Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis

Study Overview
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Brief Summary
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RATIONALE: Thalidomide may stop the growth of systemic mastocytosis by blocking blood flow to the disease. PURPOSE: This phase II trial is studying how well thalidomide works in treating patients with relapsed or progressive systemic mastocytosis.

Detailed Description
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OBJECTIVES: Primary Determine the objective response rate at 6 months in patients with systemic mastocytosis treated with thalidomide. Secondary Determine the tolerability of this drug in these patients. OUTLINE: This is a multicenter study. Patients receive oral thalidomide once daily for 6 months in the absence of disease progression.

Official Title
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Phase II Study of Thalidomide in Mastocytosis

Conditions
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Non Neoplastic Condition, Precancerous Condition

Intervention / Treatment
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* Drug: thalidomide


Participation Criteria
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Eligibility Criteria
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DISEASE CHARACTERISTICS: Diagnosis of systemic mastocytosis Aggressive or borderline (smoldering) disease (in first line or more) Relapsed or progressive disease Measurable or evaluable disease Presence of c-Kit D816V mutation in the skin, spine, or infiltrated organs No nonsymptomatic mastocytosis PATIENT CHARACTERISTICS: Life expectancy > 3 months Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception for 1 month prior to, during, and until first menstrual cycle after completion of study treatment Bilirubin < 2 times normal (unrelated to disease) Liver enzymes < 2 times normal (unrelated to disease) Creatinine ≤ 300 mmol/L No central or peripheral neuropathy leading to psychiatric concerns No HIV positivity No active infection or other serious underlying illness that would preclude treatment No history of thromboembolism or deep vein thrombosis No geographical, social, or psychological reasons preventing medical monitoring PRIOR CONCURRENT THERAPY: More than 4 weeks since prior antitumor therapy (e.g., chemotherapy, radiotherapy) No other concurrent treatment specific for this disease No concurrent participation in another experimental drug trial

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 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

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Thalidomide<br>Use of thalidomide | Drug: thalidomide<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Objective reduction of the infiltration rate at 6 months |  | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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aggressive systemic mastocytosis, smoldering systemic mastocytosis