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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)


Phase 2
18 Years
N/A
Open (Enrolling)
Both
Myelodysplastic Syndromes

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Trial Information

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)


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.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- 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
leukemia

- No therapy-related MDS

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

PATIENT CHARACTERISTICS:

Age:

- 18 and over

Performance status:

- ECOG 0-2

Life expectancy:

- At least 3 months

Hematopoietic:

- 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

Hepatic:

- Bilirubin no greater than 2 mg/dL OR

- SGOT/SGPT no greater than 2 times normal

- No active or chronic hepatitis B or C

Renal:

- Creatinine no greater than 2 mg/dL

Cardiovascular:

- No symptomatic cardiac disease

- No congestive heart failure (even if medically controlled)

- No myocardial infarction within the past 6 months

Pulmonary:

- 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

PRIOR CONCURRENT THERAPY:

Biologic therapy:

- 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

Chemotherapy:

- 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

Radiotherapy:

- No prior radiotherapy

Surgery:

- No prior organ transplantation

Other:

- 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

Type of Study:

Interventional

Study Design:

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

Principal Investigator

Elizabeth C. Squiers, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Sangstat Medical Corporation

Authority:

United States: Federal Government

Study ID:

CDR0000068709

NCT ID:

NCT00017550

Start Date:

August 2001

Completion Date:

Related Keywords:

  • Myelodysplastic Syndromes
  • refractory anemia
  • refractory anemia with excess blasts
  • de novo myelodysplastic syndromes
  • previously treated myelodysplastic syndromes
  • Myelodysplastic Syndromes
  • Preleukemia

Name

Location

H. Lee Moffitt Cancer Center and Research InstituteTampa, Florida  33612
Sylvester Cancer Center, University of MiamiMiami, Florida  33136
Mount Sinai Medical Center, NYNew York, New York  10029
New York Presbyterian Hospital - Cornell CampusNew York, New York  10021
Comprehensive Cancer Center at Wake Forest UniversityWinston-Salem, North Carolina  27157-1082
Medical College of WisconsinMilwaukee, Wisconsin  53226
University of Florida Health Science CenterGainesville, Florida  32610-0296
Tulane University School of MedicineNew Orleans, Louisiana  70112
New York Medical CollegeValhalla, New York  10595
Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsBaltimore, Maryland  21231-2410
Cleveland Clinic Taussig Cancer CenterCleveland, Ohio  44195
University of Kansas Medical CenterKansas City, Kansas  66160-7353
Holden Comprehensive Cancer CenterIowa City, Iowa  52242-1009
University of Nebraska Medical CenterOmaha, Nebraska  68198-3330
Hackensack University Medical CenterHackensack, New Jersey  07601
James P. Wilmot Cancer CenterRochester, New York  14642
Veterans Affairs Medical Center - Tampa (Haley)Tampa, Florida  33612
Winship Cancer Institute of Emory UniversityAtlanta, Georgia  30322
Rush Cancer InstituteChicago, Illinois  60612
Washington Cancer InstituteWashington, District of Columbia  20010
Saint Louis University Cancer CenterSaint Louis, Missouri  63110
Siteman Cancer CenterSaint Louis, Missouri  63110
Texas Oncology P.A.Dallas, Texas  75230-2503
Indiana Blood and Marrow TransplantBeech Grove, Indiana  46107
University of Missouri Kansas City School of MedicineKansas City, Missouri  64111