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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00782067
First received: October 28, 2008
Last updated: May 15, 2013
Last verified: May 2013
History of Changes
  Purpose

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
Leukemia
 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:

MedlinePlus related topics: Cancer Leukemia
U.S. FDA Resources

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: 117
Study Start Date: October 2008
Estimated Study Completion Date: July 2013
Estimated Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Midostaurin Drug: Midostaurin

  Eligibility

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

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
  • Patients who would be suitable for imatinib therapy (e.g. having ASM with eosinophilia and known positivity for the FIP1L1-PDGFRα fusion or known to be KIT D816V negative) unless they have demonstrated relapse or disease progression on prior imatinib therapy

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 failed more than two prior SM therapies (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 would be suitable for imatinib therapy (e.g. having 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00782067

  Show 31 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Additional Information:
Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.  This link exits the ClinicalTrials.gov site
Related Info  This link exits the ClinicalTrials.gov site

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: May 15, 2013
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
C-Findings
tyrosine kinase inhibitor
 KIT mutation
AHNMD
Systemic
Aggressive

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

ClinicalTrials.gov processed this record on June 18, 2013