Multicentre, open-label, dose-escalation phase I/II study to evaluate the tolerability, safety, efficacy and pharmacokinetics of repeated continuous intravenous PPMX-T003 in patients with aggressive NK cell leukaemia (ANKL) (physician-initiated clinical trial).

JRCT ID: jRCT2061230008

Registered date:28/04/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Aggressive NK-cell Leukemia
• Date of first enrollment: 21/09/2023
• Target sample size: 7
• Study type: Interventional

Intervention(s)

Repeated and continuous intravenous administration of the investigational drug (PPMX-T003).
Multiple starting doses.

Outcome(s)

• Primary Outcome: Tolerability and safety of PPMX-T003 repeated continuous intravenous administration (first course)

Secondary Outcome

Tolerability and safety of PPMX-T003 repeated continuous intravenous administration (including DLT evaluation after the second course)
Efficacy of PPMX-T003 repeated continuous intravenous infusion
Serum drug concentrations during repeated and continuous intravenous PPMX-T003 administration
Anti-drug antibody production

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: < 75age old
• Gender: Both

Include criteria

1) Patients who are at least 18 years old and less than 75 years old at the time of obtaining consent
2) Patients confirmed to have been diagnosed with ANKL (regardless of whether the disease is first or recurrent) based on diagnostic criteria developed with reference to the World Health Organisation (WHO) 4th edition (2017) criteria.
3) Patients with abnormal liver function or hepatomegaly due to the primary disease
4) Patients with an Eastern Cooperative Oncology Group (ECOG) performance status(PS) of 3 or less
5) For women of childbearing potential, women of childbearing potential and men, patients who agree to use adequate contraception from the time consent is obtained until 24 weeks after the last dose.
6) Patients for whom written consent to participate in this clinical trial has been obtained from the individual or a surrogate.

Exclude criteria

1) Patients eligible to receive chemotherapy as treatment for ANKL
2) Patients whose tests performed at screening show abnormalities not attributable to the underlying disease (ANKL) and who have been judged by the investigator or others to be problematic for study participation (e.g. physical examination, standard 12-lead ECG, chest radiography, haematology, blood chemistry, urine analysis, immunological tests, etc.)
3) Patients treated with other investigational drugs or unapproved therapies within 16 weeks prior to enrolment
4) Patients who, at the time of screening, had not received hepatitis B virus surface antigen (HBsAg), hepatitis B virus core antibody (HBcAb), hepatitis B virus surface antibody (HBcAb), hepatitis B virus core antibody (HBcAb), hepatitis B virus core antibody (HBcAb) or hepatitis B virus surface antibody (HBcAb), Hepatitis B virus surface Antibody (HBsAb), Hepatitis C virus (HCV) antibody, Human Immunodeficiency Virus (HIV) antibody/antigen, or syphilis serological response positive. (except that HBsAb positive with a vaccination history, HBsAb positive but negative for HBV-DNA* and HCV antibody positive but negative for HCV-RNA*.)
5) Pregnant and lactating female patients
6) Other patients deemed inappropriate by the investigator or others to participate in this study.
*Hepatitis virus testing should be performed once a month during the period of administration to ensure that there are no safety issues.

Related Information

• Primary Sponsor: ando kiyoshi