Trial · NCT02113982
Tagraxofusp in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm or Acute Myeloid Leukemia
NCT02113982, Phase 1, Phase 2, Completed, Stemline Therapeutics, Inc.; results first posted 2024-08-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Tagraxofusp in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm or Acute Myeloid Leukemia. Trials, record NCT02113982. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02113982
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Tagraxofusp in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm or Acute Myeloid Leukemia
- NCT number
- NCT02113982
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 138 participants, actual
- Lead sponsor
- Stemline Therapeutics, Inc.
- Conditions
- Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), Acute Myeloid Leukemia (AML)
- Interventions
- Tagraxofusp
- First posted
- 2014-04-15
- Last updated
- 2024-08-01
- Results first posted
- 2024-08-01
- Limitations and caveats
This study was limited by lack of randomization due to the ultra-rare nature of blastic plasmacytoid dendritic cell neoplasm (BPDCN).~Patients lacked established response criteria for BPDCN prior to STML-401-0114.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | MTD (Stage 1) | 21-day period after the first dose (cycle 1) | Maximum tolerated dose (MTD) in both patients with BPDCN and AML. MTD defined as the highest dose level where less than 2/3 or 2/6 patients experienced a DLT |
| Primary | CR Rate in First-line BPDCN (Stage 3, Pivotal Cohort) | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 3.5 years | Complete response (CR) rate, defined as the percentage who achieved CR+ CR(clinical) with minimal residual skin abnormality (CRc) after treatment with Tagraxofusp. CR criteria according to Cheson BD et al, The International Harmonization Project on Lymphoma. Revised response criteria for malignant lymphoma. J Clin Oncol 2007;25:579-586: Marrow: normalization of blast percentage (≤5%). Peripheral blood: normalization of neutrophil count (≥ 1,000/µL) and platelet count (≥ 100,000/µL) - absence of leukemic blasts Skin: 100% clearance of all skin lesions from baseline; no new lesions in patients without lesions at baseline Nodal masses: regression to normal size on CT Spleen, liver: not palpable, nodules disappeared CRc criteria: all the above except for skin: marked clearance of all skin lesions from baseline; residual hyperpigmentation or abnormality with BPDCN identified on biopsy (or no biopsy performed) |
| Secondary | CR Rate in First-line BPDCN, R/R BPDCN and AML | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 5 years | CR rate, defined as the percentage of patients who achieved CR+ CR with minimal residual skin abnormality (CRc) after treatment with Tagraxofusp. CR criteria according to Cheson BD et al, The International Harmonization Project on Lymphoma. Revised response criteria for malignant lymphoma. J Clin Oncol 2007;25:579-586: Marrow: normalization of blast percentage (≤5%) Peripheral blood: normalization of neutrophil count (≥ 1,000/µL) and platelet count (≥ 100,000/µL) - absence of leukemic blasts Skin: 100% clearance of all skin lesions from baseline; no new lesions in patients without lesions at baseline Nodal masses: regression to normal size on CT Spleen, liver: not palpable, nodules disappeared CRc criteria: all the above except for skin: marked clearance of all skin lesions from baseline; residual hyperpigmentation or abnormality with BPDCN identified on biopsy (or no biopsy performed) |
| Secondary | Duration of CR in First-line BPDCN, R/R BPDCN and AML | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 5 years | Duration of complete response (CR) defined as the time from when the criteria are first met for CR+CRc (whichever was recorded first) until the date that the criteria for relapse after CR+CRc are met, according to the previously reported criteria for CR and CRc and following criteria of relapse after CR+CRc (Cheson BD et al. J Clin Oncol 2007;25:579-586.): Marrow: blast percentage \>5% (if no peripheral blasts, then confirmation aspirate required ≥ 1week later) Peripheral blood: presence of leukemic blasts Skin: increase in skin score greater than the sum of nadir plus 50% baseline score Nodal masses: appearance of a new lesion(s) \>1.5 cm in any axis, ≥50% increase from nadir in SPD of more than one node, or ≥50% increase from nadir in longest diameter of a previously identified node \>1cm in short axis Spleen, liver: \>50% increase from nadir in the SPD of any previous lesions |
| Secondary | ORR in First-line BPDCN, R/R BPDCN and AML | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 5 years | Objective response rate (ORR) defines as the percentage of patients who achieved CR (CR+CRc), CR with incomplete blood count recovery (CRi), or partial response (PR) after treatment with Tagraxofusp according to the previously reported definitions of CR, CRc and the following one of CRi and PR: CRi: CR with incomplete of neutrophil and/or platelet count and absence of leukemic blast in the peripheral blood Marrow: PR defined as decrease by ≥50% in blast percentage to 5-25% Peripheral blood: PR defined as normalization of neutrophil count (≥ 1,000/µL) and platelet count (≥ 100,000/µL) Skin: 50%-\<100% clearance of all skis lesions from baseline; no new lesions in patients without lesions at baseline Nodal masses: ≥50% decrease in SPD of up to 6 largest dominant masses; no increase in size of other nodes Spleen, liver: ≥50% in SPD of nodules (for single nodule in greatest transverse diameter); no increase in size of liver or spleen |
| Secondary | OS in First-line BPDCN, R/R BPDCN and AML | From first infusion to treatment discontinuation when survival is assessed every 90 days up to end of follow-up or death, up to 5 years | Overall survival (months) defined as the time from the date of first infusion of Tagraxofusp to the date of death from any cause |
| Secondary | Bridge to SCT in First-line BPDCN, R/R BPDCN and AML | at pre-defined intervals up to achievement of outcome enabling an SCT for a period, up to 5 years | Bridge to Stem Cell Transplant (SCT) defined as the percentage of patients who received SCT subsequent to achieving an Investigator-determined outcome from Tagraxofusp that was deemed by the Investigator to enable an SCT |
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 | MTD (Stage 1) | 21-day period after the first dose (cycle 1) | Patients with First-line Blastic Plasmacytoid Dendritic Cell Neoplasm, R/R Blastic Plasmacytoid Dendritic Cell Neoplasm and Acute Myeloid Leukemia participating to stage 1 (dose-escalation) | µg/kg/day | NUMBER |
| PRIMARY | CR Rate in First-line BPDCN (Stage 3, Pivotal Cohort) | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 3.5 years | Pre-defined efficacy primary end point assessed in Stage 3, standalone, pivotal efficacy cohort of only patients with first-line BPDCN exposed to 12 μg/kg/day were enrolled. | percentage of participants | NUMBER |
| SECONDARY | CR Rate in First-line BPDCN, R/R BPDCN and AML | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 5 years | Efficacy population including evaluable patients treated at 12 μg/kg/day in the first-line BPDCN, R/R BPDCN and AML. | percentage of evaluable patients | NUMBER |
| SECONDARY | Duration of CR in First-line BPDCN, R/R BPDCN and AML | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 5 years | Efficacy population including evaluable patients treated at 12 μg/kg/day in the first-line BPDCN, R/R BPDCN and AML | months | MEDIAN |
| SECONDARY | ORR in First-line BPDCN, R/R BPDCN and AML | at pre-defined treatment cycle intervals from randomization up to disease progression, up to 5 years | Efficacy population including evaluable patients treated at 12 μg/kg/day in the first-line BPDCN, R/R BPDCN and AML | percentage of evaluable patients | NUMBER |
| SECONDARY | OS in First-line BPDCN, R/R BPDCN and AML | From first infusion to treatment discontinuation when survival is assessed every 90 days up to end of follow-up or death, up to 5 years | Efficacy population including evaluable patients treated at 12 μg/kg/day in the first-line BPDCN, R/R BPDCN and AML | Months | MEDIAN |
| SECONDARY | Bridge to SCT in First-line BPDCN, R/R BPDCN and AML | at pre-defined intervals up to achievement of outcome enabling an SCT for a period, up to 5 years | Efficacy population including evaluable patients treated at 12 μg/kg/day in the first-line BPDCN, R/R BPDCN and AML | percentage of evaluable patients | NUMBER |
Source ClinicalTrials.gov
Results by group19
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| MTD (Stage 1) | First-line BPDCN | 12 | µg/kg/day | ||
| MTD (Stage 1) | R/R BPDCN | 12 | µg/kg/day | ||
| MTD (Stage 1) | Acute Myeloid Leukemia | 16 | µg/kg/day | ||
| CR Rate in First-line BPDCN (Stage 3, Pivotal Cohort) | First-line BPDCN | 53.8 | 95% Confidence Interval 25.1 to 80.8 | percentage of participants | |
| CR Rate in First-line BPDCN, R/R BPDCN and AML | First-line BPDCN | 56.9 | 95% Confidence Interval 44 to 69.2 | percentage of evaluable patients | |
| CR Rate in First-line BPDCN, R/R BPDCN and AML | R/R BPDCN | 15.8 | 95% Confidence Interval 3.4 to 39.6 | percentage of evaluable patients | |
| CR Rate in First-line BPDCN, R/R BPDCN and AML | Acute Myeloid Leukemia | 3 | 95% Confidence Interval 0.1 to 14.2 | percentage of evaluable patients | |
| Duration of CR in First-line BPDCN, R/R BPDCN and AML | First-Line BPDCN | 24.9 | Full Range 0.99 to 57.36 | months | |
| Duration of CR in First-line BPDCN, R/R BPDCN and AML | R/R BPDCN | 3.6 | Full Range 3.02 to 13.93 | months | |
| Duration of CR in First-line BPDCN, R/R BPDCN and AML | Acute Myeloid Leukemia | 8.6 | Full Range NA to NA | months |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 results by groupParticipant flow168
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Stage 1: Dose Escalation | STARTED | Stage 1 - AML Indication: 7 µg/kg/Day Dosage | 3 | |
| Stage 1: Dose Escalation | STARTED | Stage 1 - AML Indication: 9 µg/kg/Day Dosage | 3 | |
| Stage 1: Dose Escalation | STARTED | Stage 1 - AML Indication: 12 µg/kg/Day Dosage | 2 | |
| Stage 1: Dose Escalation | STARTED | Stage 1 - AML Indication: 16 µg/kg/Day Dosage | 6 | |
| Stage 1: Dose Escalation | STARTED | 1L - Stage 1 - BPDCN Indication: 7 µg/kg/Day Dosage | 3 | |
| Stage 1: Dose Escalation | STARTED | 1L - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage | 3 | |
| Stage 1: Dose Escalation | STARTED | R/R - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage | 3 | |
| Stage 1: Dose Escalation | STARTED | Stage 2 - AML Indication: 12 µg/kg/Day Dosage | 0 | |
| Stage 1: Dose Escalation | STARTED | Stage 2 - AML Indication: 16 µg/kg/Day Dosage | 0 | |
| Stage 1: Dose Escalation | STARTED | 1L - Stage 2 - BPDCN Indication: 12 µg/kg/Day Dosage | 0 |
1 to 10 of 168Source ClinicalTrials.gov
See all 168 participant flowAdverse events3,192
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 | Stage 1 - AML Indication: 7 µg/kg/Day Dosage | 1 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Stage 1 - AML Indication: 9 µg/kg/Day Dosage | 0 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Stage 1 - AML Indication: 12 µg/kg/Day Dosage | 0 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Stage 1 - AML Indication: 16 µg/kg/Day Dosage | 4 | 6 | |
| other | Anaemia | Blood and lymphatic system disorders | 1L - Stage 1 - BPDCN Indication: 7 µg/kg/Day Dosage | 1 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | 1L - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage | 1 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | R/R - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage | 1 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Stage 2 - AML Indication: 12 µg/kg/Day Dosage | 8 | 34 | |
| other | Anaemia | Blood and lymphatic system disorders | Stage 2 - AML Indication: 16 µg/kg/Day Dosage | 0 | 1 | |
| other | Anaemia | Blood and lymphatic system disorders | 1L - Stage 2 - BPDCN Indication: 12 µg/kg/Day Dosage | 3 | 13 |
1 to 10 of 3,192Source ClinicalTrials.gov
See all 3,192 adverse eventsAdverse event totals42
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Stage 1 - AML Indication: 7 µg/kg/Day Dosage | 0 | 3 |
| Total, all-cause mortality | Stage 1 - AML Indication: 9 µg/kg/Day Dosage | 0 | 3 |
| Total, all-cause mortality | Stage 1 - AML Indication: 12 µg/kg/Day Dosage | 1 | 2 |
| Total, all-cause mortality | Stage 1 - AML Indication: 16 µg/kg/Day Dosage | 4 | 6 |
| Total, all-cause mortality | 1L - Stage 1 - BPDCN Indication: 7 µg/kg/Day Dosage | 1 | 3 |
| Total, all-cause mortality | 1L - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage | 0 | 3 |
| Total, all-cause mortality | R/R - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage | 1 | 3 |
| Total, all-cause mortality | Stage 2 - AML Indication: 12 µg/kg/Day Dosage | 3 | 34 |
| Total, all-cause mortality | Stage 2 - AML Indication: 16 µg/kg/Day Dosage | 0 | 1 |
| Total, all-cause mortality | 1L - Stage 2 - BPDCN Indication: 12 µg/kg/Day Dosage | 1 | 13 |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 adverse event totalsPapers3
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/NCT02113982
GET https://conductscience.org/api/trials/v1/NCT02113982/flow?page=2