Clinical Trial: Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
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 The ORR was defined as the percentage of participants who classified as confirmed responders (major response (MR) or partial response (PR). A major responder had complete resolution of at least one C-Finding and no progression in other C-Findings. A partial responder showed a measurable improvement in one or more C-Finding(s) without confirmed progression in other C-Findings. A C-Finding was a Clinical Finding, which was considered by the investigator and corroborated by the Study Steering Committee (SSC) Chairperson or designee, attributable to the mast cell disease component and not the associated hematological clonal non-mast cell lineage disease (AHNMD) component or any other cause.
Original Primary Outcome: 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 ]
Current Secondary Outcome:
- Duration of response [ Time Frame: 5 years ]
- Time to response [ Time Frame: 5 years ]
- Overall survival (OS) [ Time Frame: 5 years ]
Original Secondary Outcome:
- Duration of response
- Time to response
- Adverse event rate
- Overall survival (OS)
- T mutational status at diagnosis and after 6 cycles of therapy [ Time Frame: at diagnosis and after 6 cycles of therapy ]
Information By: Novartis
Dates:
Date Received: October 28, 2008
Date Started: October 13, 2008
Date Completion: July 10, 2017
Last Updated: March 8, 2017
Last Verified: March 2017
