About this trial
This is a multicenter, Phase I/II study in patients with non-resectable hepatocellular carcinoma following TACE treatment.
Phase I (Open-label dose escalation)
This study will be an open-label study with an Accelerated Phase and a Standard Phase. For the Accelerated Phase of the study, one patient per dose level (1 mg/kg, and 2 mg/kg) is planned. For the dose levels in the standard phase (4 mg/kg, 8 mg/kg and 16 mg/kg), it will follow the Fibonacci's rule of 3 + 3 design. All eligible patients who have received TACE treatment and recovered well, will be administrated PTX-9908 Injection intravenously one dose per day for 5 days on Week 1 (excludes weekends and public holidays), and one dose per week (on Day 8, Day 15, and Day 22) for 3 consecutive weeks. The 4-week treatment period, will be followed by a 2-week follow-up period.
Phase II (Randomized placebo controlled dose expansion)
The objective of phase II is to further evaluate the safety, tolerability and antitumor activity of PTX-9908 Injection for patients with non-resectable hepatocellular carcinoma following TACE treatment. Approximately 24 eligible patients who have received TACE treatment and recovered, will be randomized to PTX-9908 Injection using the predetermined dose in phase I or the vehicle placebo in a 2:1 ratio. PTX-9908 Injection or placebo will be administered intravenously one dose per day for 5 days in Week 1 (excludes weekends and public holidays), and one dose per week till Week 12 (Day 78). The 12-week treatment period, will be followed by a 2-week follow-up period.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Unresectable hepatocellular carcinoma and at intermediate-stage HCC (BCLC stage B or Child-Pugh class A/B with large or multifocal HCC, no vascular invasion, or extrahepatic spread) with completed TACE procedure in 4 weeks before day 1 of study intervention infusion.
Recovered from TACE treatment and procedure related toxicities including ALT/AST and bilirubin within normal limit or reference numeric value (reference value is defined as the test value before TACE procedure).
ECOG (Eastern Cooperative Oncology Group) performance status < 2.
Absolute neutrophil count > 1,200/µL
Disqualifiers
1. Patient with Child-Pugh B8-9.
Patient who has had anti-cancer therapy including surgery, radiotherapy, immunotherapy, or chemotherapy (except in TACE regimen) within 4 weeks prior to the screening visit.
Patient who has received any other investigational agents within 4 weeks prior to the screening visit.
Patient who has not recovered from the side effects of the earlier investigational agent or had anti-cancer therapy including surgery, radiotherapy, immunotherapy, or chemotherapy.
Trial design
Parallel
Treatments tested in this trial
PTX-9908 Injection
DrugProposed dose cohorts:1 mg/kg, 2 mg/kg, 4 mg/kg, 8 mg/kg, and 16 mg/kg. Frequency: Phase I: one dose per day for 4 consecutive weeks (20 doses). Phase II (A)Daily Dose Regimen one dose per day for 12 consecutive weeks (60 doses). (B) Daily for first week, followed by weekly treatment Regimen One dose per day for 5 consecutive days in Week 1 (5 doses), and one dose per week till for 11 weeks (11 doses). Duration: 4 weeks (Phase I) and 12 weeks (Phase II).
Placebo
Drugwater for injection Phase II (A)Daily Dose Regimen one dose per day for 12 consecutive weeks (60 doses). (B) Daily for first week, followed by weekly treatment Regimen One dose per day for 5 consecutive days in Week 1 (5 doses), and one dose per week till for 11 weeks (11 doses). Duration: 12 weeks (Phase II)
Treatment groups
Trial outcomes
Primary outcomes
Safety as measured by the number and severity of Adverse Events
Safety as measured by number of participants with a dose limiting toxicity (DLT)
Any of the following, if judged to be associated with PTX-9908 Injection or the combination of TACE with PTX-9908 Injection (i.e., with possible causality) that occur within the DLT evaluation window, will be considered as a DLT. 1. All Grade 4 (i.e., life-threatening) non-hematological and hematological toxicities (except for Grade 4 lymphopenia). 2. Grade 3 anemia with clinically significant bleeding 3. All grades of febrile neutropenia. 4. All Grade 3 non-laboratory/non-hematologic treatment-emergent adverse events, with the exception for nausea, vomiting, or diarrhea that resolves within 3 days. 5. Grade 3 thrombocytopenia with clinically significant bleeding is a DLT. 6. Grade 3 cytokine release syndrome of any duration is a DLT. 7. Grade 3 laboratory adverse events that are judged by the investigator as clinically significant.
Secondary outcomes
Recommended Phase 2 Dose (RP2D)
The RP2D was based on the highest dose in which 0/6 or 1/6 participants experienced DLT with at least 2 out of no more than 6 participants experiencing DLT at the next higher dose level, and the assessment of any relevant chronic toxicity.
Maximum Plasma Concentration (Cmax) of single-dose and repeat-dose of PTX-9908 Injection
Pharmacokinetics (PK) is a branch of pharmacology concerned with the rate at which drugs are absorbed, distributed, metabolized, and eliminated by the body. Cmax=maximum observed plasma concentration of PTX-9908 Injection administered with IV dose derived from plasma concentration versus time data.
Terminal Half-life (T-Half) of single-dose and repeat-dose of PTX-9908 Injection
PK is a branch of pharmacology concerned with the rate at which drugs are absorbed, distributed, metabolized, and eliminated by the body. T-Half=terminal-phase elimination half-life in plasma of PTX-9908 Injection administered with IV.
Overall Tumor Response
Number of participants with a best overall tumor response of complete Response (CR), Partial Response (PR), Stable Disease (SD), and Progressive Disease (PD). Overall tumor response will be assessed using contrast-enhanced CT imaging according to mRECIST criteria following the 1.1 release of RECIST.
Locations
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Sponsors and contacts
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