Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis

Sponsor

Centre Hospitalier Universitaire, Amiens (Other)

Overall Status

Completed

CT.gov ID

NCT00769587

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

0.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Non Neoplastic Condition Precancerous Condition	Drug: thalidomide	Phase 2

Detailed Description

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

20 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Phase II Study of Thalidomide in Mastocytosis

Study Start Date :

Jun 1, 2007

Actual Primary Completion Date :

May 1, 2011

Actual Study Completion Date :

Oct 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Thalidomide Use of thalidomide	Drug: thalidomide

Arm

Intervention/Treatment

Experimental: Thalidomide

Use of thalidomide

Drug: thalidomide

Outcome Measures

Primary Outcome Measures

Objective reduction of the infiltration rate at 6 months [6 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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