Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis

This study has been completed.
Information provided by (Responsible Party):
Centre Hospitalier Universitaire, Amiens Identifier:
First received: October 8, 2008
Last updated: July 27, 2015
Last verified: July 2015

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 Intervention Phase
Non Neoplastic Condition
Precancerous Condition
Drug: thalidomide
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Study of Thalidomide in Mastocytosis

Resource links provided by NLM:

Further study details as provided by Centre Hospitalier Universitaire, Amiens:

Primary Outcome Measures:
  • Objective reduction of the infiltration rate at 6 months [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Enrollment: 20
Study Start Date: June 2007
Study Completion Date: October 2012
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Thalidomide
Use of thalidomide
Drug: thalidomide

Detailed Description:



  • Determine the objective response rate at 6 months in patients with systemic mastocytosis treated with thalidomide.


  • 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.


Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • 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


  • 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


  • 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
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Please refer to this study by its identifier: NCT00769587

Centre Hospitalier Universitaire d'Amiens
Amiens, France, 80054
Sponsors and Collaborators
Centre Hospitalier Universitaire, Amiens
Study Chair: Gandhi Damaj Centre Hospitalier Universitaire, Amiens
  More Information

No publications provided

Responsible Party: Centre Hospitalier Universitaire, Amiens Identifier: NCT00769587     History of Changes
Other Study ID Numbers: CDR0000599564  CHU-AMIENS-PI06  EUDRACT-2006-005087-10  INCA-RECF0510 
Study First Received: October 8, 2008
Last Updated: July 27, 2015
Health Authority: France : Afssaps

Keywords provided by Centre Hospitalier Universitaire, Amiens:
aggressive systemic mastocytosis
smoldering systemic mastocytosis

Additional relevant MeSH terms:
Mastocytosis, Systemic
Precancerous Conditions
Urticaria Pigmentosa
Mastocytosis, Cutaneous
Neoplasms by Histologic Type
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Pathologic Processes
Pigmentation Disorders
Skin Diseases
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Anti-Bacterial Agents
Anti-Infective Agents
Antineoplastic Agents
Growth Inhibitors
Growth Substances
Immunologic Factors
Immunosuppressive Agents
Leprostatic Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on February 11, 2016