Evidence Development in Cancer Treatment - Real World: PREDiCTrw

Overview

This pilot clinical trial aims to assess the real world quality of life and survival of patients treated with therapy that has preliminary evidence of efficacy but uncertainty of the magnitude of clinical benefit or cost effectiveness in subjects with cancer. The goal of this study is to collect real world evidence with respect to quality of life and outcomes to support decision making.

Detailed description

Clinical practice involves incorporating new data into treatment recommendations including non randomized phase I/II studies. Clinicians' decision-making is swayed by alternative endpoints like response rate (RR), depth of response and progression free survival (PFS), presumed to be surrogates for overall survival (OS). Determination of the added value of these new therapies in terms of outcomes and quality of life (QOL) is challenging in the absence of comparators in trials resulting in increased uncertainty in terms of outcomes, quality of life and cost-effectiveness.

With the possibility of a randomized clinical trial evidence being low in certain populations, the use of real world data (RWD) can provide information regarding therapies with preliminary evidence of efficacy but uncertainty of the magnitude of clinical benefit or cost effectiveness. With RWD, patients may receive access to therapies and participate in the evidence generation package.

This study proposes to use RWD to generate evidence to evaluate therapies with preliminary evidence of efficacy but uncertainty of the magnitude of clinical benefit or cost effectiveness. The key components include regular interval disease assessments (eg radiographic imaging) and collection of patient reported outcomes (PROs) using standardized QOL questionnaires. The aim is to provide high quality real world evidence (RWE) for assessment and economic modelling to reduce uncertainty and facilitate decision-making.

Interventions

• Behavioral QOL - quality of life
  QOL assessments using EQ5D (Euroqol 5 dimension) +/- ESAS (Edmonton Symptom Assessment Scale) +/- CPC (Canadian Problem Checklist) every 4-8 weeks +/- 2 weeks
• Diagnostic test Radiographic or laboratory evaluation
  Radiographic or laboratory evaluation every 12 weeks +/- 2 weeks

Primary outcome measures

• Overall survival [Time frame: From date of initiation until the date of death from any cause, whichever came first, assessed up to 100 months]

Secondary outcome measures (5)

• Response rate [Time frame: From date of initiation until the date of death from any cause, whichever came first, assessed up to 100 months]
• Progression/event free survival [Time frame: From date of initiation until the date of progression or date of death from any cause, whichever came first, assessed up to 100 months]
• Quality of life assessments [Time frame: From date of initiation until the date of death from any cause, whichever came first, assessed up to 100 months]
• Quality adjust survival [Time frame: From date of initiation until the date of death from any cause, whichever came first, assessed up to 100 months]
• Physician assessed response rate [Time frame: From date of initiation until the date of death from any cause, whichever came first, assessed up to 100 months]

Eligibility criteria

Inclusion criteria

• Subjects with cancer for which there remains ongoing questions regarding clinical effectiveness and/or cost effectiveness regarding a therapeutic agent
• Eastern Co-operative Group (ECOG) 0-2
• Life expectancy of at least 12 weeks
• Adequate hematologic and end organ function for drug treatment per the clinician's assessment
• Asymptomatic or treated brain metastases permitted
• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of less than 1% per year during the treatment period and for at least 5 months after the last dose.
• For men: agreement to remain abstinent (refrain from heterosexual intercourse with a female partner of childbearing potential or who is pregnant) or use contraceptive measures, and agreement to refrain from donating sperm, during the treatment period and for at least 5 months after the last dose.
• Ability to give informed consent for the study procedures defined in this protocol.

Exclusion criteria

• Treatment with any approved or investigational agent or participation in another clinical trial with therapeutic intent within 14 days prior to enrollment.
• Inability to complete quality of life questionnaires
• Pregnancy or breastfeeding.
• Any significant cardiovascular disease, comorbidity (i.e. recent major infection, HIV, tuberculosis) or major surgical procedure within 21 days that in the opinion of the investigator renders the proposed treatment unsafe.
• Subjects who are otherwise felt by the treating clinician to be unfit to proceed with this protocol.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Supportive care

Study locations

Canada · 1 center

• BC Cancer — Vancouver

Identifiers

NCT: NCT06242912 · PREDiCTrw

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗