Clinical trial NCT00004208: Antithymocyte Globulin and Cyclosporine in Treating Patients With Myelodysplastic Syndrome
Official title: Antithymocyte Globulin (ATG) and Cyclosporine (CSA) to Treat Patients With Myelodysplastic Syndrome (MDS). A Randomized Trial Comparing ATG + CSA With Best Supportive Care
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Myelodysplastic Syndromes
Keywords: refractory anemia
refractory anemia with ringed sideroblasts
refractory anemia with excess blasts
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
Lead sponsor: Swiss Group for Clinical Cancer Research
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 86
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
Primary purpose: TREATMENT

Brief summary: The main objective of this trial is to evaluate the efficacy and toxicity of intensive immuno-suppression with ATG + CSA versus best supportive care in patients with transfusion dependent low and intermediate risk myelodysplastic syndrome (MDS).

Detailed description: This trial will answer the question whether immuno-suppression improves hematopoesis and reduces transfusion requirements analogous to patients with aplastic anemia as the short-term outcome, and whether immuno-suppression accelerates leukemic transformation and influences survival as the long-term outcome.

Primary endpoint: best response (CR + PR) rate at month 6

Eligibility criteria: Hypoplastic MDS, or MDS subtype RA, RAS or RAEB with ≤ 10% blasts, or RAEB with 10-20% blasts and the patient refusing or not eligible for the MDS high risk protocol (EORTC 06961)

* Transfusion dependence \< 24 months or neutrophils \< 0.5 × 109/l.
* ECOG/SAKK performance status ≤ 2
* Age \> 18 years
* No active uncontrolled infection
* No prior chemotherapy or radiotherapy
* No history of heart failure, clinically relevant cardiac arrhythmia or other hemato-oncological disease

Primary outcomes:
measure: Best response rate (CR + PR)
timeFrame: 6 months
Secondary outcomes:
measure: Response rate at month 3, time to response, quality and duration of response, and proportions of relapse/progression in responders
timeFrame: 2 and 5 years after first response
measure: Overall survival
timeFrame: 2 and 5 years
measure: Leukemia-free survival
timeFrame: 2 and 5 years
measure: Transformation-free survival
timeFrame: 2 and 5 years
Other outcomes:
Locations:
city: Basel
country: Switzerland
facility: Universitaetsspital-Basel
Study officials:
affiliation: Kantonsspital Basel
name: Jakob R. Passweg, MS
role: STUDY_CHAIR
References:
citation: Passweg JR, Giagounidis AA, Simcock M, Aul C, Dobbelstein C, Stadler M, Ossenkoppele G, Hofmann WK, Schilling K, Tichelli A, Ganser A. Immunosuppressive therapy for patients with myelodysplastic syndrome: a prospective randomized multicenter phase III trial comparing antithymocyte globulin plus cyclosporine with best supportive care--SAKK 33/99. J Clin Oncol. 2011 Jan 20;29(3):303-9. doi: 10.1200/JCO.2010.31.2686. Epub 2010 Dec 13.
citation: Passweg JR, Giagounidis A, Simcock M, et al.: Immunosuppression for patients with low and intermediate risk myelodysplastic syndrome: a prospective randomized multicenter trial comparing antithymocyte globulin + cyclosporine with best supportive care: SAKK 33/99. [Abstract] Blood 110 (11): A-1461, 2007.