Indolent Systemic Mastocytosis Clinical Trial
Official title:
Phase II Single Arm Open Pilot Study to Demonstrate the Efficacy of Midostaurin in Symptom Improvement and Decrease of Mast Cell Burden in Patients With Indolent or Smoldering Systemic Mastocytosis.
Rationale: Patients with indolent or smoldering systemic mastocytosis can have severe disabling symptoms. Almost all patients have fatigue, a compromised quality of life, hampering normal functioning. Because this form of mastocytosis is not considered life-threatening, mast cell eradication has never been applied and patients receive only symptomatic therapy with histamine blockers. Midostaurin, a c-KIT inhibitor has shown activity regarding symptom control and decrease of malignant mast cells in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia
Objective:
Primary: To study in a pilot phase II trial the efficacy of midostaurin administered at an
oral dose of 100 mg twice daily in patients with indolent or smoldering systemic
mastocytosis on mediator symptom reduction, documented by the Mastocytosis Symptom
Assessment Questionnaire, measured at 3 months.
Secondary:
1. To study whether symptom improvement persists at 6 months, and whether midostaurin can
reduce mast cell infiltration in the skin and bone marrow, documented by decrease of
serum tryptase, decrease of urticaria pigmentosa and decrease of bone marrow mast
cells.
2. To assess safety and tolerability of midostaurin in the above mentioned settings
Study design: Single arm, open label pilot phase II study.
Study population: Adult patients (n=20) with histologically documented systemic
mastocytosis, indolent or smoldering subtype, with severe symptoms, not controlled by
histamine 1 and 2 blockers.
Intervention: treatment with Midostaurin, twice daily 100 mg orally for 6 months
continuously.
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04655118 -
Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis
|
Phase 2 | |
Recruiting |
NCT04910685 -
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
|
Phase 2/Phase 3 | |
Completed |
NCT03632811 -
Adaptation of the Questionnaire "Regarding Patient's Quality of Life With Mastocytosis" in the French Language
|
||
Completed |
NCT02808793 -
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002
|
Phase 1 | |
Completed |
NCT02478957 -
Treatment of Indolent Systemic Mastocytosis With PA101
|
Phase 2 | |
Completed |
NCT03770273 -
Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis
|
Phase 2 | |
Recruiting |
NCT04333108 -
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment
|
Phase 3 | |
Not yet recruiting |
NCT06210698 -
Angioedema Biomarker Research Study
|
||
Completed |
NCT00814073 -
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis
|
Phase 3 | |
Active, not recruiting |
NCT03731260 -
(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
|
Phase 2 |