change in endocrine function: glucose metabolism Measure: Glucose Tolerance Testing

To evaluate the change in MDS progression

To evaluate the change in hematological function expressed in frequency/total amount of blood transfusions during trial Measure: milliliters of blood

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

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

Novartis Pharmaceuticals, Phone: 800-340-6843

Novartis Investigative Site, Herston QLD 4029, Australia; Not yet recruiting

Novartis Investigative Site, Perth WA 6000, Australia; Not yet recruiting

Novartis Investigative Site, Randwick NSW 2031, Australia; Not yet recruiting

Hôpital Enfant-Jésus, Quebec G1J 1Z4, Canada; Not yet recruiting
Yolaine Hebert, Phone: +1-418-649-0252, Ext: 3115, Email: yolaine.hebert.cha@ssss.gouv.qc.ca
Vincent Laroche, M.D., Principal Investigator

Novartis Investigative Site, Herlev 2730, Denmark; Not yet recruiting

Novartis Investigative Site, Kobenhavn 2100, Denmark; Not yet recruiting

Novartis Investigative Site, Chaiwan, Hong Kong; Not yet recruiting

Novartis Investigative Site, Shatin, Hong Kong; Recruiting

Novartis Investigative Site, Bologna 40138, Italy; Not yet recruiting

Novartis Investigative Site, Cagliari 09100, Italy; Not yet recruiting

Novartis Investigative Site, Calabria, Italy; Recruiting

Novartis Investigative Site, Firenze FI, 50134, Italy; Not yet recruiting

Novartis Investigative Site, Rotondo 71013, Italy; Not yet recruiting

Novartis Investigative Site, Selangor, Malaysia; Not yet recruiting

Novartis Investigative Site, Chihuahua 31000, Mexico; Not yet recruiting

Novartis Investigative Site, Cuauhtemoc 06726, Mexico; Not yet recruiting

Novartis Investigative Site, Guanajuato, Mexico; Not yet recruiting

Novartis Investigative Site, Nuevo Leon 64710, Mexico; Not yet recruiting

Novartis Investigative Site, Auckland 0622, New Zealand; Not yet recruiting

Novartis Investigative Site, Auckland, New Zealand; Not yet recruiting

Novartis Investigative Site, Christchurch 8011, New Zealand; Not yet recruiting

Novartis Investigative Site, Petersburg 191024, Russian Federation; Not yet recruiting

Novartis Investigative Site, Petersburg 197022, Russian Federation; Not yet recruiting

Novartis Investigative Site, Basel 4301, Switzerland; Not yet recruiting

Novartis Investigative Site, Bern 3010, Switzerland; Not yet recruiting

Novartis Investigative Site, Luzem 6004, Switzerland; Not yet recruiting

Novartis Investigative Site, Zurich 8091, Switzerland; Not yet recruiting

Novartis Investigative Site, Bangkok 10700, Thailand; Not yet recruiting

Novartis Investigative Site, Bangkok 10330, Thailand; Not yet recruiting

Novartis Investigative Site, Bangkok 10400, Thailand; Not yet recruiting

Novartis Investigative Site, Chang Mai 50200, Thailand; Not yet recruiting

Novartis Investigative Site, Khon Kaen 40002, Thailand; Not yet recruiting

Novartis Investigative Site, Cardiff CF14 4XW, United Kingdom; Not yet recruiting

Novartis Investigative Site, Glasgow G12 OXL, United Kingdom; Not yet recruiting

Novartis Investigative Site, Leeds LS1 3EX, United Kingdom; Not yet recruiting

Novartis Investigative Site, Nottingham NG5 1PB, United Kingdom; Not yet recruiting

Novartis Investigative Site, Oxford, United Kingdom; Not yet recruiting

Novartis Investigative Site, Truro TR1 3LJ, United Kingdom; Not yet recruiting

Novartis Investigative Site, Orbassano, 10043, Italy; Not yet recruiting

University of Alberta, Edmonton, Alberta T6G 2R7, Canada; Not yet recruiting
Vanessa Pierce, Phone: +1-780-407-6090, Email: Vanessa.Pierce@albertahealthservices.ca
Anthony Woods, M.D., Principal Investigator

Novartis Investigative Site, Bournemouth, Bh7 7dw, United Kingdom; Not yet recruiting

Vancouver General Hospital, Vancouver, British Columbia V5Z 1M9, Canada; Not yet recruiting
Jill Clark, Phone: +1-604-875-4111, Ext: 62887, Email: jclark2@bccancer.bc.ca
Thomas Nevill, M.D., Principal Investigator

St. Paul's Hospital, Vancouver, British Columbia V6Z 2A5, Canada; Not yet recruiting
Waldrum Marketa, Phone: +1-604-682-2344, Ext: 62321, Email: mwaldrum@providencehealth.bc.ca
Linda Vickars, M.D., Principal Investigator

UCSD Moores Cancer Center, La Jolla, California 92093-0960, United States; Not yet recruiting
Avideh Rashed, Phone: 858-822-6296, Email: arashed@ucsd.edu
Peter Curtin, M.D., Principal Investigator

Long Beach Veterans Administration Medical Center, Long Beach, California 90822, United States; Not yet recruiting
Sakineh Khalaghizadeh, Phone: 562-826-8000, Ext: 3393, Email: Sakineh.khalaghizadeh@va.gov
Gordon G. McLaren, M.D., Principal Investigator

Stanford University, Stanford, California 94305, United States; Not yet recruiting
Mai Tran, Phone: 650-723-8594, Email: maitran@stanford.edu
Peter Greenberg, M.D., Principal Investigator

University of Chicago Medical Center, Chicago, Illinois 60637, United States; Not yet recruiting
Peggy Peggy, Phone: 773-702-0267, Email: mgreen@medicine.bsd.uchicago.edu
Andrew Artz, M.D., Principal Investigator

Rush University Medical Center, Chicago, Illinois 60612-3824, United States; Not yet recruiting
Jodi Palonis, Phone: 312-942-3327, Email: Jodi_r_palonis@rush.edu
Jamile Shammo, M.D., Principal Investigator

Cancercare Manitoba-Hematology/Oncology/Manitoba Blood and Bone Marrow Program, Winnipeg, Manitoba R3E 0V9, Canada; Not yet recruiting
Elizabeth Lylyk, Phone: +1-204-787-1199, Email: elizabeth.lylyk@cancercare.mb.ca
Rajat Kumar, M.D., Principal Investigator

Hackensack University Medical Center, Hackensack, New Jersey 07601, United States; Not yet recruiting
Sora Limor, RN, Phone: 201-996-5900, Email: slimor@humed.com
Stuart Goldberg, M.D., Principal Investigator

Wake Forest University of Health Sciences, Winston-Salem, North Carolina 27157, United States; Not yet recruiting
Jennifer MacLean, Phone: 336-713-3539, Email: jemaclea@wfubmc.edu
Bayard Powell, M.D., Principal Investigator

William Osler Hospital, Brampton, Ontario L6R 3J7, Canada; Not yet recruiting
Johnson Dartey, Phone: +1-905-494-2120, Ext: 57264, Email: johnson_dartey@oslerhc.org
Sandeep Sendev, M.D., Principal Investigator

Niagara Health System - Oncology Clinic, St. Catharines, Ontario L2R 7C6, Canada; Not yet recruiting
Linda Iles, Phone: +1-905-684-7271, Ext: 43801, Email: linda.illes@niagarahealth.on.ca
Brian Findlay, M.D., Principal Investigator

University of Texas MD Anderson Cancer Center, Houston, Texas 77030, United States; Not yet recruiting
Monica Kwari, Phone: 713-794-1557, Email: mkwari@mdanderson.org
Guillermo Garcia-Manero, M.D., Principal Investigator

Starting date: March 2010
Last updated: July 21, 2010