Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis
Primary Purpose
Non Neoplastic Condition, Precancerous Condition
Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
thalidomide
Sponsored by
About this trial
This is an interventional treatment trial for Non Neoplastic Condition focused on measuring aggressive systemic mastocytosis, smoldering systemic mastocytosis
Eligibility Criteria
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
Sites / Locations
- Centre Hospitalier Universitaire d'Amiens
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Thalidomide
Arm Description
Use of thalidomide
Outcomes
Primary Outcome Measures
Objective reduction of the infiltration rate at 6 months
Secondary Outcome Measures
Full Information
NCT ID
NCT00769587
First Posted
October 8, 2008
Last Updated
April 25, 2016
Sponsor
Centre Hospitalier Universitaire, Amiens
1. Study Identification
Unique Protocol Identification Number
NCT00769587
Brief Title
Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis
Official Title
Phase II Study of Thalidomide in Mastocytosis
Study Type
Interventional
2. Study Status
Record Verification Date
April 2016
Overall Recruitment Status
Completed
Study Start Date
June 2007 (undefined)
Primary Completion Date
May 2011 (Actual)
Study Completion Date
October 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Centre Hospitalier Universitaire, Amiens
4. Oversight
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Non Neoplastic Condition, Precancerous Condition
Keywords
aggressive systemic mastocytosis, smoldering systemic mastocytosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Thalidomide
Arm Type
Experimental
Arm Description
Use of thalidomide
Intervention Type
Drug
Intervention Name(s)
thalidomide
Primary Outcome Measure Information:
Title
Objective reduction of the infiltration rate at 6 months
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gandhi Damaj
Organizational Affiliation
Centre Hospitalier Universitaire, Amiens
Official's Role
Study Chair
Facility Information:
Facility Name
Centre Hospitalier Universitaire d'Amiens
City
Amiens
ZIP/Postal Code
80054
Country
France
12. IPD Sharing Statement
Learn more about this trial
Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis
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