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| Sponsors and Collaborators: |
Office of Rare Diseases (ORD) Rare Diseases Clinical Research Network |
|---|---|
| Information provided by: | Office of Rare Diseases (ORD) |
| ClinicalTrials.gov Identifier: | NCT00466843 |
Purpose
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 | Intervention | Phase |
|---|---|---|
|
Myelodysplastic Syndrome |
Drug: Antithymocyte globulin (ATG) Drug: Prednisone |
Phase II |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | Mechanism and Response of Thymoglobulin in Patients With Myelodysplastic Syndrome (MDS) |
| Estimated Enrollment: | 54 |
| Study Start Date: | April 2007 |
| Estimated Study Completion Date: | February 2010 |
| Estimated Primary Completion Date: | February 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Experimental
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
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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.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| 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 | |
| Principal Investigator: | Alan List, MD | H. Lee Moffitt Cancer Center |
More Information
| Responsible Party: | H. Lee Moffitt Cancer Center ( Alan List, MD ) |
| Study ID Numbers: | RDCRN 5406 |
| Study First Received: | April 25, 2007 |
| Last Updated: | September 2, 2008 |
| ClinicalTrials.gov Identifier: | NCT00466843 |
| Health Authority: | United States: Federal Government |
|
Abnormal hematopoiesis Leukemia Autoimmune Disease |
|
Antilymphocyte Serum Myelodysplastic syndromes Prednisone Leukemia Preleukemia Autoimmune Diseases |
Precancerous Conditions Hematologic Diseases Myelodysplasia Myelodysplastic Syndromes Bone Marrow Diseases |
|
Anti-Inflammatory Agents Disease Immunologic Factors Antineoplastic Agents, Hormonal Antineoplastic Agents Physiological Effects of Drugs Hormones, Hormone Substitutes, and Hormone Antagonists Immunosuppressive Agents |
Glucocorticoids Hormones Pharmacologic Actions Neoplasms Pathologic Processes Therapeutic Uses Syndrome |
