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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

Description

Brief Summary
The purpose of this study was to evaluate deferasirox and placebo with regard to event-free survival (EFS) (a composite primary endpoint including death and non-fatal events related to cardiac and liver function and transformation to AML) in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload.


Detailed Description
A screening period lasting up to 35 days with two screening visits (Visit 1 and Visit 2 - at least 14 days apart) was used to assess patient eligibility. Eligible patients were randomized in a 2:1 ratio in deferasirox and placebo arms respectively. All patients who were randomized in this study started study treatment at 10 mg/kg/day and could be titrated up to 40 mg/kg/day based on dose modification guidelines. Patients who met a non-fatal event, as specified in the protocol, discontinued from the study treatment and were followed for safety (28 days) and then evaluated with visits every three months if they agreed to move into the post treatment evaluation phase. Subsequent to the evaluation period, or at the end of treatment if a patient and treating physician decided that a patient would not participate in the evaluation period, patients were followed by phone monitoring every six months for their need for iron chelation therapy (ICT) and survival follow-up (OS) until study end. Patients who did not meet a non-fatal event continued study treatment as long as the patient and the treating physician felt it was in the best interest for the patient or until the study terminated. After termination of study treatment, all patients continued to be followed for safety and endpoints during the evaluation period at visits occurring every three months. Subsequent to the evaluation period, or at the end of treatment if a patient and treating physician decided that a patient would not participate in the evaluation period, patients were followed every 6 months for ICTs and OS.

Phase

Phase 3 - a treatment has shown activity against a particular disease, where it is either added to existing treatment or compared to the standard treatment.

Inclusion and Exclusion Criteria

  • Weigh between 35-135 kilograms
  • Low or int-1 risk MDS
  • Ferritin >1000 micrograms/liter at screening
  • History of transfusion of 15 to 75 Packed Red Blood Cells (PRBC) units
  • Anticipated to be transfused with at least 8 units of PRBCs annually during the study
  • Women of child-bearing potential using effective methods of contraception during dosing of study treatment

  • More than 6 months of cumulative ICT (such as daily deferasirox (Exjade

Sites

  • California

    • USC/Kenneth Norris Comprehensive Cancer Center USC, Los Angeles, California, 90033
    • Tower Cancer Research Tower Oncology, Beverly Hills, California, 90211
    • City of Hope Medical Center City of Hope Medical Center, Duarte, California, 91010
    • University of California at Los Angeles UCLA, Los Angeles, California, 90095
    • Long Beach VA Medical Center Dept. of Long Beach VA Med.Ctr, Long Beach, California, 90822
    • Long Beach VA Medical Center Dept. of Long Beach VA Med.Ctr, Long Beach, California, 90822
    • Pacific Cancer Medical Center, Inc. PAC Center, Anaheim, California, 92801
    • Pacific Cancer Medical Center, Inc. PAC Center, Anaheim, California, 92801
    • Comprehensive Cancer Center CCC - Palm Spriings, Palm Springs, California, 92262
    • University of California at San Diego, Moores Cancer Ctr Dept. of MooresCancerCenter(3), San Diego, California, 92103
    • Stanford University Medical Center Division of Hematology 2, Stanford, California, 94304

  • Nevada

    • Comprehensive Cancer Centers of Nevada CCC Nevada US Onc, Las Vegas, Nevada, 89109
    • VA Southern Nevada Healthcare System VASNHS, Las Vegas, Nevada, 89106
    • Southern Nevada Cancer Research Foundation Dept.of So. NV CancerRes.Fdn, Las Vegas, Nevada, 89106

  • Idaho

    • Mountain States Tumor Institute Mountain States, Boise, Idaho, 83712

  • Washington

    • Columbia Basin Hematology CBHO, Kennewick, Washington, 99336
    • Columbia Basin Hematology & Oncology CBHO, Kennewick, Washington, 99336
    • Yakima Valley Memorial Hospital North Star Lodge Cancer Center, Yakima, Washington, 98902
    • Swedish Cancer Institute, Seattle, Washington, 98107

  • Texas

    • Cancer Center of the High Plains, Amarillo, Texas, 79106
    • Texas Cancer Center - Abilene, Abilene, Texas, 79606
    • Hendrick Cancer Center, Abilene, Texas, 79601
    • Cancer Care Centers of South Texas / HOAST CCC of So.TX- MedicalCenter(2), San Antonio, Texas, 78229
    • Cancer Care Centers of South Texas / HOAST CCC of So.TX- MedicalCenter(2), San Antonio, Texas, 78229
    • Cancer Care Centers of South Texas / HOAST CCC of So. TX- San Antonio, San Antonio, Texas, 78229
    • Texas Oncology, P.A. Texas Oncology Lewisville, Fort Worth, Texas, 76104
    • ClinRx Research ClinRx Rearch (2), Denton, Texas, 76210
    • Texas Oncology, P.A. TX Onc, Dallas, Texas, 75251
    • Texas Oncology TX Onc, Dallas, Texas, 75251
    • Texas Oncology, P.A. Presbyterian Hospital (2), Dallas, Texas, 75246
    • US Oncology Central Monitoring, Dallas, Texas, 75246
    • Texas Oncology Presbyterian Hospital (2), Dallas, Texas, 75246
    • South Texas Institute of Cancer South Texas, Corpus Christi, Texas, 78405
    • Tyler Cancer Center Dept.ofTylerCancerCtr., Tyler, Texas, 75702
    • Baylor College of Medicine Baylor College of Medicine (2), Houston, Texas, 77030
    • The Methodist Hospital The Methodist Hospital, Houston, Texas, 77030

  • Montana

    • Glacier View Research Institute - Cancer SC, Kalispell, Montana, 59901
    • Glacier View Research Institute - Cancer SC, Kalispell, Montana, 59901

  • Oklahoma

    • University of Oklahoma Health Sciences Center Univ OK HSC, Oklahoma City, Oklahoma, 73104

  • Iowa

    • Siouxland Hematology-Oncology Associates LLP SHO, Sioux City, Iowa, 51101
    • Siouxland Hematology-Oncology Associates LLP SHO, Sioux City, Iowa, 51101
    • Cedar Valley Medical Specialsts Dept of Oncology, Waterloo, Iowa, 50701
    • University of Iowa Hospitals & Clinics U of Iowa Hosp. & Clinics, Iowa City, Iowa, 52242

  • Kansas

    • Kansas City Cancer Center KCCC - South, Overland Park, Kansas, 66210
    • Kansas City Cancer Center KCCC - South, Overland Park, Kansas, 66210

  • Missouri

    • Midwest Cancer Care Physicians MMCC, Kansas City, Missouri, 64131
    • Mercy Medical Research Institute SC, Manchester, Missouri, 63021
    • Mercy Medical Research Institute SC, Manchester, Missouri, 63021
    • Washington University School of Medicine WUSCM, St. Louis, Missouri, 63110

  • Louisiana

    • Willis-Knighton Cancer Center Dept of Onc, Shreveport, Louisiana, 71103
    • LSU HEALTH SCIENCES CENTER/ LSU SCHOOL OF MEDICINE Feist-Weiller Cancer Center(3), New Orleans, Louisiana, 70115

  • Arkansas

    • Hematology Oncology Services of Arkansas HOSA 2, Little Rock, Arkansas, 72205

  • Minnesota

    • Veterans Medical Center -Minneapolis MVMC, Minneapolis, Minnesota, 55417
    • Essentia Health Duluth Clinic Duluth Clinic, Duluth, Minnesota, 55805

  • Tennessee

    • The West Clinic Dept. of the West Clinic, Memphis, Tennessee, 38120

  • Wisconsin

    • Dean Health System, Madison, Wisconsin, 53717

  • Illinois

    • University of Chicago Medical Center Dept. of U. of Chicago Hosp(3), Chicago, Illinois, 60546
    • Rush University Medical Center Div.ofHematology&Oncology (2), Chicago, Illinois, 60612
    • North Shore University Health System North Shore, Evanston, Illinois, 60201
    • North Shore University Health System NSU, Evanston, Illinois, 60201

  • Michigan

    • Cancer and Hematology Centers of West Michigan, PC Dept of Oncology, Grand Rapids, Michigan, 49546

  • Sarawak

    • Novartis Investigative Site, Kuching, Sarawak, 93586

  • RC

    • Novartis Investigative Site, Reggio Calabria, RC, 89124

  • CT

    • Novartis Investigative Site, Catania, CT, 95124

  • Italy

    • Novartis Investigative Site, Napoli, 80136

  • ME

    • Novartis Investigative Site, Messina, ME, 98125

  • FI

    • Novartis Investigative Site, Firenze, FI, 50134
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