Midostaurin in Indolent Systemic Mastocytosis
|ClinicalTrials.gov Identifier: NCT01920204|
Recruitment Status : Unknown
Verified January 2015 by Prof.dr. J.C. Kluin-Nelemans, University Medical Centre Groningen.
Recruitment status was: Active, not recruiting
First Posted : August 9, 2013
Last Update Posted : January 19, 2015
|Condition or disease||Intervention/treatment||Phase|
|Indolent Systemic Mastocytosis||Drug: Midostaurin,||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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.|
|Study Start Date :||August 2013|
|Estimated Primary Completion Date :||March 2015|
|Estimated Study Completion Date :||May 2015|
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
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01920204
|University Medical Center Groningen|
|Groningen, Netherlands, 9700RB|
|Principal Investigator:||J.C. Kluin-Nelemans, MD, PhD||University Medical Center Groningen|