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Midostaurin in Indolent Systemic Mastocytosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01920204
Recruitment Status : Unknown
Verified January 2015 by Prof.dr. J.C. Kluin-Nelemans, University Medical Center Groningen.
Recruitment status was:  Active, not recruiting
First Posted : August 9, 2013
Last Update Posted : January 19, 2015
Information provided by (Responsible Party):
Prof.dr. J.C. Kluin-Nelemans, University Medical Center Groningen

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

Condition or disease Intervention/treatment Phase
Indolent Systemic Mastocytosis Drug: Midostaurin, Phase 2

Detailed Description:


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.


  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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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.
Study Start Date : August 2013
Estimated Primary Completion Date : March 2015
Estimated Study Completion Date : May 2015

Arm Intervention/treatment
Experimental: Midostaurin
Treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously.
Drug: Midostaurin,
Midostaurin, twice daily 100 mg orally, continuously for 6 months
Other Name: PKC412

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Persistence of improvements [ Time Frame: 6 months ]
    persistence of improvement symptom score at 6 months.

  2. 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.

  3. Adverse events [ Time Frame: 6 months ]
    Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with Indolent Systemic Mastocytosis (ISM) or Smouldering Systemic Mastocytosis (SSM) according to the WHO criteria
  • Presence of the D816V c-KIT mutation
  • Serum tryptase > 20 mg/l
  • Serious mediator-related symptoms that cannot be controlled by H1 and H2 blocking drugs. Symptoms will be scored by an adapted MSAF (mastocytosis symptom assessment form) with at least:

    • a pre-study score of 4 or more on 3 non-related items,
    • or a pre-study score of 5 or more on 2 non-related items.
    • one item from the scoring list can be replaced by flushes 7 or more per week or anaphylactic attacks 1 or more per week.
  • Age >18 years
  • Willingness to apply optimal contraceptive measures (double barrier method, both men and women) for women below the age of 55, men at all ages; for both: if sexually active.
  • Written informed consent

Exclusion Criteria:

  • Aggressive systemic mastocytosis, mast cell leukemia, or ASM with or without accompanying non-clonal related non-mast cell disorder (SM-ANHMD).
  • Any known other present malignancy, non-melanoma skin cancers excluded
  • History of malignancy within the last 5 years, non-melanoma skin cancers excluded
  • Any serious comorbidity interfering with therapy compliance and follow-up compliance
  • Pregnancy
  • Patients not willing or who are not able to comply with contraceptive measures

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01920204

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University Medical Center Groningen
Groningen, Netherlands, 9700RB
Sponsors and Collaborators
University Medical Center Groningen
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Principal Investigator: J.C. Kluin-Nelemans, MD, PhD University Medical Center Groningen
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Prof.dr. J.C. Kluin-Nelemans, Prof.dr., University Medical Center Groningen Identifier: NCT01920204    
Other Study ID Numbers: UMCG41973
First Posted: August 9, 2013    Key Record Dates
Last Update Posted: January 19, 2015
Last Verified: January 2015
Keywords provided by Prof.dr. J.C. Kluin-Nelemans, University Medical Center Groningen:
Additional relevant MeSH terms:
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Mastocytosis, Systemic
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Skin Diseases
Immune Complex Diseases
Immune System Diseases
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action