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A Phase II Study of the Efficacy of Rabbit Anti-thymocyte Globulin (rATG) in Patients With Low and Intermediate-1 Risk Myelodysplastic Syndrome

Phase 2
18 Years
70 Years
Not Enrolling
Myelodysplastic Syndrome (MDS)

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Trial Information

A Phase II Study of the Efficacy of Rabbit Anti-thymocyte Globulin (rATG) in Patients With Low and Intermediate-1 Risk Myelodysplastic Syndrome

Inclusion Criteria:

- Patient provided signed written informed consent.

- Patient had pathologically confirmed low or intermediate-1 risk MDS at the time of
MDS diagnosis and at the time of screening.

- Patient had received no more than 1 prior treatment for MDS.

- Patient exhibited at least 1 hematologic cytopenia (anemia, neutropenia, or
thrombocytopenia) over a period of ≥1 week.

- Patient had documentation of any prior transfusion requirements.

- Patient had an Eastern Cooperative Oncology Group (ECOG) performance score of 0, 1,
or 2.

- Patient was ≥18 and ≤70 years of age at time of signing the informed consent document

- Patient was able to adhere to study visit schedule and all other protocol

- Patient was willing to practice a medically approved method of birth control during
participation in the study (at least 12 months after the last infusion of rATG)
(fertile male and female patients).

Exclusion Criteria:

- Patient was pregnant or lactating.

- Patient has had prior treatment with any ATG.

- Patient has received any immunomodulatory or immunosuppressing agents (excluding
steroids) <12 weeks prior to the first infusion of rATG.

- Patient has had a prior hematopoietic stem cell transplantation and/or other organ

- Patient has had a prior allergic reaction to rabbit proteins or excipients.

- Patient had any of the following subtypes of MDS: refractory anemia with ringed
sideroblasts (RARS); chronic myelomonocytic leukemia (CMML) if white blood counts
>13x10^9/L; or other MDS/myeloproliferative diseases (MPD).

- Patient had MDS associated with a 5q chromosomal deletion unless the patient received
prior lenalidomide treatment <4 weeks prior to the first infusion of rATG.

- Patient had MDS presumed secondary to exposure to chemicals or treatment with
radiotherapy or chemotherapy.

- Patient received any investigational agents within 4 weeks prior to the first
infusion of rATG.

- Patient has any of the following abnormalities: serum creatinine >1.5 x upper limit
of normal (ULN); aspartate transaminase (AST) and alanine transaminase (ALT) >2.5 x
ULN; or serum total bilirubin >1.5 x ULN, except for unconjugated hyperbilirubinemia
related to the patient's MDS.

- Patient received any treatment with non-steroidal anti-inflammatory drugs (NSAIDs)
within 14 days prior to the start of treatment.

- Patient was known to be human immunodeficiency virus (HIV) positive.

- Patient had any prior diagnosis of malignancy other than MDS, unless the patient had
been disease-free for at least 5 years following the completion of curative intent

- Patient had any serious medical condition (other than MDS) that would limit survival
to <2 years.

- Patient had active acute or chronic infection, including cytomegaloviremia (CMV)
infection or deep tissue infection.

- Patient had any other serious medical condition, uncontrolled illness (including, but
not limited to, symptomatic congestive heart failure, unstable angina pectoris, or
cardiac arrhythmia), social condition, or psychiatric illness that would prevent the
patient from signing the informed consent document (ICD), or would place the patient
at unacceptable risk if he/she participated in the study, or that would limit
compliance with study requirements.

Type of Study:


Study Design:

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

Outcome Measure:

Number of Participants Who Achieved Hematologic Improvement (HI)

Outcome Description:

This is a measure of HI in the erythroid, platelet, and neutrophil lineages. Note that HI was observed in the erythroid lineage only, which is defined as a participant who had a >=1.5 g/dL increase in hemoglobin from baseline (pretreatment value must have been <11 g/dL) and who had a relevant reduction of units of red blood cell (RBC) transfusions by an absolute number of >=4 RBC transfusions over 8 weeks as compared with the pretreatment transfusion number in the previous 8 weeks. These criteria were taken from the 2006 International Working Group criteria.

Outcome Time Frame:

12 months

Safety Issue:


Principal Investigator

Medical Monitor

Investigator Role:

Study Director

Investigator Affiliation:



United States: Food and Drug Administration

Study ID:




Start Date:

October 2007

Completion Date:

July 2009

Related Keywords:

  • Myelodysplastic Syndrome (MDS)
  • Myelodysplastic Syndromes
  • Preleukemia