Trial · NCT00233454
Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia
NCT00233454, Phase 2, Completed, Jason Robert Gotlib; results first posted 2015-07-09
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia. Trials, record NCT00233454. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00233454
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia
- NCT number
- NCT00233454
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 26 participants, actual
- Lead sponsor
- Jason Robert Gotlib
- Conditions
- Systemic Mastocytosis, Aggressive (ASM), Leukemia, Mast Cell, Hematological Non-mast Cell Lineage Disease (AHNMD)
- Interventions
- Midostaurin
- First posted
- 2005-10-05
- Last updated
- 2018-09-20
- Results first posted
- 2015-07-09
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Subjects With Clinical Response [Partial Response (PR) + Complete Response (CR)] | 2 months | Clinical Response \[PR + CR\] will be assessed after 2 cycles of treatment, with each cycle being 28 days (4 weeks) in length. Except as otherwise noted, the minimum criteria for PR is improvement by at least 50% from the baseline value towards the indicated value for one or more of the criteria below: BONE MARROW \& BLOOD * ANC \<1000/uL * Hb \<10 g/dL * Platelets \>100,000/uL LIVER * If hepatomegaly with ascites, decrease in frequency of paracenteses by 50% * Elevated enzyme levels \> upper limit of normal (ULN) * Hypoalbuminemia \< ULN * Portal hypertension \> ULN SPLEEN * If palpable splenomegaly with hypersplenism/thrombocytopenia, hypersplenism markers improved GI TRACT * If malabsorption with hypoalbuminemia and/or weight loss, albumin improved BONES * If huge osteolyses or/and severe osteoporosis with pathologic fractures, partial resolution of osteolyses Subjects with PR or greater continue, those without response discontinue. |
| Secondary | Overall Survival (OS) | 11 months | Overall survival will be assessed after 12 cycles of treatment, with each cycle being 28 days (4 weeks) in length. 12 cycles of treatment is considered to be about 11 months. |
| Secondary | Overall Survival (OS) | 40 months | Overall survival was assessed as the median duration of survival at the time of data cut-off, and reported with 95% confidence interval. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Subjects With Clinical Response [Partial Response (PR) + Complete Response (CR)] | 2 months | participants | NUMBER | |
| SECONDARY | Overall Survival (OS) | 11 months | participants | NUMBER | |
| SECONDARY | Overall Survival (OS) | 40 months | months | MEDIAN |
Source ClinicalTrials.gov
Results by group3
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Subjects With Clinical Response [Partial Response (PR) + Complete Response (CR)] | Midostaurin | 25 | participants | ||
| Overall Survival (OS) | Midostaurin | 20 | participants | ||
| Overall Survival (OS) | Midostaurin | 40 | 95% Confidence Interval 27.3 to 52.7 | months |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Midostaurin | 26 | |
| Overall Study | 2 Months (for Response Rate) | Midostaurin | 26 | |
| Overall Study | COMPLETED | Midostaurin | 20 | |
| Overall Study | NOT COMPLETED | Midostaurin | 6 |
Source ClinicalTrials.gov
Adverse events156
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 | Anemia | Blood and lymphatic system disorders | Midostaurin | 11 | 26 | 12 |
| other | Blood and lymphatic system disorders - Other, lymphadenopathy | Blood and lymphatic system disorders | Midostaurin | 1 | 26 | 1 |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Midostaurin | 4 | 26 | 5 |
| other | Atrial fibrillation | Cardiac disorders | Midostaurin | 1 | 26 | 1 |
| other | Atrioventricular block first degree | Cardiac disorders | Midostaurin | 1 | 26 | 1 |
| other | Sinus bradycardia | Cardiac disorders | Midostaurin | 1 | 26 | 1 |
| other | Blurred vision | Eye disorders | Midostaurin | 3 | 26 | 3 |
| other | Cataract | Eye disorders | Midostaurin | 3 | 26 | 3 |
| other | Abdominal distension | Gastrointestinal disorders | Midostaurin | 7 | 26 | 7 |
| other | Abdominal pain | Gastrointestinal disorders | Midostaurin | 5 | 26 | 6 |
1 to 10 of 156Source ClinicalTrials.gov
See all 156 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Midostaurin | 7 | 26 |
| Total, other adverse events | Midostaurin | 26 | 26 |
| Total, serious adverse events | Midostaurin | 12 | 26 |
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/NCT00233454
GET https://conductscience.org/api/trials/v1/NCT00233454/events?page=2