Antithymocyte Globulin and Cyclosporine in Treating Patients With Myelodysplastic Syndrome

Antithymocyte Globulin (ATG) and Cyclosporine (CSA) to Treat Patients With Myelodysplastic Syndrome (MDS). A Randomized Trial Comparing ATG + CSA With Best Supportive Care

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00004208

Enrollment

Registered

2003-01-27

Start date

2000-08-31

Completion date

2011-10-31

Last updated

2015-03-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

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

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

Interventions

DRUGATG + CSA

Mérieux; horse antithymocyte globulin (ATG) + Cyclosporine A (CSA)

BEHAVIORALSupportive care

Patients randomized to this arm will be treated as outpatients.

Study design

Allocation

RANDOMIZED

Intervention model

FACTORIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion 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

Design outcomes

Primary

Measure	Time frame
Best response rate (CR + PR)	6 months

Secondary

Measure	Time frame
Response rate at month 3, time to response, quality and duration of response, and proportions of relapse/progression in responders	2 and 5 years after first response
Overall survival	2 and 5 years
Leukemia-free survival	2 and 5 years
Transformation-free survival	2 and 5 years

Countries

Switzerland

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026