Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome

June 1, 2009 updated by: Office of Rare Diseases (ORD)

Mechanism and Response of Thymoglobulin in Patients With Myelodysplastic Syndrome (MDS)

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

54

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
    • Florida
      • Tampa, Florida, United States, 33612
        • Recruiting
        • H. Lee Moffitt Cancer Center
        • Contact:
          • Tera Uliano, RN
          • Phone Number: 813-745-1706
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic Foundation - Case Western University
        • Contact:
        • Principal Investigator:
          • Jaroslaw P. Maciejewski, MD, PhD
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Recruiting
        • Penn State University
        • Contact:
        • Principal Investigator:
          • Thomas P. Loughran, Jr., MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Bone marrow response and hematologic improvement
Time Frame: Measured at Week 16 or 24
Measured at Week 16 or 24
Bone marrow cytogenetic response
Time Frame: Measured at Week 16 or 24
Measured at Week 16 or 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Office of Rare Diseases (ORD)

Collaborators

Rare Diseases Clinical Research Network

Investigators

  • Principal Investigator: Alan List, MD, H. Lee Moffitt Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2007

Primary Completion (Anticipated)

February 1, 2010

Study Completion (Anticipated)

February 1, 2010

Study Registration Dates

First Submitted

April 25, 2007

First Submitted That Met QC Criteria

April 25, 2007

First Posted (Estimate)

April 27, 2007

Study Record Updates

Last Update Posted (Estimate)

June 2, 2009

Last Update Submitted That Met QC Criteria

June 1, 2009

Last Verified

June 1, 2009

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RDCRN 5406

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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