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To determine the MTD of PT01 based on the toxicity observed during Cycle 1 of the Dose Escalation Phase and to investigate the safety and tolerability of PT01 when administered intravenously(IV) to subjects with advanced malignancies

PT01 (Pegtomarginase) breaks down arginine in the blood stream, reducing the supply of arginine and stopping the cancer cells from growing. Normal cells can continue to make their own arginine inside the cell, so PT01 targets the rapidly growing cancer cells.

PT01 (Pegtomarginase)

Donna Madore

Jane DeVane

Clinical Trials Referral Office

This is a first-in-human (FIH) Phase 1 dose escalation study to evaluate the safety, tolerability, PK, PD, and preliminary activity of PT01 administered IV in subjects with advanced malignancies.'

The study consists of a Dose Escalation Phase and a Dose Expansion Phase, both of which include a 28-day Screening Period, Baseline, a Treatment Period (comprised of 28-day cycles with weekly dosing on Days 1, 8, 15, and 22), and a Follow-up Period. Unique to the Dose Escalation Phase is the inclusion of Cycle 0 during which a single dose of PT01 will be administered before Cycle 1 for detailed exploration of the PK/PD relationship.

All PT01 IV doses will be administered at the clinical site.

A Phase I Open-label Study for Subjects With Advanced Malignancies

A Phase I Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity o f Ascending Doses of PT0I (Pegtomarginase) in Subjects With Advanced Malignancies

Dose-limiting toxicity (DLT) for determination of MTD and/or RP2D. The grading of toxicity is based on the NCI CTCAE v4.03 criteria.

Reduction and duration of arginine for determination of BED

Number of participants with treatment related changes from baseline in vital signs, laboratory parameters and 12-lead ECG findings.

Tumor response according to RECIST v1.1 (best ORR for complete response \[CR\] and partial response \[PR\], stable disease, progressive disease), duration of response, PFS Tumor biomarkers in appropriate tumor types

Spots Global Cancer Trial Database for pt01 (pegtomarginase)