NCT02415608

Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis

A Phase 2 Study of Ibrutinib in Advanced Systemic Mastocytosis

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Jason Robert Gotlib · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase 2 trial studies ibrutinib to see how well it works in treating patients with systemic (affecting the entire body) mastocytosis that has spread to other parts of the body and usually cannot be cured or controlled with treatment (advanced). Systemic mastocytosis is a disease in which too many mast cells (a type of immune system cell) are found throughout the body. Mast cells give off chemicals such as histamine that can cause flushing (a hot, red face), itching, abdominal cramps, muscle pain, nausea, vomiting, diarrhea, low blood pressure, and shock. Ibrutinib may stop the growth of mast cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVE: Evaluate the response rate to ibrutinib in patients with advanced systemic mastocytosis (SM) (aggressive systemic mastocytosis \[ASM\] or mast cell leukemia \[MCL\], or SM-associated hematologic non-mast cell disorder \[AHNMD\]) by the end of 6 cycles (6 months). SECONDARY OBJECTIVES: * Evaluate the tolerability and safety profile of ibrutinib in patients with advanced SM. * Evaluate the pharmacokinetic (PK) profile of ibrutinib in a subset of patients with advanced SM. * Evaluate changes in histopathology (blood and bone marrow) of patients with advanced SM in response to ibrutinib therapy. * Evaluate changes in mastocytosis related symptom scores and quality-of-life (QOL) using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF). * Evaluate the duration of response (DoR) and time to response (TTR). * Evaluate progression-free survival (PFS) and overall survival. OUTLINE: Patients receive ibrutinib orally (PO) once daily (QD) on days 1 to 28. Treatment repeats every 28 days for up to 6 months in the absence of disease progression or unacceptable toxicity. Patients achieving an unconfirmed or confirmed clinical improvement (CI), partial response (PR), or complete response (CR) by the end of course 6 will be permitted to continue maintenance courses of ibrutinib on an ongoing basis until loss of response/progressive disease, or unacceptable toxicity. After completion of study treatment, patients are followed up for 30 days and then every 6 months thereafter.

Conditions

• Aggressive Systemic Mastocytosis
• Mast Cell Leukemia
• Systemic Mastocytosis

Interventions

Type	Name	Description
DRUG	Ibrutinib	Given orally in 28-day cycles

Timeline

• Start date: 2015-03-01
• Primary completion: 2016-11-04
• Completion: 2017-06-14
• First posted: 2015-04-14
• Last updated: 2018-09-20
• Results posted: 2018-09-20

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study