Trial Search Results

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

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.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Novartis Pharmaceuticals

Intervention(s):

  • Drug: Midostaurin (PKC412)

Phase:

Phase 2

Eligibility


Key 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

Key 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

Ages Eligible for Study

18 Years - N/A

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Cancer Clinical Trials Office
650-498-7061
Not Recruiting