Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
First received: October 28, 2008
Last updated: January 20, 2016
Last verified: January 2016
This study will investigate if the drug midostaurin taken orally twice daily is effective and safe in treating patients with aggressive systemic mastocytosis or mast cell leukemia with or without an additional hematological neoplasm.

Condition Intervention Phase
Drug: Midostaurin
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single Arm, Phase II, Open-Label Study to Determine the Efficacy of 100mg Twice Daily Oral Dosing of Midostaurin Administered to Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Overall response rate according to established criteria by assessing clinical findings at the end of 6 cycles [ Time Frame: at the end of 6 cycles ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Duration of response [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
  • Time to response [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
  • Adverse event rate [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
  • Overall survival (OS) [ Time Frame: throughout the study ] [ Designated as safety issue: No ]
  • KIT mutational status at diagnosis and after 6 cycles of therapy [ Time Frame: at diagnosis and after 6 cycles of therapy ] [ Designated as safety issue: No ]

Enrollment: 116
Study Start Date: October 2008
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: July 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Midostaurin Drug: Midostaurin


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed diagnosis of aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL) according to WHO criteria for SM and established criteria for ASM and MCL (Valent et al 2003), presenting with at least one measurable C-Finding.
  • ECOG performance status of 0-3
  • Life expectancy > 12 weeks
  • ECG: QTc interval ≤ 450 ms
  • Meeting the following laboratory values:
  • AST and ALT must be ≤ 5 x Upper Limit of Normal (ULN) if this elevation is solely due to ASM/MCL, otherwise AST, ALT must be ≤ 2.5 x ULN
  • Serum Bilirubin must be ≤ 3 x Upper Limit of Normal (ULN) if this elevation is solely due to ASM/MCL, otherwise serum bilirubin must be

    • 1.5 x ULN
  • Serum Creatinine ≤ 2.0 mg/dL

Exclusion Criteria:

  • Any other concurrent severe known disease (except carcinoma in-situ) concurrent severe and/or uncontrolled medical condition including congestive heart failure grade III or IV according to the NYHA classification or with ejection fraction < 50%, etc.
  • Patients with any pulmonary infiltrate including those suspected to be of infectious origin. Exception: Patients with a pleural effusion related to the disease under study as confirmed by the investigator are permitted to enter the study
  • Patients who have demonstrated relapse to 3 or more prior regimens of SM treatment (not including those given for supportive care)
  • Patients who have received any investigational agent, chemotherapy, interferon-α, or 2-chlorodeoxyadenosine (2-CdA, cladribine) within 30 days prior to first dose
  • Patients who have ASM with eosinophilia and known positivity for the FIP1L1-PDGFRα fusion unless they have demonstrated relapse or disease progression on prior imatinib therapy

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00782067

  Show 32 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Additional Information:
No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00782067     History of Changes
Other Study ID Numbers: CPKC412D2201  2008-000280-42 
Study First Received: October 28, 2008
Last Updated: January 20, 2016
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
Belgium: Federal Agency for Medicines and Health Products, FAMHP
Canada: Health Canada
France: Direction Générale de la Santé
Germany: Federal Institute for Drugs and Medical Devices
Italy: Ministry of Health
Netherlands: Ministry of Health, Welfare and Sport
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Novartis:
Aggressive systemic mastocytosis
mast cell leukemia
tyrosine kinase inhibitor
KIT mutation

Additional relevant MeSH terms:
Leukemia, Mast-Cell
Mastocytosis, Systemic
Urticaria Pigmentosa
Behavioral Symptoms
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Mastocytosis, Cutaneous
Neoplasms by Histologic Type
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Pigmentation Disorders
Skin Diseases
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on February 11, 2016