Phase 1/2 study of NS-401 in patients with blastic plasmacytoid dendritic cell neoplasm

JRCT ID: jRCT2031220023

Registered date:16/04/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: blastic plasmacytoid dendritic cell neoplasm
• Date of first enrollment: 06/07/2022
• Target sample size: 13
• Study type: Interventional
• Intervention(s): NS-401 at a dose of at 12mcg/kg/day administered by IV infusion for 5 consecutive days followed by 16-day resting period per 21-day cycles.

Outcome(s)

• Primary Outcome: to evaluate the pharmacokinetic, tolerability (DLT) , safety and efficacy (complete remission rate)

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) The Patient has histological and/or cytological evidence of BPDCN by pathologic assessment according to WHO classification (2017), 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) The patient is >=18 years old.
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 <=1.5 mg/dL
- Serum albumin >=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) The patient must agree to use acceptable contraceptive methods for the duration of the study and for at least 2 months after the discontinuation of study treatment.
6) The patient is expected to survive for 12 or longer weeks after the start of treatment.

Exclude 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 investigational 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)The patient is receiving immunosuppressive therapy - with the exception of low-dose prednisone (<=10 mg/day) - for treatment or prophylaxis of graft-versus-host disease (GVHD). If the patient has been on immunosuppressive treatment or prophylaxis for GVHD, the treatment(s) must have been discontinued at least 14 days prior to treatment and there must be no evidence of Grade >=2 GVHD.
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)The patient is pregnant or breast feeding

Related Information

• Primary Sponsor: Higashioka Masaya