Know Cancer

or
forgot password

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


Phase 2
18 Years
N/A
Open (Enrolling)
Both
Leukemia

Thank you

Trial Information

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


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

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall response rate according to established criteria by assessing clinical findings at the end of 6 cycles

Outcome Time Frame:

at the end of 6 cycles

Safety Issue:

No

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CPKC412D2201

NCT ID:

NCT00782067

Start Date:

October 2008

Completion Date:

July 2013

Related Keywords:

  • Leukemia
  • Aggressive systemic mastocytosis
  • mast cell leukemia
  • C-Findings
  • tyrosine kinase inhibitor
  • KIT mutation
  • AHNMD
  • Systemic
  • Aggressive
  • Aggression
  • Leukemia
  • Leukemia, Mast-Cell
  • Mastocytosis
  • Urticaria Pigmentosa
  • Mastocytoma
  • Mastocytosis, Systemic

Name

Location

University of PennsylvaniaPhiladelphia, Pennsylvania  19104
Oregon Health & Science University Dept. Hematologic MalignanciesPortland, Oregon  97239
Georgia Health Sciences University Dept.ofMedicalCollegeOfGeorgiaAugusta, Georgia  30912
University of California at Los Angeles Dept. of Hematology ClinicLos Angeles, California  90095
Dana Farber Cancer Institute Hematology / OncologyBoston, Massachusetts  02115
Stanford University Medical Center Stanford University 2Stanford, California  94305-5750
University of Colorado Dept of Univ. of ColoradoAurora, Colorado  80045
University of Michigan Comprehensive Cancer Center DeptofMichiganCancerCenter(3)Ann Arbor, Michigan  48109-0944
Memorial Sloan Kettering Cancer Center Dept. of MSKCC (2)New York, New York  10021
Virginia Commonwealth University SCRichmond, Virginia  23284