Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis

NCT00769587 | Completed DMC

• 4 other identifiers: PI06-DR-DAMAJCHU-AMIENS-PI06EUDRACT-2006-005087-10INCA-RECF0510
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Non Neoplastic Condition; Precancerous Condition

Keywords

aggressive systemic mastocytosis; smoldering systemic mastocytosis

Outcome Measures

Primary Outcomes (1)

• Objective reduction of the infiltration rate at 6 months

  6 months

Study Arms (1)

Thalidomide

EXPERIMENTAL

Use of thalidomide

Drug: thalidomide

Interventions

thalidomide DRUG

Thalidomide

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Centre Hospitalier Universitaire, Amiens: lead

Study Sites (1)

Centre Hospitalier Universitaire d'Amiens

Amiens, 80054, France

Location

Related Publications (1)

• Gruson B, Lortholary O, Canioni D, Chandesris O, Lanternier F, Bruneau J, Grosbois B, Livideanu C, Larroche C, Durieu I, Barete S, Sevestre H, Diouf M, Chaby G, Marolleau JP, Dubreuil P, Hermine O, Damaj G. Thalidomide in systemic mastocytosis: results from an open-label, multicentre, phase II study. Br J Haematol. 2013 May;161(3):434-42. doi: 10.1111/bjh.12265. Epub 2013 Feb 22.

  PMID: 23432617 BACKGROUND

MeSH Terms

Conditions

Precancerous Conditions; Mastocytosis, Systemic

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Neoplasms; Mastocytosis; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Mast Cell Activation Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Gandhi Damaj

  Centre Hospitalier Universitaire, Amiens

  STUDY CHAIR

Study Design

• Study Type: interventional
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2008
• First Posted: October 9, 2008
• Study Start: June 1, 2007
• Primary Completion: May 1, 2011
• Study Completion: October 1, 2012
• Last Updated: September 22, 2025

Record last verified: 2025-09

Locations

FR (1)