Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis

This study has been completed.
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: October 8, 2008
Last updated: May 12, 2011
Last verified: July 2009

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
Nonneoplastic Condition
Precancerous Condition
Drug: thalidomide
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Primary Purpose: Treatment
Official Title: Phase II Study of Thalidomide in Mastocytosis

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

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

Estimated Enrollment: 25
Study Start Date: June 2007
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
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

Additional Information:
No publications provided 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: May 12, 2011
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
aggressive systemic mastocytosis
smoldering systemic mastocytosis

Additional relevant MeSH terms:
Mastocytosis, Systemic
Precancerous Conditions
Neoplasms by Histologic Type
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
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 March 26, 2015