Umbrella Study for Single Patient Treatments in Oncology

NCT06285500 | Recruiting DMC

• 2 other identifiers: UNIQUE23-5095
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Rare Diseases; Advanced Cancer

Keywords

single patient protocols

Outcome Measures

Primary Outcomes (1)

• Overall response rate for the overall cohorts and the number of patients accessing precision targeted therapy

  3 years

Secondary Outcomes (3)

• Overall survival rate

  5 years

• Progression-free survival

  5 years

• Number of participants with adverse events as assessed by CTCAE v5.0

  3 years

Study Arms (3)

Group 1

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

Other: Patient-specific treatments

Group 2

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

Other: Patient-specific treatments

Group 3

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

Other: Patient-specific treatments

Interventions

Patient-specific treatments OTHER

Treatment will be decided specifically for the patient based on biomarker test results.

Group 1; Group 2; Group 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients have advanced and rare cancer with limited treatment options.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University Health Network, Toronto: lead

Study Sites (1)

University Health Network, Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

RECRUITING

MeSH Terms

Conditions

Rare Diseases

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Amit Oza, Dr.

  University Health Network, Toronto

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Amit Oza, Dr.

CONTACT

416 946 4450; amit.oza@uhn.ca

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 7, 2024
• First Posted: February 29, 2024
• Study Start: February 1, 2024
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2029
• Last Updated: December 16, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)