Phase II Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
Novartis
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Jason Robert Gotlib, Stanford University
ClinicalTrials.gov Identifier:
NCT00233454
First received: October 3, 2005
Last updated: June 16, 2015
Last verified: June 2015
Results First Received: June 16, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Systemic Mastocytosis, Aggressive (ASM)
Leukemia, Mast Cell
Hematological Non-mast Cell Lineage Disease (AHNMD)
Intervention: Drug: Midostaurin

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Midostaurin

100 mg midostaurin twice daily as oral capsules

Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR


Participant Flow:   Overall Study
    Midostaurin  
STARTED     26  
2 Months (for Response Rate)     26  
COMPLETED     20  
NOT COMPLETED     6  
Death                 6  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Midostaurin

100 mg midostaurin twice daily as oral capsules

Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR


Baseline Measures
    Midostaurin  
Number of Participants  
[units: participants]
  26  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     13  
>=65 years     13  
Age  
[units: years]
Median (Full Range)
  65   (24.8 to 79.6)  
Gender  
[units: participants]
 
Female     11  
Male     15  
Region of Enrollment  
[units: participants]
 
United States     26  



  Outcome Measures
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1.  Primary:   Subjects With Clinical Response [Partial Response (PR) + Complete Response (CR)]   [ Time Frame: 2 months ]

2.  Secondary:   Overall Survival (OS)   [ Time Frame: 11 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Jason R Gotlib, MD, Associate Professor of Medicine (Hematology)
Organization: Stanford University Medical Center
phone: 650-867-2823
e-mail: jason.gotlib@stanford.edu


Publications of Results:
Gotlib JR, George TI, Linder A, et al. "Phase II Trial of the Tyrosine Kinase Inhibitor PKC412 in Advanced Systemic Mastocytosis: Preliminary Results." Blood. 16 November 2006;108(11)16:abs3609
Gotlib JR, George TI, Corless C, et al. "The KIT Tyrosine Kinase Inhibitor Midostaurin (PKC412) Exhibits a High Response Rate in Aggressive Systemic Mastocytosis(ASM): Interim Results of a Phase 2 Trial." Blood. 16 November 2007;110(11):abs 3536
Gotlib JR, DeAngelo DJ, George TI, et al. "KIT Inhibitor Midostaurin Exhibits a High Rate of Clinically Meaningful and Durable Responses in Advanced Systemic Mastocytosis: Report of a Fully Accrued Phase II Trial." Blood. 19 November 2010;116(21):abs316

Other Publications:

Responsible Party: Jason Robert Gotlib, Stanford University
ClinicalTrials.gov Identifier: NCT00233454     History of Changes
Other Study ID Numbers: IRB-13704, 95242, HEMMPD0003, CPKC412D2201, 2213
Study First Received: October 3, 2005
Results First Received: June 16, 2015
Last Updated: June 16, 2015
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board
United States: Data and Safety Monitoring Board