An observational study in Advanced Cancer and Rare Diseases, sponsored by University Health Network, Toronto. Recruiting at 1 site in Canada. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-12-16.
Sponsored by University Health Network, Toronto · Observational
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.
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):
Under the UNIQUE framework, data from participants will be evaluated. The following data will be collected:
203 studies on the registry are indexed under Rare Diseases; 112 are open to participants now.
This study's planned enrollment of 400 is above the median of 200 across 125 observational studies indexed under Rare Diseases.
Browse Rare Diseases studies →University Health Network, Toronto is the lead sponsor of 1,411 studies on the registry; 292 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 3 (18%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients have advanced and rare cancer with limited treatment options.
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.
N-of-1 treatment with marketed drugs used on or off-label as per SOC.
Other: Patient-specific treatments
N-of-1 treatment with drugs accessed from SAP.
Other: Patient-specific treatments
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.
Overall response rate for the overall cohorts and the number of patients accessing precision targeted therapy
Time frame: 3 years
Overall survival rate
Time frame: 5 years
Progression-free survival
Time frame: 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).
Time frame: 3 years
Plan to share: No
No publications or documents are linked to this record.
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University Health Network, Toronto