DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



Effects of Antithymocyte Globulin in Adults With Myelodysplastic Syndrome

Information source: Office of Rare Diseases (ORD)
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Myelodysplastic Syndrome

Intervention: Antithymocyte globulin (ATG) (Drug); Prednisone (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Office of Rare Diseases (ORD)

Official(s) and/or principal investigator(s):
Alan List, MD, Principal Investigator, Affiliation: H. Lee Moffitt Cancer Center

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.

Clinical Details

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

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

Bone marrow response and hematologic improvement

Bone marrow cytogenetic response

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.

Eligibility

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

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

Locations and Contacts

UCLA Oncology Center, Los Angeles, California 90095, United States; Not yet recruiting
Troy Overfield, Email: toverfield@mednet.ucla.edu
Ronald Paquette, MD, Principal Investigator

H. Lee Moffitt Cancer Center, Tampa, Florida 33612, United States; Recruiting
Tera Uliano, RN, Phone: 813-745-1706
Alan List, MD, Principal Investigator

Cleveland Clinic Foundation - Case Western University, Cleveland, Ohio 44195, United States; Recruiting
Robin Heggeland, RN, Email: heggelr@ccf.org
Jaroslaw P. Maciejewski, MD, PhD, Principal Investigator

Penn State University, Hershey, Pennsylvania 17033, United States; Recruiting
Lynn Ruiz, Email: lruiz@psu.edu
Thomas P. Loughran, Jr., MD, Principal Investigator

Additional Information

Related publications:

Kochenderfer JN, Kobayashi S, Wieder ED, Su C, Molldrem JJ. Loss of T-lymphocyte clonal dominance in patients with myelodysplastic syndrome responsive to immunosuppression. Blood. 2002 Nov 15;100(10):3639-45. Epub 2002 Jul 5.

Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS. Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein-deficient clones. Br J Haematol. 2001 Dec;115(4):1015-22.

Molldrem JJ, Leifer E, Bahceci E, Saunthararajah Y, Rivera M, Dunbar C, Liu J, Nakamura R, Young NS, Barrett AJ. Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med. 2002 Aug 6;137(3):156-63.

Starting date: April 2007
Last updated: June 1, 2009

Page last updated: August 20, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017