Umbrella Study for Single Patient Treatments in Oncology (UNIQUE)

University Health Network, Toronto

Status: Enrolling

Conditions: Advanced Cancer; Rare Diseases

Treatments: Other: Patient-specific treatments

Study type: Observational

Funder types: Other

Identifiers

NCT06285500

23-5095 (Other Identifier)

UNIQUE

Details and patient eligibility

About

The purpose of this study is to collect data for assessing the improvement of the overall response rate for the overall cohorts and the proportion of patients accessing precision targeted therapy.

Full description

The UNIQUE umbrella protocol is an overarching framework to study precision medicine in patients with precise molecular data, who have exhausted all established treatment options and who are not eligible for any of the ongoing trials. Participants who have received/who may receive the following treatments may join the study and will be assigned to a study cohort (group):

• Group 1 - Health Canada approved/marketed drug(s) used on or off-label as per Standard of Care (SOC)
• Group 2 - Drugs accessed from Special Access Program (SAP)
• Group 3 - Non-marketed investigational agents

Under the UNIQUE framework, data from participants will be evaluated. The following data will be collected:

• Demographic data (for example: sex, race, month and year of birth)
• Medical history
• Cancer characteristics including biomarkers
• Treatment history
• response to treatment

Enrollment: 400 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria & Exclusion Criteria:

Patient's existing genomic information from tumor molecular profiling will be discussed in the hospital expert molecular tumor board rounds consisting of representatives from specialist genomic profiling and medical oncology departments to decide N of 1 treatment for the patient. The discussion will surround the best next therapeutic option in the patient's cancer subtype with or without clear standard of care guidelines. Therefore, specific eligibility criteria aside from individual patient's medical history is not applicable.

Trial design

400 participants in 3 patient groups

Group 1

Description:

N-of-1 treatment with marketed drugs used on or off-label as per SOC.

Treatment:

Other: Patient-specific treatments

Group 2

Description:

N-of-1 treatment with drugs accessed from SAP.

Treatment:

Other: Patient-specific treatments

Group 3

Description:

N-of-1 treatment with non-marketed investigational agents.

Treatment:

Other: Patient-specific treatments