Tagraxofusp in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm or Acute Myeloid Leukemia (NCT02113982) | Clinical Trial Compass
CompletedPhase 1/2
Tagraxofusp in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm or Acute Myeloid Leukemia
United States138 participantsStarted 2014-09
Plain-language summary
This is a 4-stage, non-randomized, open-label, dose escalation and expansion, multicenter study. A cycle of therapy is 21 days. Stage 1 was a dose-escalation stage. During Stages 2-4, patients are treated at the MTD or maximum tested dose at which multiple DLTs are not observed during Stage 1.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. The patient has a diagnosis of AML (Protocol Stages 1 and 2) or BPDCN (Protocol Stages 1-4) according to WHO classification (AML; excluding acute promyelocytic leukemia \[APL, FAB M3\]) or confirmed by hematopathology (BPDCN) (Facchetti et al. 2008).
✓. The patient must meet one of the following (a) or (b) or (c):
✓. Has evidence of persistent or recurrent AML (Protocol Stages 1 and 2) in the peripheral blood and/or bone marrow that is refractory to, or has relapsed from, their most recent prior line of treatment.
✓. Has previously untreated AML (Protocol Stages 1 and 2) and is considered to be at high risk for disease progression and/or is unlikely to derive more than transient benefit from standard therapy by having at least one of the following:
✓. Has histological and/or cytological evidence of BPDCN by pathologic assessment at the investigative site according to WHO classification (Facchetti et al. 2008) by a pathologist with expertise in hematologic malignancies, that can be measured for treatment response and is either:
✓. The patient is ≥ 18 years old.
✓. The patient has an ECOG performance score (PS) of 0-2.
✓. The patient has adequate baseline organ function, including cardiac, renal, and hepatic function:
Exclusion criteria
✕. The patient has a diagnosis of acute promyelocytic leukemia (APL; FAB M3).
✕. The patient has persistent clinically significant toxicities Grade ≥ 2 from previous chemotherapy (excluding alopecia, nausea, fatigue, and liver function tests (as mandated in the inclusion criteria)).
What they're measuring
1
MTD (Stage 1)
Timeframe: 21-day period after the first dose (cycle 1)
2
CR Rate in First-line BPDCN (Stage 3, Pivotal Cohort)
Timeframe: at pre-defined treatment cycle intervals from randomization up to disease progression, up to 3.5 years
✕. The patient has received treatment with chemotherapy, wide-field radiation, or biologic therapy within 14 days of study entry.
✕. The patient has received treatment with another investigational agent within 14 days of study entry.
✕. The patient has previously received treatment with Tagraxofusp.
✕. The patient has an active malignancy and/or cancer history (excluding AML, BPDCN, or antecedent MDS) that may confound the assessment of the study endpoints. Patients with a past cancer history (within 2 years of entry) with substantial potential for recurrence and/or ongoing active malignancy must be discussed with the Sponsor before study entry. Patients with the following neoplastic diagnoses are eligible: non-melanoma skin cancer, carcinoma in situ, cervical intraepithelial neoplasia, organ-confined prostate cancer with no evidence of progressive disease.
✕. The patient has clinically significant cardiovascular disease (e.g., uncontrolled or any NYHA Class 3 or 4 congestive heart failure, uncontrolled angina, history of myocardial infarction or stroke within 6 months of study entry, uncontrolled hypertension or clinically significant arrhythmias not controlled by medication).
✕. The patient has uncontrolled, clinically significant pulmonary disease (e.g., COPD, pulmonary hypertension) that in the opinion of the investigator would put the patient at significant risk for pulmonary complications during the study.