Trial · NCT00782067
Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
NCT00782067, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2017-06-06
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia. Trials, record NCT00782067. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00782067
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Midostaurin in Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia
- NCT number
- NCT00782067
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 116 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Leukemia
- Interventions
- Midostaurin (PKC412)
- First posted
- 2008-10-29
- Last updated
- 2018-11-15
- Other registries' IDs
- eudract:2008-000280-42
- Results first posted
- 2017-06-06
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Overall Response Rate (ORR) | 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. |
| Secondary | Median Time to Duration of Response (DoR) | 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. |
| Secondary | Median Time to Response (TTR) | 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). |
| Secondary | Median Time to Progression-Free Survival (PFS) | 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. |
| Secondary | Median Time to Overall Survival (OS) | 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. |
| Secondary | Long-term Safety and Tolerability of Midostaurin | 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) |
| Secondary | Histopathologic Response | 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. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Overall Response Rate (ORR) | 6 months | Primary Efficacy Population: participants assigned to study treatment who met diagnostic criteria for aggressive systemic mastocytosis or mast cell leukemia and presented with at least 1 measurable C-Finding at study entry and/or participants with transfusion dependent anemia due to their underlying disease at study entry as confirmed by the SCC. | Percentage of participants | NUMBER |
| SECONDARY | Median Time to Duration of Response (DoR) | Up 5 years | Primary Efficacy Population (PEP), which consisted of all participants with a confirmed response, was considered. | Months | MEDIAN |
| SECONDARY | Median Time to Response (TTR) | Up 5 years | Primary Efficacy Population (PEP), which consisted of all participants with a confirmed response, was considered. | Months | MEDIAN |
| SECONDARY | Median Time to Progression-Free Survival (PFS) | Up 5 years | Primary Efficacy Population (PEP), which consisted of all participants with a confirmed response, was considered. | Months | MEDIAN |
| SECONDARY | Median Time to Overall Survival (OS) | Up 5 years | Primary Efficacy Population (PEP), which consisted of all participants with a confirmed response and the Full Analysis Set (FAS), which consisted of all patients who received at least one dose of study drug were considered. | Months | MEDIAN |
| SECONDARY | Long-term Safety and Tolerability of Midostaurin | Up to 30 days after last dose of study treatment | Safety Set (SS) | Percentage of participants | NUMBER |
| SECONDARY | Histopathologic Response | Up 5 years | Primary Efficacy Population (PEP); for each patient, the best improvement relative to Baseline was considered. | Percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group13
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Overall Response Rate (ORR) | Midostaurin (PKC412) | 59.6 | 95% Confidence Interval 48.6 to 69.8 | Percentage of participants | |
| Median Time to Duration of Response (DoR) | Midostaurin (PKC412) | 31.4 | 95% Confidence Interval 10.8 to NA | Months | |
| Median Time to Response (TTR) | Midostaurin (PKC412) | 0.3 | Full Range 0.1 to 3.7 | Months | |
| Median Time to Progression-Free Survival (PFS) | Midostaurin (PKC412) | 17.0 | 95% Confidence Interval 10.2 to 24.8 | Months | |
| Median Time to Overall Survival (OS) | Midostaurin (PKC412) | Primary Efficacy Population (PEP) (n=89) | 26.8 | 95% Confidence Interval 17.6 to 34.4 | Months |
| Median Time to Overall Survival (OS) | Midostaurin (PKC412) | Full Analysis Set (FAS) (n=116) | 28.7 | 95% Confidence Interval 20.3 to 38.0 | Months |
| Long-term Safety and Tolerability of Midostaurin | Midostaurin (PKC412) | AEs by Primary System Organ Class (SOC) (n=116) | 100 | Percentage of participants | |
| Long-term Safety and Tolerability of Midostaurin | Midostaurin (PKC412) | SAEs by Primary System Organ Class (SOC) (n=88) | 75.9 | Percentage of participants | |
| Long-term Safety and Tolerability of Midostaurin | Midostaurin (PKC412) | Deaths by Primary System Organ Class (SOC) (n=25) | 21.6 | Percentage of participants | |
| Histopathologic Response | Midostaurin (PKC412) | >50% decrease in mast cell (MC) (n=41) | 46.1 | Percentage of participants |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Midostaurin (PKC412) | 116 | |
| Overall Study | Safety Set (SS) | Midostaurin (PKC412) | 116 | |
| Overall Study | Primary Efficacy Population (PEP) | Midostaurin (PKC412) | 89 | |
| Overall Study | COMPLETED | Midostaurin (PKC412) | 0 | |
| Overall Study | NOT COMPLETED | Midostaurin (PKC412) | 116 |
Source ClinicalTrials.gov
Adverse events255
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Midostaurin (PKC412) | 39 | 116 | |
| other | Leukopenia | Blood and lymphatic system disorders | Midostaurin (PKC412) | 7 | 116 | |
| other | Neutropenia | Blood and lymphatic system disorders | Midostaurin (PKC412) | 17 | 116 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Midostaurin (PKC412) | 23 | 116 | |
| other | Vertigo | Ear and labyrinth disorders | Midostaurin (PKC412) | 6 | 116 | |
| other | Abdominal distension | Gastrointestinal disorders | Midostaurin (PKC412) | 7 | 116 | |
| other | Abdominal pain | Gastrointestinal disorders | Midostaurin (PKC412) | 34 | 116 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Midostaurin (PKC412) | 10 | 116 | |
| other | Ascites | Gastrointestinal disorders | Midostaurin (PKC412) | 9 | 116 | |
| other | Constipation | Gastrointestinal disorders | Midostaurin (PKC412) | 29 | 116 |
1 to 10 of 255Source ClinicalTrials.gov
See all 255 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Midostaurin (PKC412) | 25 | 116 |
| Total, other adverse events | Midostaurin (PKC412) | 115 | 116 |
| Total, serious adverse events | Midostaurin (PKC412) | 88 | 116 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00782067
GET https://conductscience.org/api/trials/v1/NCT00782067/events?page=2