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Antithymocyte Globulin Compared With Supportive Care in Treating Patients With Myelodysplastic Syndrome

Information source: National Cancer Institute (NCI)
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Myelodysplastic Syndromes

Intervention: anti-thymocyte globulin (Biological)

Phase: Phase 2

Status: Active, not recruiting

Sponsored by: Sangstat Medical Corporation

Official(s) and/or principal investigator(s):
Elizabeth C. Squiers, MD, Study Chair, Affiliation: Sangstat Medical Corporation


RATIONALE: Immunosuppressive therapy may improve bone marrow abnormalities and may be effective treatment for myelodysplastic syndrome. It is not yet known whether immunosuppressive therapy is more effective than supportive care in treating myelodysplastic syndrome. PURPOSE: Randomized phase II trial to compare the effectiveness of antithymocyte globulin with that of supportive care in treating patients who have myelodysplastic syndrome.

Clinical Details

Official title: An Open Label, Prospective, Stratified, Randomized, Controlled, Multi-Center, Phase IIB Study of the Impact of Thymoglobulin Therapy on Transfusion Needs of Patients With Early Myelodysplastic Syndrome (MDS)

Study design: Allocation: Randomized, Masking: Open Label, Primary Purpose: Treatment

Detailed description: OBJECTIVES:

- Compare the clinical response rate of patients with early myelodysplastic syndrome

treated with rabbit anti-thymocyte globulin vs standard supportive care.

- Evaluate the safety of anti-thymocyte globulin in these patients.

- Compare the time to and duration of clinical response, rates of partial response and

therapy failure, and rate of disease progression in patients treated with these regimens.

- Compare the ECOG performance score, number of transfusions and/or growth factor use,

and maximum time between transfusions in patients treated with these regimens.

- Compare the infection risk, use of medical resources, and quality of clinical response

in patients treated with these regimens. OUTLINE: This is a randomized, open-label, multicenter study. Patients are stratified according to myelodysplastic syndrome (MDS) subtype (refractory anemia (RA) vs RA with excess blasts or hypocellular MDS). Patients are randomized to 1 of 2 treatment arms.

- Arm I: Patients receive rabbit anti-thymocyte globulin (ATG) IV over at least 8-12

hours on days 1-4.

- Arm II: Patients receive standard supportive therapy for 6 months. At the end of 6

months, patients may receive ATG as in arm I. Patients are followed for 6 months. PROJECTED ACCRUAL: A total of 72 patients (48 in arm I and 24 in arm II) will be accrued within a minimum of 6 months.


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



- Histologically or cytologically confirmed early myelodysplastic syndrome (MDS) with

less than 10% bone marrow blasts

- Refractory anemia (RA)

- RA with excess blasts (RAEB)

- Hypocellular myelodysplasia

- Low or intermediate-1 prognostic risk

- Transfusion-dependent

- Need for 2 or more units of red blood cells or platelets per month for 2 or more

months prior to study OR

- History of prior transfusions and 2 consecutive (at least 21 days apart)

hemoglobin levels less than 8. 0 g/dL or platelet counts less than 20,000/mm^3 during the past 2 months

- Hemoglobin no greater than 12. 0 g/dL after prior transfusion

- No myelosclerosis occupying more than 30% of bone marrow space

- No RA with ringed sideroblasts, RAEB in transformation, or chronic myelomonocytic


- No therapy-related MDS

- No history of immune-related hematologic disorder (e. g., idiopathic thrombocytopenic


- 18 and over

Performance status:

- ECOG 0-2

Life expectancy:

- At least 3 months


- See Disease Characteristics

- No other causes of cytopenia unrelated to MDS (e. g., gastrointestinal blood loss)

- Iron present on marrow examination OR

- Transferrin saturation at least 20% and ferritin at least 50 ng/mL


- Bilirubin no greater than 2 mg/dL OR

- SGOT/SGPT no greater than 2 times normal

- No active or chronic hepatitis B or C


- Creatinine no greater than 2 mg/dL


- No symptomatic cardiac disease

- No congestive heart failure (even if medically controlled)

- No myocardial infarction within the past 6 months


- No severe pulmonary disease

- If history of pulmonary insufficiency, must have pO_2 at least 90 mm/Hg on room air

or pCO_2 no greater than 40 mm/Hg Other:

- No history of unresolved B12 or folate deficiency since diagnosis of MDS

- No untreated acute or chronic infection (afebrile for 7 days without antibiotics

prior to study)

- No active or chronic HIV

- No concurrent cytomegalovirus infection

- No other malignancy within the past 2 years except adequately treated localized

squamous cell or basal cell skin cancer or carcinoma in situ of the cervix

- No concurrent drug or alcohol abuse

- No significant medical or psychosocial problems

- No known allergy to rabbit protein

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception


- At least 8 weeks since prior biologic agents, colony-stimulating factors, or epoetin

alfa for MDS

- At least 8 weeks since other prior investigational biologic agents

- No prior or concurrent bone marrow transplantation

- No concurrent epoetin alfa

- No concurrent growth factors except filgrastim (G-CSF) or sargramostim (GM-CSF) for

neutropenic fevers

- No other concurrent biologic agents


- At least 8 weeks since prior cytotoxic drugs for MDS

- Concurrent chemotherapy for clinical indications of disease progression or leukemic

transformation allowed Endocrine therapy:

- At least 8 weeks since prior androgenic hormonal therapy for MDS

- At least 8 weeks since prior danazol for MDS


- No prior radiotherapy


- No prior organ transplantation


- At least 8 weeks since prior investigational drugs

- At least 8 weeks since prior immunosuppressive drugs or other drugs for MDS

- No concurrent immunosuppressive therapy

- No other concurrent experimental drugs

Locations and Contacts

Foothills Hospital, Calgary, Alberta T2N 2T9, Canada

Department of Medicine, Vancouver, British Columbia V5Z 4E3, Canada

Washington Cancer Institute, Washington, District of Columbia 20010, United States

University of Florida Health Science Center, Gainesville, Florida 32610-0296, United States

Sylvester Cancer Center, University of Miami, Miami, Florida 33136, United States

H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida 33612-9497, United States

Veterans Affairs Medical Center - Tampa (Haley), Tampa, Florida 33612, United States

Winship Cancer Institute of Emory University, Atlanta, Georgia 30322, United States

Rush Cancer Institute, Chicago, Illinois 60612, United States

Indiana Blood and Marrow Transplant, Beech Grove, Indiana 46107, United States

Holden Comprehensive Cancer Center, Iowa City, Iowa 52242-1009, United States

University of Kansas Medical Center, Kansas City, Kansas 66160-7357, United States

Tulane University School of Medicine, New Orleans, Louisiana 70112, United States

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland 21231-2410, United States

University of Missouri Kansas City School of Medicine, Kansas City, Missouri 64111, United States

Saint Louis University Cancer Center, Saint Louis, Missouri 63110-2539, United States

Siteman Cancer Center, Saint Louis, Missouri 63110, United States

University of Nebraska Medical Center, Omaha, Nebraska 68198-7680, United States

Hackensack University Medical Center, Hackensack, New Jersey 07601, United States

Mount Sinai Medical Center, NY, New York, New York 10029, United States

New York Presbyterian Hospital - Cornell Campus, New York, New York 10021, United States

James P. Wilmot Cancer Center, Rochester, New York 14642, United States

New York Medical College, Valhalla, New York 10595, United States

Comprehensive Cancer Center at Wake Forest University, Winston-Salem, North Carolina 27157-1082, United States

Cleveland Clinic Taussig Cancer Center, Cleveland, Ohio 44195, United States

Princess Margaret Hospital, Toronto, Ontario M5G 2M9, Canada

Texas Oncology P.A., Dallas, Texas 75230-2503, United States

Medical College of Wisconsin, Milwaukee, Wisconsin 53226-3596, United States

Additional Information

Clinical trial summary from the National Cancer Institute's PDQ® database

Starting date: August 2001
Last updated: February 6, 2009

Page last updated: August 20, 2015

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