blastic plasmacytoid dendritic cell neoplasm
Conditions
Interventions
Administer 12 mcg/kg/day by intravenous infusion over 15 minutes once daily for 5 consecutive days, followed by a 16-day treatment-free interval. This 21-day period constitutes one cycle, and administ
Sponsors
Higashioka Masaya
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) The Patient has histological and/or cytological evidence of BPDCN by pathologic assessment according to WHO classification (2022), that is either: - Previously untreated. - Persistent or recurrent in the peripheral blood, bone marrow, spleen, lymph nodes, skin, or other sites after previous treatment with at least 1 line of systemic therapy for BPDCN, e.g., hematopoietic stem cell transplant or chemotherapy. 2) Written informed consent has been obtained from a legally acceptable representative who has the capacity to consent, has received sufficient explanation, and fully understands the information, and who can respect the subject's will; Japanese male or female subjects aged 2 to 17 years at the time of consent by the representative, with assent obtained from the subject based on their free will whenever possible. 3) The patient has an ECOG PS of 0-2. 4) The patient has adequate baseline organ function, including cardiac, renal, and hepatic function: - Left ventricular ejection fraction (LVEF) >=institutional lower limit of normal as measured by multigated acquisition (MUGA) scan or echocardiography and no clinically significant abnormalities on a 12-lead electrocardiogram - Serum creatinine =3.2 g/dL - Bilirubin <=1.5 mg/dL - Aspartate transaminase (AST) and alanine transaminase (ALT) <=2.5 times the upper limit of normal (ULN) 5) Patients capable of complying with the study protocol requirements. 6) Patients of childbearing potential who agree to use appropriate contraception (e.g., barrier methods, contraceptive drugs) from the start of the treatment period until 2 months after the end or discontinuation of the treatment period. 7) The patient is expected to survive for 12 or longer weeks after the start of treatment.
Exclusion criteria
Exclusion criteria: 1) The patient has persistent clinically significant toxicities Grade >=2 from previous chemotherapy. 2) The patient has received treatment with chemotherapy, wide-field radiation, or biologic therapy within 14 days prior to treatment. 3) The patient has received treatment withstem hematopoietic cell transplant within 1 year prior to treatment. 4) The patient has received treatment with another study agent or unapproved drugs within 14 days prior to treatment.. 5) The patient has an active malignancy and/or cancer history (within 2 years prior to treatment.) that may confound the assessment of the study endpoints. 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 6) The patient has clinically significant cardiovascular disease (e.g., uncontrolled or any New York Heart Association Class 3 or 4 congestive heart failure, uncontrolled angina, history of myocardial infarction, unstable angina or stroke within 6 months prior to treatment., uncontrolled hypertension or clinically significant arrhythmias not controlled by medication). 7) The patient has uncontrolled, clinically significant pulmonary disease (e.g., chronic obstructive pulmonary disease, pulmonary hypertension) that in the opinion of the Investigator would put the patient at significant risk for pulmonary complications during the study. 8) The patient has known central nervous system (CNS) infiltration. If suspected, the patient with CNS infiltration should be ruled out with relevant imaging and/or examination of cerebrospinal fluid. 9) Patients receiving immunosuppressive agents for the treatment or prevention of graft-versus-host disease (GVHD), except for low-dose prednisolone (=<10 mg/day). If the patient has received immunosuppressive agents for the treatment or prevention of GVHD, administration of immunosuppressive agents must be discontinued at least 14 days prior to the start of study drug administration, and the absence of GVHD requiring treatment must be confirmed. 10) The patient has uncontrolled intercurrent illness. 11) The patient has known positive status for human immunodeficiency virus or active or chronic Hepatitis B or Hepatitis C. 12) The patient is oxygen-dependent. 13) Patients with a history of hypersensitivity to any component of the investigational drug or to proteins derived from Escherichia coli. 14) For female patients of childbearing potential, a pregnancy test must be performed during the observation period. Breastfeeding women may be enrolled if breastfeeding is discontinued; however, only patients who agree not to breastfeed during treatment and for one week after the final administration will be eligible. 15) Any other patients deemed inappropriate for participation by the principal or sub-investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pharmacokinetics, immunogenicity, safety, and efficacy | — |
Contacts
Public ContactOperations Clinical
Nippon Shinyaku Co., Ltd.
Outcome results
None listed