Trial · NCT01807598
Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia
NCT01807598, Phase 2, Completed, Jason Robert Gotlib; results first posted 2019-01-29
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia. Trials, record NCT01807598. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01807598
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia
- NCT number
- NCT01807598
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 10 participants, actual
- Lead sponsor
- Jason Robert Gotlib
- Conditions
- Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Systemic Mastocytosis
- Interventions
- Brentuximab vedotin
- First posted
- 2013-03-08
- Last updated
- 2019-01-29
- Results first posted
- 2019-01-29
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response Rate (ORR) Per Consensus International Response Criteria (Rate of Complete or Partial Remissions or Clinical Improvement) | Up to 1 year | Overall response rate (ORR) is sum of complete response (CR); partial response (PR); and clinical improvement (CI) in 1 year, ie, ORR=CR+PR+CI. The outcome is the number of participants without dispersion. CR is following with response duration(RD) ≥12 weeks (wk) * No mast cell disease * Tryptase \<20 ng/mL * Neutrophils ≥1x10e9/L with normal differential * Hemoglobin ≥11 g/dL * Platelets ≥100x10e9/L * No hepatosplenomegaly * No systemic mastocytosis(SM)-related organ damage PR is following with RD ≥12wk * Neither CR or progressive disease(PD) * Neoplastic mast cells reduced ≥50% * Tryptase reduced ≥50% * 1+ disease finding resolved CI is following with RD ≥12wk * Neither CR; PR; or PD * 1+ of findings above Stable disease (SD) Not CR, PR, Cl, or PD Progressive disease (PD) Any of: * Worsening organ damage * Doubling of laboratory abnormality * New transfusion dependence of ≥4 units red cells or platelets at 8wk •+ ≥100% in transfusions for 8wk * 10-cm+ splenomegaly |
| Secondary | Brentuximab Vedotin Toxicity | Up to 1 year | Toxicity was assessed as the number of adverse events considered possibly, probably, or definitely-related to treatment with brentuximab vedotin. The outcome is reported as the total number of related events, a number without dispersion, and the number of related events (without dispersion) considered to be either a hematologic toxicity or non-hematologic toxicity. |
| Secondary | Percent Change of CD30 Expression on Neoplastic Mast Cells | Baseline and up to 1 year | The presence of the CD30 marker (epitope) on neoplastic (cancerous) mast cells in core bone marrow biopsy samples was assessed by immunohistochemical methods, before and after brentuximab vedotin treatment. CD30 is a member of the TNF-receptor (TNF-R) superfamily, and is a transmembrane glycoprotein receptor that is normally at very low levels on the surface of activated T-cells. Overexpression of the CD30 marker is indicative of T-cell lymphoproliferative disorders and neoplastic mast cells, and the effect of a particular treatment on CD30 expression may be related to the efficacy of that treatment. The outcome is reported as the median percentage change in the level of CD30 detected, reported with full range. |
| Secondary | Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Total Symptom Score | Up to 1 year | The clinical effect of patient symptoms associated with systemic mastocytosis or mast cell leukemia were assessed with the patient-reported outcome survey entitled Myeloproliferative Neoplasm Symptom Assessment Form with Mast Cell Disorder Symptoms \[(MPN-SAF(MCD)\]. The MPN-SAF(MCD) is a 36-question survey, with possible responses ranged from 0 to 10, with 0 indicating not present or no effect; 1 meaning present but most favorable; and 10 meaning worst imaginable (least favorable). Total range is 0 to 360, with 0 being best possible, and 360 being worst possible. Total symptom score is calculated as the sum of the individual survey scores reported at baseline and after treatment, with lower numbers indicating less effect, and larger numbers indicated greater effect. The outcome is reported as mean score value with dispersion. |
| Secondary | Quality of Life (QoL) Score Using a Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) | Up to 1 year | Overall quality of life (QoL) was assessed by a single specific question from the 36-question Myeloproliferative Neoplasm Symptom Assessment Form with Mast Cell Disorder Symptoms \[(MPN-SAF(MCD)\] survey. Possible responses for theQoL question range from 0 to 10, with 0 indicating "as good as it can be" (most favorable), and 10 meaning "as bad as it can be" (least favorable). The QoL score was obtained at baseline and after treatment. The outcome is reported as mean score with dispersion. |
| Secondary | Duration of Response (DOR) | Up to 1 year | Duration of response (DOR) is defined as the time from the start of the first confirmed response until the date of the first documented and confirmed disease progression or death due to ASM or MCL. For participants with a confirmed clinical response, the outcome was to be reported as the median DOR with standard deviation. |
| Secondary | Time to Response (TTR) | Up to 1 year | Time to response (TTR) is defined as the time from the date of start of treatment to the date of first confirmed response. For participants with a confirmed clinical response, the outcome was to be reported as the median TTR with standard deviation. |
| Secondary | Progression-free Survival (PFS) | Up to 1 year | Progression-free survival (PFS) is defined as the time from the date of start of treatment until disease progression; death; or initiation of new therapy. The outcome is reported as the mean number of days of PFS, with 95% confidence interval. Progressive disease (PD) is any of the following: * Worsening of any organ damage * Doubling of any laboratory abnormality * New transfusion dependence of ≥ 4 units red cells or platelets at 8 wk * ≥ 100% increase in transfusions for 8 wk * 10-cm+ splenomegaly |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response Rate (ORR) Per Consensus International Response Criteria (Rate of Complete or Partial Remissions or Clinical Improvement) | Up to 1 year | 1 of 10 participants were not evaluable due to early death during Cycle 1 (considered unrelated to study treatment) | participants | NUMBER |
| SECONDARY | Brentuximab Vedotin Toxicity | Up to 1 year | Related adverse events | NUMBER | |
| SECONDARY | Percent Change of CD30 Expression on Neoplastic Mast Cells | Baseline and up to 1 year | CD30 marker data was not obtained for some participants. | percentage change in CD30+ cells | MEDIAN |
| SECONDARY | Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Total Symptom Score | Up to 1 year | score on a scale | MEAN | |
| SECONDARY | Quality of Life (QoL) Score Using a Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) | Up to 1 year | score on a scale | MEAN | |
| SECONDARY | Duration of Response (DOR) | Up to 1 year | Duration of response (DOR) can not be determined if no participants had a response. | ||
| SECONDARY | Time to Response (TTR) | Up to 1 year | Time to response (TTR) can not be determined if no participants had a response. | ||
| SECONDARY | Progression-free Survival (PFS) | Up to 1 year | Days | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Response Rate (ORR) Per Consensus International Response Criteria (Rate of Complete or Partial Remissions or Clinical Improvement) | Brentuximab Vedotin | 0 | participants | ||
| Brentuximab Vedotin Toxicity | Brentuximab Vedotin | Total related adverse events (toxicities) | 36 | Related adverse events | |
| Brentuximab Vedotin Toxicity | Brentuximab Vedotin | Related hematologic toxicity | 12 | Related adverse events | |
| Brentuximab Vedotin Toxicity | Brentuximab Vedotin | Related non-hematologic toxicity | 24 | Related adverse events | |
| Percent Change of CD30 Expression on Neoplastic Mast Cells | Brentuximab Vedotin | -1 | Full Range -62 to 45 | percentage change in CD30+ cells | |
| Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Total Symptom Score | Brentuximab Vedotin | Baseline (Pre-treatment) | 36.2 | Standard Deviation 18.1 | score on a scale |
| Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Total Symptom Score | Brentuximab Vedotin | After Treatment | 23.6 | Standard Deviation 15.6 | score on a scale |
| Quality of Life (QoL) Score Using a Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) | Brentuximab Vedotin | Baseline (Pre-treatment) | 5 | Standard Deviation 2.7 | score on a scale |
| Quality of Life (QoL) Score Using a Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) | Brentuximab Vedotin | After Treatment | 2.6 | Standard Deviation 2.7 | score on a scale |
| Progression-free Survival (PFS) | Brentuximab Vedotin | 210 | 95% Confidence Interval 77 to 343 | Days |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Brentuximab Vedotin | 10 | |
| Overall Study | COMPLETED | Brentuximab Vedotin | 4 | |
| Overall Study | NOT COMPLETED | Brentuximab Vedotin | 6 |
Source ClinicalTrials.gov
Adverse events42
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 | Neutropenia | Blood and lymphatic system disorders | Brentuximab Vedotin | 3 | 10 | 11 |
| other | Anemia | Blood and lymphatic system disorders | Brentuximab Vedotin | 2 | 10 | 3 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Brentuximab Vedotin | 1 | 10 | 1 |
| other | Fatigue | General disorders | Brentuximab Vedotin | 4 | 10 | 9 |
| other | Abdominal Pain | Gastrointestinal disorders | Brentuximab Vedotin | 2 | 10 | 4 |
| other | Anorexia | Metabolism and nutrition disorders | Brentuximab Vedotin | 2 | 10 | 3 |
| other | Constipation | Gastrointestinal disorders | Brentuximab Vedotin | 2 | 10 | 2 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Brentuximab Vedotin | 2 | 10 | 2 |
| other | Depression | Psychiatric disorders | Brentuximab Vedotin | 2 | 10 | 2 |
| other | Diarrhea | Gastrointestinal disorders | Brentuximab Vedotin | 2 | 10 | 3 |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Brentuximab Vedotin | 1 | 10 |
| Total, other adverse events | Brentuximab Vedotin | 10 | 10 |
| Total, serious adverse events | Brentuximab Vedotin | 4 | 10 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 31350306 | DERIVED |
Source PubMed
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/NCT01807598
GET https://conductscience.org/api/trials/v1/NCT01807598/events?page=2