Umbrella Study for Single Patient Treatments in Oncology (UNIQUE)

UNIQUE: Umbrella N-of-1 Tumor Trials - Umbrella Protocol for Oncology Single Patient Protocols

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

Study Overview

• Status: Recruiting
• Conditions: Advanced Cancer; Rare Diseases
• Intervention / Treatment: Other: Patient-specific 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 Type: Observational
• Enrollment (Estimated): 400

Contacts and Locations

• Study Contact: Name: Amit Oza, Dr.; Phone Number: 416 946 4450; Email: amit.oza@uhn.ca

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • Recruiting
      • University Health Network, Princess Margaret Cancer Centre
      • Contact: Amit Oza, Dr.; Phone Number: 416 946 4450; Email: amit.oza@uhn.ca

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Patients have advanced and rare cancer with limited treatment options.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Group 1; N-of-1 treatment with marketed drugs used on or off-label as per SOC.	Other: Patient-specific treatments; Treatment will be decided specifically for the patient based on biomarker test results.
Group 2; N-of-1 treatment with drugs accessed from SAP.	Other: Patient-specific treatments; Treatment will be decided specifically for the patient based on biomarker test results.
Group 3; N-of-1 treatment with non-marketed investigational agents.	Other: Patient-specific treatments; Treatment will be decided specifically for the patient based on biomarker test results.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Overall response rate for the overall cohorts and the number of patients accessing precision targeted therapy	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival rate		5 years
Progression-free survival rate		5 years
Number of participants with adverse events as assessed by CTCAE v5.0	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).	5 years

Collaborators and Investigators

• Sponsor: University Health Network, Toronto
• Investigators: Principal Investigator: Amit Oza, Dr., University Health Network, Toronto

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2024
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2029

Study Registration Dates

• First Submitted: February 7, 2024
• First Submitted That Met QC Criteria: February 22, 2024
• First Posted (Actual): February 29, 2024

Study Record Updates

• Last Update Posted (Actual): February 29, 2024
• Last Update Submitted That Met QC Criteria: February 22, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Rare Diseases
• Other Study ID Numbers: UNIQUE; 23-5095 (Other Identifier: University Health Network)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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