Phase I Prognostic Online (PIPO) Tool (PIPO)

Phase I Prognostic Online (PIPO) Tool: a New Aid to Improve Patient Selection for Early Phase Clinical Trials

Clinical trials are studies that test new treatments to see if they work and are safe for people with certain illnesses. Phase I clinical trials are the earliest stage of testing, where researchers give a new treatment to a small group of people to see if it's safe and how much of it can be given before causing harmful side effects.

Selecting patients for these trials is challenging, especially since most of them have already tried other treatments that didn't work. To make sure patients are not harmed during the trial, researchers need to estimate how long they might live, so they don't give the new treatment to someone who might not live long enough to see if it works.

Doctors use a variety of tools to estimate how long a patient might live, but it's not always easy to do. To help with this, researchers have developed a new tool called the Phase I prognostic online (PIPO) tool. It helps estimate how long a patient might live by looking at different factors like age, the type of cancer, and other medical conditions. This tool can help doctors decide if a patient should be included in a clinical trial or not.

The researchers plan to test this tool to see how well it works, with the goal of using it in clinical practice to help doctors estimate life expectancy and select patients for clinical trials. This will make it easier for doctors to help their patients get access to new treatments that might help them.

Study Overview

• Status: Completed
• Conditions: Oncology
• Intervention / Treatment: Drug: Immune checkpoint inhibitor, targeted agents

Detailed Description

Patient selection in phase I clinical trials continues to be a challenge even in the new "era" with immune checkpoints inhibitors (ICIs) and combinations with small molecules or targeted agents (TAs). Phase 1 trialists are more frequently dealing with novel compounds and new trial designs with large expansion cohort, looking for preliminary evidence of anti-tumor activity and safety in different tumor types and disease settings. During these last years, certain phase 1 trials have had registrational value transforming the classical drug development path. Despite these trends, patients recruited in phase 1 trials are mostly refractory to standard-of-care therapies, where quality of life and life expectancy remain a cornerstone for trial selection. The need to balance potential risks of toxicity and benefits of investigational drugs in this particularly vulnerable cancer population is critical. Most phase 1 clinical trials use life expectancy of at least 3 months as a specific inclusion criterion to minimize the chances of clinical deterioration during the dose limiting toxicity (DLT) assessment period. Nevertheless, there is no consensus on how to objectively make this estimation in real practice.

Several prognostic scores have been developed for phase 1 trials. Most are based on overall survival (OS) prediction but not in the specific timepoint of survival rate at 3 months (3m). These scores were developed for patients treated with cytotoxic agents or targeted drugs in phase 1 units of the Royal Marsden Hospital (RMH score) and the MD Anderson Cancer Center (MDACC). Additionally, prognostic scores specifically for ICIs have been designed at Gustave Roussy Hospital, Paris, France, (GRIm-Score) or MDACC (MDA-ICI) while the Lung Immune Prognostic Index (LIPI) score has been validated as a prognostic score not only for ICIs, but also chemotherapy and TAs.

Most recently, the investigators proposed the Phase I prognostic online (PIPO) tool that was developed interchangeably for TAs and ICIs. The PIPO tool avoid dichotomization of continuous or ordinary variables and provide estimation of specific survival probabilities for each patient before enrolment in a phase 1 trial. To better validate the tool, the investigators plan a prospective validation to assess its prognostic capacity in order to be used as a decision support system in clinical practice of drug development units for objectively estimating life expectancy criterion in phase 1 trials.

• Study Type: Observational
• Enrollment (Actual): 586

Contacts and Locations

Study Locations

• Spain
  • Barcelona, Spain
    • Vall d'Hebron Institue of Oncology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Phase 1 patients that will receive IT or targeted agents

Description

Inclusion Criteria:

- Participants must have been enrolled in a VHIO phase 1 clinical trial or phase 2 basket trial included in this study.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients candidates for Phase 1 trials	Drug: Immune checkpoint inhibitor, targeted agents; Immune checkpoint inhibitor, targeted agents

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival	overall survival as a continuous variable and 3 months overall survival	1 year

Collaborators and Investigators

• Sponsor: Vall d'Hebron Institute of Oncology

Study record dates

Study Major Dates

• Study Start (Actual): February 2, 2023
• Primary Completion (Actual): April 30, 2026
• Study Completion (Actual): April 30, 2026

Study Registration Dates

• First Submitted: May 11, 2026
• First Submitted That Met QC Criteria: May 11, 2026
• First Posted (Actual): May 18, 2026

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 19, 2026
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Antineoplastic Agents, Immunological; Antineoplastic Agents; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Therapeutic Uses; Immune Checkpoint Inhibitors
• Other Study ID Numbers: PR(AG)675/2020

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No