An Expanded Access Program for Decitabine in Patients With Myelodysplastic Syndrome (MDS)

The purpose of this study is to provide Decitabine to patients with Myelodysplastic syndrome (MDS) of all FAB (French-American-British) subtypes and Intermediate-1, Intermediate-2, and High-Risk International Prognostic Scoring System groups, including both previously treated and untreated patients.

Study Overview

• Status: No longer available
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: decitabine; Drug: decitabine

Detailed Description

Myelodysplastic Syndromes (MDS), which includes a diverse group of bone marrow disorders that result in ineffective production of blood cells, frequently occurs in elderly patients. Historically, the available treatments for MDS have been symptomatic and supportive, and have not been shown to be effective in producing sustained improvement in hematopoiesis (production of all types of blood cells ) or in delaying leukemic evolution (leukemia is a serious disease in which too many white blood cells are produced, causing weakness and sometimes death). This project is an open-label (all people involved know the identity of the intervention), multicenter, international single arm, Phase 3b study to provide expanded access to Decitabine for patients with myelodysplastic syndromes (MDS). The purpose of this study is to provide Decitabine to patients with Myelodysplastic syndrome (MDS) according some medical classifications: FAB (French-American-British) subtypes and Intermediate-1, Intermediate-2, and High-Risk International Prognostic Scoring System groups, including previously treated and untreated for MDS. The secondary objectives are to evaluate the safety and tolerability of Decitabine, as well as the overall response rate according to the International Working Group (IWG) 2000 and IWG 2006 response criteria, hematologic improvement, cytogenetic response rates (evaluation based on genetic), time to acute myeloid leukemia progression or death (evaluate the length of time that passes prior to onset of leukemia progression and/or death), blood product transfusion requirements per patient (with corresponding dates to collect the number of transfusion independent days), days in the hospital (including reason for hospitalization and the ward within the hospital where the hospitalizations occur) and, optionally, quality of life assessment (EORTC QLQ C-30). 3-day cycle: Decitabine will be administered as a 15mg/m2 administered by continuous infusion over 3 hours repeated every 8 hours for 3 days. 5-day cycle: Another optional schedule could be adopted (at discretion of investigators), Decitabine at a dose of 20mg/m² administered intravenously over 1 hour, once daily for 5 consecutive days, of a 4-week cycle. Treatment may be continued as long as the patient continues to benefit.

• Study Type: Expanded Access

Contacts and Locations

Study Locations

• Brazil
  • Belo Horizonte, Brazil
  • Botucatu, Brazil
  • Brasilia, Brazil
  • Campinas, Brazil
  • Fortaleza, Brazil
  • Pinheiros, Brazil
  • Rio De Janeiro, Brazil
  • Sao Paulo, Brazil
  • São Paulol, Brazil
• Hong Kong
  • Hong Kong, Hong Kong
• Thailand
  • Bangkok, Thailand

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Must have a diagnosis of MDS (de novo / primary or secondary) of all FAB subtypes and Intermediate-1, Intermediate-2, and High-Risk International Prognostic Scoring System groups
• Has an Eastern Oncology Cooperative Group (ECOG) performance status of 0-2
• Adequate hepatic and renal function as measured by the following pre-treatment laboratory criteria within 21 days of starting treatment with Decitabine (laboratory measure of liver function no more than 2.5 times the upper limit of normal, laboratory measure of total bilirubin and serum creatinine no more than 1.5 times the upper limit of normal)
• Female patients must be postmenopausal, or surgically sterile, or abstinent, or, if sexually active, be practicing an effective method of birth control (e.g., prescription oral contraceptives, contraceptive injections, intrauterine device, double-barrier method, contraceptive patch, male partner sterilization) before entry and throughout the study
• Have a negative serum or urine pregnancy test at screening
• Male subjects should be advised not father a child while on or within 2 months of completion of Decitabine therapy

Exclusion Criteria:

• Have a diagnosis of AML (>20% bone marrow blasts) or other progressive malignant disease
• has previously been treated with Azacitidine or Decitabine
• Have uncontrolled heart disease or uncontrolled congestive heart failure
• Have uncontrolled restrictive or obstructive pulmonary disease
• Have active viral or bacterial infection
• Have known positive serology for HIV
• Have a mental illness or any other condition that could prevent full cooperation with the treatment and monitoring requirements of the study
• Have known hypersensitivity to any of the excipients of decitabine
• Are pregnant or breast-feeding

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Janssen-Cilag Farmaceutica Ltda.
• Collaborators: Janssen Korea, Ltd., Korea

Study record dates

Study Major Dates

• Study Start: June 1, 2008
• Primary Completion (Actual): November 1, 2011
• Study Completion (Actual): November 1, 2011

Study Registration Dates

• First Submitted: December 3, 2009
• First Submitted That Met QC Criteria: December 3, 2009
• First Posted (Estimate): December 4, 2009

Study Record Updates

• Last Update Posted (Estimate): April 18, 2013
• Last Update Submitted That Met QC Criteria: April 17, 2013
• Last Verified: April 1, 2013

More Information

Terms related to this study

• Keywords: Decitabine; Myelodysplastic Syndromes
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Preleukemia; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Decitabine
• Other Study ID Numbers: CR015037; DACOGENMYE3001 (Other Identifier: Janssen-Cilag Farmaceutica Ltda.)