A Phase I, Open Label, Multicenter Study to Evaluate the Safety and Efficacy of PUR001 Administered Intravenously in Adult Patients With Advanced Solid Tumors
Overview
- Phase
- Phase 1
- Intervention
- PUR001
- Conditions
- Advanced Solid Tumors
- Sponsor
- Purinomia Biotech, Inc.
- Enrollment
- 23
- Locations
- 1
- Primary Endpoint
- Dose Limiting Toxicity (DLT) and Maximum Tolerated Dose (MTD), if reached
- Status
- Recruiting
- Last Updated
- 3 years ago
Overview
Brief Summary
This is a Phase I, First-In-Human, open label, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-cancer activity of PUR001, an anti-CD39 monoclonal antibody, in adult patients with advanced solid tumors, as monotherapy. A "3+3" design will be used to determine MTD and RP2D. .
Detailed Description
This is a Phase I, First-In-Human, open label, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-cancer activity of PUR001 in adult patients with advanced solid tumors, as monotherapy. The primary objective of this study is to determine the dose-limiting toxicity(ies) (DLT) and/or maximum tolerated dose (MTD) of PUR001 monotherapy in patients with advanced/metastatic solid tumors. Study consists of two parts: Dose Escalation Cohort and Dose Expansion Cohort. Dose limiting toxicity (DLT) will be evaluated and managed per the pre-defined DLT criteria and rules specified in the protocol. MTD and/or RP2D will be confirmed in a dose confirmation cohort.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Able to sign informed consent and comply with the protocol
- •≥ 18 years of age, at the time of signing informed consent
- •Histologically or cytologically documented advanced/metastatic solid tumors who have received at least one line of prior systemic chemotherapy and progressed
- •At least one measurable lesion as defined by RECIST V1.1 criteria for solid tumors
- •ECOG performance status of 0 or 1
- •Adequate organ function confirmed at screening and within 10 days of initiating treatment, as evidenced by:
- •Absolute neutrophil count (ANC) ≥ 1.5 × 10\^9/L
- •Hemoglobin (Hgb) ≥ 8 g/dl
- •Platelets (plt) ≥ 75 × 10\^9/L
- •AST/SGOT and ALT/SGPT ≤ 2.5 × Upper Limit of Normal (ULN) or ≤ 5.0 × ULN if liver metastases are present
Exclusion Criteria
- •Women who are pregnant or lactating
- •Women of child-bearing potential (WOCBP) who do not use adequate birth control.
- •Patients with untreated brain or central nervous system (CNS) metastases or brain/CNS metastases that have progressed (e.g., evidence of new or enlarging brain metastasis or new neurological symptoms attributable to brain/CNS metastases) Note: Patients with treated brain metastases that are off corticosteroids and have been clinically stable for 28 days are eligible for enrollment
- •Patients with a known concurrent malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin, carcinoma in situ of the cervix or other noninvasive or indolent malignancy that has previously undergone potentially curative therapy
- •Impaired cardiac function or significant diseases, including but not limited to any of the following:
- •LVEF \< 45% as determined by MUGA scan or ECHO
- •Congenital long QT syndrome
- •QTcF ≥ 480 msec on screening ECG
- •Unstable angina pectoris ≤ 3 months prior to starting study drug
- •Acute myocardial infarction ≤ 3 months prior to starting study drug
Arms & Interventions
Monotherapy classic "3+3" design dose escalation and expansion
Intervention: PUR001
Outcomes
Primary Outcomes
Dose Limiting Toxicity (DLT) and Maximum Tolerated Dose (MTD), if reached
Time Frame: 1-1.5 years
Safety and tolerability of PUR001 as a single agent
Secondary Outcomes
- Number of participants with treatment-related adverse events (AEs)(1-1.5 years)
- Area under the concentration time curve (AUC 0-last)(1-1.5 years)
- Volume of Distribution (Vd)(1-1.5 years)
- Duration of Response (DOR)(1-1.5 years)
- Progression free survival (PFS)(1-1.5 years)
- Maximum plasma concentration (Cmax)(1-1.5 years)
- Objective Response Rate (ORR)(1-1.5 years)
- Number of participants with positive Anti-therapeutic antibody (ATA)(1-1.5 years)
- Time to Maximum Plasma Concentration (Tmax)(1-1.5 years)
- Mean Residence Time (MRT)(1-1.5 years)
- Disease Control Rate (DCR)(1-1.5 years)
- Half-life (T1/2)(1-1.5 years)