Low And Intermediate-1 Risk Myelodysplastic Syndrome in adult patients 70 years or younger MedDRA version: 9.1 Level: LLT Classification code 10028533 Term: Myelodysplastic syndrome
Conditions
Interventions
Trade Name: Thymoglobulin
Product Name: Thymoglobulin
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: rabbit anti-human thymocyte immunoglobulin
Concentration unit: mg/ml mi
Sponsors
Genzyme Europe BV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Provides signed written informed consent. 2.Has pathologically confirmed MDS with a low or intermediate-1 risk score as assessed by IPSS at the time of MDS diagnosis and at time of screening. 3.Has received no more than 1 prior treatment for MDS (excluding steroids, transfusions, antibiotics and other supportive care therapies) and has recovered to = Grade 1 or baseline non-hematological toxicities related to the prior treatment, which is restricted to the following modalities (administered in an investigational or non-investigational setting): oHematopoietic growth factors, either alone or in combination, 4 weeks prior to the first infusion of rATG. ? For patients for whom erythropoiesis stimulating agents (ESAs) are available therapies for MDS (country specific), the Investigator must determine that the patient either did not respond to a trial of an ESA or did not meet the criteria predictive of an intermediate or high response to ESA. See Appendix D for the erythropoietin (EPO) predictive model. oAzacitidine, = 4 weeks prior to the first infusion of rATG; oDecitabine, = 4 weeks prior to the first infusion of rATG; oLenalidomide, = 4 weeks prior to the first infusion of rATG; oThalidomide, = 4 weeks prior to the first infusion of rATG 4.Exhibited at least 1 of the following hematologic cytopenias as determined from at least 2 time points over a period of = 1-week. oAnemia (hemoglobin =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1.Is pregnant or lactating. 2.Has had prior treatment with any ATG. 3.•Has received any immunomodulatory or immunosuppressing agents (excluding steroids) 13 x 10*9 /L, or •other MDS types of the MDS/myeloproliferative diseases (MPD) group according to WHO classification. 7.Has MDS associated with a 5q chromosomal deletion based upon local bone marrow cytogenetic analysis, unless the patient received prior lenalidomide treatment. 8.Has MDS presumed secondary to exposure to chemicals or treatment with radiotherapy or chemotherapy. 9.Received any investigational agents within 4 weeks prior to the first infusion of rATG, except for the allowable investigational agents outlined in Inclusion Criterion #3. 10.Has a serum creatinine >1.5 x upper limit of normal (ULN). 11.Received any treatment with non-steroidal anti-inflammatory drugs (NSAID) within 14 days prior to start of study treatment. 12.Has aspartate transaminase (AST) and alanine transaminase (ALT) >2.5 x ULN. 13.Has serum total bilirubin >1.5 ULN except for unconjugated hyperbilirubinemia related to the patient’s MDS. 14.Is known to be human immunodeficiency virus (HIV) positive. 15.Has any prior diagnosis of malignancy other than MDS, unless the patient has been disease-free for at least 5 years following the completion of curative intent therapy with the following exceptions: •Patients with treated basal-cell skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia, regardless of the disease-free duration, are eligible for this study if definitive treatment for the condition has been completed, •Patients with organ-confined prostate cancer, with no evidence of recurrent or progressive disease based on prostate-specific antigen (PSA) values, are also eligible for this study, if hormonal therapy has been initiated or a radical prostatectomy has been performed. 16.Any serious medical condition (other than MDS) that would limit survival to <2 years. 17.Active acute or chronic infection, including active cytomegaloviremia (CMV) infection with positive immunoglobulin M (IgM) titers, and/or CMV-DNA-PCR positive copy numbers, or deep tissue infection. 18.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 will prevent the patient from signing the ICD, or will place the patient at unacceptable risk if he/she participates in the study, or that would limit compliance with study requirements.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the Hematolgic Improvement (HI) rate during the 12-month study period with rATG (Thymoglobulin), as defined by the IWG and published in 2006 by Cheson and colleagues;Secondary Objective: •To determine the duration of HI. •To determine the disease remission (Complete Remission (CR) +Partial Remission (PR))rate. •To determine the duration of disease remission (CR + PR). •To determine the Transfusion Independence (TI) rate. •To determine the duration of TI. •To determine the relapse rate after HI, CR, or PR. •To determine the cytogenetic response and marrow CR rates. •To determine progression free survival. •To determine the rate of transformation to Acute Myeloid Leukemia (AML) ;Primary end point(s): Hematologic improvement as assessed by the Investigator according to IWG guidelines | — |
Countries
France, Germany, Netherlands, United Kingdom
Outcome results
None listed