Clinical Trial: Midostaurin in Indolent Systemic Mastocytosis

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

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 Sy

Brief Summary: 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

Detailed Summary:

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.


Sponsor: University Medical Center Groningen

Current Primary Outcome: Symptom Scoring [ Time Frame: 12 weeks ]

Percent change in the total score ("Sumscore") of all symptoms assessed by the Mastocytosis Symptom Assessment Form (MSAF) after 12 weeks.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Persistence of improvements [ Time Frame: 6 months ]
    persistence of improvement symptom score at 6 months.
  • Mast cell burden [ Time Frame: 6 months ]
    Percent change in the mast cell burden (bone marrow infiltrate, skin infiltrate, serum tryptase levels) after 6 months.
  • Adverse events [ Time Frame: 6 months ]
    Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.


Original Secondary Outcome: Same as current

Information By: University Medical Center Groningen

Dates:
Date Received: August 7, 2013
Date Started: August 2013
Date Completion: May 2015
Last Updated: January 15, 2015
Last Verified: January 2015