Safety evaluation study for patients with polycythemia vera

JRCT ID: jRCT2051210083

Registered date:14/09/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Polycythemia Vera
• Date of first enrollment: 15/09/2021
• Target sample size: 6
• Study type: Interventional

Intervention(s)

This drug should be administered within 48 hours after the phlebotomy. In addition, as a dose escalation design, 4 doses of 0.25 mg/kg, 0.4 mg/kg, 0.64 mg/kg, and 1 mg/kg are administered to the same subject.
As an observation and evaluation method, for each dose, in principle, inpatient observation is performed for 1 week from the day before administration, and then safety and pharmacodynamic tests are evaluated by visiting the hospital every 2 weeks. If phlebotomy is required for 4 to 12 weeks after administration, the next dose should be administered after phlebotomy.

Outcome(s)

• Primary Outcome: Number of patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the study (vital signs, Evaluation of laboratory test values, Standard 12-lead ECG, physical findings)

Secondary Outcome

Pharmacokinetics:
Measuring the concentration of PPMX-T003, calculate and assess each PK parameter
Immunogenicity:
Examining the expression rate and antibody titer of anti-drug antibodies (ADA)
Pharmacodynamics:
Assessment of the Red blood cell count, hemoglobin, hematocrit, reticulocytes, serum iron, ferritin, total iron binding capacity, transferrin saturation

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: < 75age old
• Gender: Both

Include criteria

1. Confirmed diagnosis of PV according to either the 2008 or 2016 WHO classification criteria
2. PV patients being only treated with phlebotomy and the interval is 4-9 weeks

• Exclude criteria: Patients administrated drugs for PV treatment such as hydroxyurea or ruxolitinib (aspirin is excluded)

Related Information

• Primary Sponsor: Ito Tomoki