Midostaurin in Indolent 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
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|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.|
- Symptom Scoring [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]Percent change in the total score ("Sumscore") of all symptoms assessed by the Mastocytosis Symptom Assessment Form (MSAF) after 12 weeks.
- Persistence of improvements [ Time Frame: 6 months ] [ Designated as safety issue: No ]persistence of improvement symptom score at 6 months.
- Mast cell burden [ Time Frame: 6 months ] [ Designated as safety issue: No ]Percent change in the mast cell burden (bone marrow infiltrate, skin infiltrate, serum tryptase levels) after 6 months.
- Adverse events [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.
|Study Start Date:||August 2013|
|Estimated Study Completion Date:||December 2014|
|Estimated Primary Completion Date:||July 2014 (Final data collection date for primary outcome measure)|
Treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously.
Midostaurin, twice daily 100 mg orally, continuously for 6 months
Other Name: PKC412
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.
- 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.
- 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.
|Contact: J.C. Kluin-Nelemans, MD, PhDfirstname.lastname@example.org|
|University Medical Center Groningen||Recruiting|
|Groningen, Netherlands, 9700RB|
|Principal Investigator:||J.C. Kluin-Nelemans, MD, PhD||University Medical Centre Groningen|