Clinical trial · Interventional
Imatinib in KIT-negative Systemic Mastocytosis
Imatinib Mesylate Therapy in Systemic Mastocytosis Patients Lacking KIT Mutations
NCT01297777CI-TRIAL-00023680completedPhase 4ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The aim of this study is to evaluate the efficacy in terms of clinical and biological response rates of Imatinib Mesylate therapy in patients with systemic mastocytosis lacking KIT mutations.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Systemic Mastocytosis | Systemic Mastocytosis | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Imatinib Mesylate | Drug | Imatinib Mesylate | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Imatinib mesylate
- description
- Imatinib mesylate 300 or 400 mg daily for 12 months.
- interventionNames
- Drug: Imatinib Mesylate
Primary outcomes (2)
- measure
- To evaluate the effect of Imatinib Mesylate on the grade of bone marrow mast cells infiltration.
- timeFrame
- 6 months
- description
- The grade of bone marrow infiltration is evaluated before and after 6 months of therapy by bone marrow histology and cytology, and flow cytometry performed on highly-purified bone marrow mast cells from patients with B or C findings
- measure
- To evaluate the effect of Imatinib Mesylate on the grade of bone marrow mast cells infiltration.
- timeFrame
- 12 months
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Age older than 18 years. * Diagnosis of systemic mastocytosis in the absence of c-kit mutation. * ECOG ≤ 3. * Signed informed consent. Exclusion Criteria: * Previous therapy with a tyrosin kinase inhibitor. * Positive antibodies against HIV or active viral hepatitis. * Impaired liver function (total bilirubin ≥ 2.0 mg/dl, AST or ALT \> 3 x upper limit of normal). * Impaired renal function (≥ 2.0 mg/dL). * Grade III-IV cytopenias not related to mastocytosis. * Severe cardiopathy (grade III/IV of NYHA, or left ventricular ejection fraction \< 50%). * Pregnancy or breastfeeding. * Female patients who do not use contraceptive methods.
References
Publications (7)
- BACKGROUNDZermati Y, De Sepulveda P, Feger F, Letard S, Kersual J, Casteran N, Gorochov G, Dy M, Ribadeau Dumas A, Dorgham K, Parizot C, Bieche Y, Vidaud M, Lortholary O, Arock M, Hermine O, Dubreuil P. Effect of tyrosine kinase inhibitor STI571 on the kinase activity of wild-type and various mutated c-kit receptors found in mast cell neoplasms. Oncogene. 2003 Feb 6;22(5):660-4. doi: 10.1038/sj.onc.1206120. PMID 12569358
- BACKGROUNDAkin C, Brockow K, D'Ambrosio C, Kirshenbaum AS, Ma Y, Longley BJ, Metcalfe DD. Effects of tyrosine kinase inhibitor STI571 on human mast cells bearing wild-type or mutated c-kit. Exp Hematol. 2003 Aug;31(8):686-92. doi: 10.1016/s0301-472x(03)00112-7. PMID 12901973
- BACKGROUNDZhang LY, Smith ML, Schultheis B, Fitzgibbon J, Lister TA, Melo JV, Cross NC, Cavenagh JD. A novel K509I mutation of KIT identified in familial mastocytosis-in vitro and in vivo responsiveness to imatinib therapy. Leuk Res. 2006 Apr;30(4):373-8. doi: 10.1016/j.leukres.2005.08.015. Epub 2005 Sep 22. PMID 16183119
- BACKGROUNDMa Y, Zeng S, Metcalfe DD, Akin C, Dimitrijevic S, Butterfield JH, McMahon G, Longley BJ. The c-KIT mutation causing human mastocytosis is resistant to STI571 and other KIT kinase inhibitors; kinases with enzymatic site mutations show different inhibitor sensitivity profiles than wild-type kinases and those with regulatory-type mutations. Blood. 2002 Mar 1;99(5):1741-4. doi: 10.1182/blood.v99.5.1741. PMID 11861291
- BACKGROUNDGarcia-Montero AC, Jara-Acevedo M, Teodosio C, Sanchez ML, Nunez R, Prados A, Aldanondo I, Sanchez L, Dominguez M, Botana LM, Sanchez-Jimenez F, Sotlar K, Almeida J, Escribano L, Orfao A. KIT mutation in mast cells and other bone marrow hematopoietic cell lineages in systemic mast cell disorders: a prospective study of the Spanish Network on Mastocytosis (REMA) in a series of 113 patients. Blood. 2006 Oct 1;108(7):2366-72. doi: 10.1182/blood-2006-04-015545. Epub 2006 Jun 1. PMID 16741248
- RESULTAkin C, Fumo G, Yavuz AS, Lipsky PE, Neckers L, Metcalfe DD. A novel form of mastocytosis associated with a transmembrane c-kit mutation and response to imatinib. Blood. 2004 Apr 15;103(8):3222-5. doi: 10.1182/blood-2003-11-3816. Epub 2003 Dec 24.