Thalidomide at Low Dose for the Treatment of Patient With Myelodysplastic Syndromes - THAL-SMD-200

Study Overview
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Brief Summary
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The GFM previously conducted a dose-escalating phase II trial of thalidomide in MDS with a minimum dose of 200mg/d and a maximum dose 800mg/d. Responses were evaluated according to IWG criteria at week 16 and thalidomide continued up to week 56 in responders. 82% patients received at least 8 weeks of treatment and were evaluable. 59% had hematological improvement, mainly on the erythroid lineage (Increase of Hemoglobin). Most responses were observed at low doses and between 4 and 8 weeks. The objectives of this trial (Thal-SMD-20) are to evaluate the efficacy and tolerance of lower doses thalidomide in low risk MDS patients with transfusion-dependant anemia.

Detailed Description
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Thalidomide: First part of the trial: 82 patients at 200mg/day given at bedtime x 12 weeks, decreased to 100mg/day if grade 1 or 2 side. Stopped temporally for 1 week if grade 3 or 4 side effects. Then reintroduced at the same dose. If side effects again, definitively stopped. Responses evaluated at 12 weeks according to IWG criteria for the erythroid lineage At week 12: If no Hematological improvement (HI): increased to 300mg/day for 8 weeks and then eventually to 400mg/day for 8 weeks more, if no HI. If Hematological improvement (HI): continued at the same dose. Second part of the trial: 30 patients treated at 50mg/day x 12 weeks. Responses evaluated at 12 weeks according to IWG criteria for the erythroid lineage At week 12: If no Hematological improvement (HI): increased to 100mg/day for 8 weeks and then eventually to 200mg/day for 8 weeks more, if no HI. If Hematological improvement (HI): continued at the same dose.

Official Title
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Thalidomide for the Treatment of Cytopenias of Patients With Low Risk Myelodysplastic Syndromes

Conditions
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Myelodysplastic Syndromes

Intervention / Treatment
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* Drug: Thalidomide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients ≥18 years, with IPSS Low or Int-1 MDS Transfusion dependant anemia above 2 packed red blood cells (PRBC)/month ECOG index = 0, 1, 2 No peripheral neurological disease Exclusion Criteria: MDS patients with IPSS Int-2 or High Patients with less than 2 packed red blood cells (PRBC)/month Patients with previous history of venous thrombosis Patient treated with EPO +/- G-CSF in the 2 months before inclusion in the protocol Patient having received intensive chemotherapy in the 3 months before inclusion in the protocol Patient having received Thalidomide in a previous protocol Patient presenting an iron, B12 vitamin or folic acid uncorrected deficiency Patient with peripheral neurological disease Patient not being able to subject itself to a regular clinical and biological follow-up Pregnant patient or patient in a period of lactation Patient refusing to take a contraceptive treatment through out all the study Patient receiving drugs able to interfere with the mechanism of action of Thalidomide Patient refusing to sign the informed consent.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Thalidomide|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy evaluated at week 12 according to the IWG criterias |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Low risk myelodysplastic syndromes, MDS, Bone marrow diseases, Thalidomide, Cytopenias, Anemia