Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
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|ClinicalTrials.gov Identifier: NCT00782067|
Recruitment Status : Completed
First Posted : October 29, 2008
Results First Posted : June 6, 2017
Last Update Posted : November 15, 2018
|Condition or disease||Intervention/treatment||Phase|
|Leukemia||Drug: Midostaurin (PKC412)||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||116 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||October 13, 2008|
|Actual Primary Completion Date :||December 1, 2014|
|Actual Study Completion Date :||August 24, 2017|
Experimental: Midostaurin (PKC412)
Midostaurin was administered at a dose of 100 mg twice daily (bid) in continuous cycles of 28 days until disease progression, intolerable toxicity or withdrawal due to any cause, whichever occurred first.
Drug: Midostaurin (PKC412)
Midostaurin was provided as 25 mg soft gelatin capsules for oral administration.
- Percentage of Participants With Overall Response Rate (ORR) [ Time Frame: 6 months ]
Overall Response Rate (ORR) was defined as the percentage of participants who classified as confirmed responders (Major Response (MR) or Partial Response (PR)) by the adjudication of the SSC and based on a Modified Valent Criteria.
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.
- Median Time to Duration of Response (DoR) [ Time Frame: Up 5 years ]The Duration of response (DoR) was defined as the time from first onset of confirmed response (MR or PR) to the date of first documented and confirmed progression or death due to ASM/MCL.
- Median Time to Response (TTR) [ Time Frame: Up 5 years ]The Time to response (TTR) was defined as the time from start of treatment until the date of onset of confirmed response (MR or PR).
- Median Time to Progression-Free Survival (PFS) [ Time Frame: Up 5 years ]The Progression-free survival (PFS) is defined as the time from start of treatment to the date of the first documented and confirmed progression or death due to any cause.
- Median Time to Overall Survival (OS) [ Time Frame: Up 5 years ]The Overall Survival (OS) is defined as the time from start of treatment to the date of death due to any cause.
- Long-term Safety and Tolerability of Midostaurin [ Time Frame: Up to 30 days after last dose of study treatment ]Analysis of frequencies for treatment emergent Adverse Event (AE), Serious Adverse Event (SAE) and Deaths by primary System Organ Class (SOC)
- Histopathologic Response [ Time Frame: Up 5 years ]Histopathologic response was summarized to demonstrate the change from baseline in percentage of mast cell infiltrations in the Bone Marrow (BM) and related serum tryptase levels.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00782067
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|Study Director:||Novartis Pharmaceuticals||Novartis Pharmaceuticals|