Umbrella Study for Single Patient Treatments in Oncology

Recruiting

• Conditions: Rare Diseases; Advanced Cancer
• Interventions: Other: Patient-specific treatments

• Registration Number: NCT06285500
• Lead Sponsor: University Health Network, Toronto

Brief Summary

The purpose of this study is to collect data on how advanced and rare cancers respond to biomarker-based treatments.

Detailed 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. There is no additional visit or procedure required to participate in this study. 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 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

Study Timeline

• Status Verified: 2024-02
• Study Start: 2024-02-01
• Study First Submitted: 2024-02-07
• Study First Submitted QC: 2024-02-22
• Last Update Submitted: 2024-02-22
• Study First Posted: 2024-02-29
• Last Update Posted: 2024-02-29
• Primary Completion: 2027-02-01
• Study Completion: 2029-02-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 400

Inclusion Criteria

Not provided

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Group 2	Patient-specific treatments	N-of-1 treatment with drugs accessed from SAP.
Group 1	Patient-specific treatments	N-of-1 treatment with marketed drugs used on or off-label as per SOC.
Group 3	Patient-specific treatments	N-of-1 treatment with non-marketed investigational agents.

Primary Outcome Measures

Name	Time	Method
Overall response rate for the overall cohorts and the number of patients accessing precision targeted therapy	3 years

Secondary Outcome Measures

Name	Time	Method
Overall survival rate	5 years
Number of participants with adverse events as assessed by CTCAE v5.0	5 years	For Groups 1 and 2: Only Grade 3 and above AEs/AESIs and SAEs that are related (possibly, probably or definitely) to the study drug.; For Group 3: All SAEs (regardless of causality).
Progression-free survival rate	5 years

Trial Locations

Locations (1)

University Health Network, Princess Margaret Cancer Centre; 🇨🇦 Toronto, Ontario, Canada