Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00466843
Recruitment Status : Unknown
Verified June 2009 by Office of Rare Diseases (ORD).
Recruitment status was:  Recruiting
First Posted : April 27, 2007
Last Update Posted : June 2, 2009
Sponsor:
Collaborator:
Rare Diseases Clinical Research Network
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:
Myelodysplastic syndrome (MDS) is a rare, potentially serious bone marrow disease. Currently available treatments for MDS have been only somewhat beneficial. The purpose of this study is to determine the effects of the medication antithymocyte globulin (ATG) in adults with MDS and to determine which individuals with MDS are most likely to benefit from treatment with ATG.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Drug: Antithymocyte globulin (ATG) Drug: Prednisone Phase 2

Detailed Description:

In people with MDS, the bone marrow stops making healthy blood cells and instead produces poorly functioning, malformed, and immature blood cells. This can lead to anemia resulting from too few healthy red blood cells, infection resulting from too few healthy white blood cells, and bleeding resulting from too few healthy platelets. The exact cause of MDS remains unknown, but it may be caused by abnormal autoimmune activity in which activated T cells, a type of white blood cell, prevent normal bone marrow production. ATG, a medication that inhibits immune function, can restore normal blood production in some people with MDS, but it is not known how this happens and why it does not happen in all MDS patients. The purpose of this study is to examine the effects of ATG in adults with MDS and to determine which individuals with MDS are most likely to benefit from treatment with ATG.

Based on disease severity and likely disease progression, participants will be separated into either a high-risk group or a low-risk group. Participants will be hospitalized for a 4-day period during which they will receive daily infusions of ATG. Oral prednisone will be given 2 days before hospitalization, throughout hospitalization, and then for 14 days after hospitalization to limit the side effects of ATG. Antihistamines and acetaminophen will also be given during hospitalization to reduce the chances of an allergic reaction to ATG. After discharge, all participants will attend monthly study visits that will include blood collection, review of disease symptoms, and evaluation of medication response. At Week 16, participants in the high-risk group will undergo additional blood collection, a bone marrow biopsy, and a thorough evaluation of disease progression and the effects of MDS on daily living abilities. Participants in the low-risk group will undergo these same procedures at Week 24. Follow-up for all participants may last up to 2 years.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Mechanism and Response of Thymoglobulin in Patients With Myelodysplastic Syndrome (MDS)
Study Start Date : April 2007
Estimated Primary Completion Date : February 2010
Estimated Study Completion Date : February 2010


Arm Intervention/treatment
Experimental: 1
Participants will be treated with ATG
Drug: Antithymocyte globulin (ATG)
ATG 2.5 mg/kg/day via IV will be given for 4 doses. Each participant will receive only one cycle of therapy. The daily infusion will be administered over at least 6 hours and slowed as necessary to minimize infusion-related symptoms.

Drug: Prednisone
All participants will be pre-treated with prednisone (1 mg/kg/day by mouth) 2 days prior to the first ATG does and continuing for 14 days after the final dose to prevent serum sickness




Primary Outcome Measures :
  1. Bone marrow response and hematologic improvement [ Time Frame: Measured at Week 16 or 24 ]
  2. Bone marrow cytogenetic response [ Time Frame: Measured at Week 16 or 24 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of MDS that meets International Prognostic Scoring System (IPSS) criteria for low risk, intermediate-1 risk, or intermediate-2 risk. More information about this criterion can be found in the protocol.
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
  • Willing and able to attend study visits
  • Willing to use acceptable forms of contraception prior to study entry and for the duration of the study

Exclusion Criteria:

  • Any serious medical illness that might limit survival to less than 2 years
  • Any other uncontrolled condition or illness. More information about this criterion can be found in the protocol.
  • Prior anti-lymphocyte serotherapy (received serum from an immunized animal)
  • Proliferative chronic myelomonocytic leukemia
  • MDS that is caused by radiotherapy, chemotherapy, and/or immunotherapy for cancerous or autoimmune diseases
  • Previous or current cancer. More information about this criterion can be found in the protocol.
  • Receiving any other investigational agents
  • Certain abnormal lab values. More information about this criterion can be found in the protocol.
  • History of a grade 2 National Cancer Institute common toxic criteria allergic reaction to rabbit proteins
  • Psychiatric illness that might interfere with study participation
  • HIV-1 infection
  • Pregnancy or breastfeeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00466843


Locations
Layout table for location information
United States, California
UCLA Oncology Center Not yet recruiting
Los Angeles, California, United States, 90095
Contact: Troy Overfield       toverfield@mednet.ucla.edu   
Principal Investigator: Ronald Paquette, MD         
United States, Florida
H. Lee Moffitt Cancer Center Recruiting
Tampa, Florida, United States, 33612
Contact: Tera Uliano, RN    813-745-1706      
Principal Investigator: Alan List, MD         
United States, Ohio
Cleveland Clinic Foundation - Case Western University Recruiting
Cleveland, Ohio, United States, 44195
Contact: Robin Heggeland, RN       heggelr@ccf.org   
Principal Investigator: Jaroslaw P. Maciejewski, MD, PhD         
United States, Pennsylvania
Penn State University Recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Lynn Ruiz       lruiz@psu.edu   
Principal Investigator: Thomas P. Loughran, Jr., MD         
Sponsors and Collaborators
Office of Rare Diseases (ORD)
Rare Diseases Clinical Research Network
Investigators
Layout table for investigator information
Principal Investigator: Alan List, MD H. Lee Moffitt Cancer Center
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Alan List, MD, H. Lee Moffitt Cancer Center
ClinicalTrials.gov Identifier: NCT00466843    
Other Study ID Numbers: RDCRN 5406
First Posted: April 27, 2007    Key Record Dates
Last Update Posted: June 2, 2009
Last Verified: June 2009
Keywords provided by Office of Rare Diseases (ORD):
Abnormal hematopoiesis
Leukemia
Autoimmune Disease
Additional relevant MeSH terms:
Layout table for MeSH terms
Preleukemia
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Prednisone
Antilymphocyte Serum
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents