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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
Centre Hospitalier Universitaire, Amiens
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Non Neoplastic Condition focused on measuring aggressive systemic mastocytosis, smoldering systemic mastocytosis

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept 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

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

First Posted
October 8, 2008
Last Updated
April 25, 2016
Sponsor
Centre Hospitalier Universitaire, Amiens
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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

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Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis

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