An observational study in Cancer, sponsored by Oslo University Hospital. Recruiting at 1 site in Norway. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-08-15.
Sponsored by Oslo University Hospital · Observational
Off-label drug use, where a marketed drug is used outside its approved indication, may allow early access to new and promising treatments. However, its use can be a source of controversy, due to limited evidence for clinical benefit and lack of cost/QALY-estimates, leading to challenging prioritization issues. The number of drugs suitable for off-label use is expected to further increase in the coming years, owing to the rapid progress in the field of oncology, in particular with the current era of precision medicine and targeted therapies. This also challenges the traditional method of running clinical trials, with eligible patient populations commonly being small, underpinning the importance of gaining supplementary real-world evidence from well performed observational studies.
This prospective observational study will therefore assess real-world outcomes of patients treated with off-label anti-cancer drugs, including efficacy in terms of response rates, time to progression/relapse measures and survival; patient-reported outcome measures (PROMS) and self-reported side-effects/toxicity; as well as collecting blood samples for a biobank for further translational research. Further, the study will give a descriptive analysis of the current practice of off-label use of anti-cancer drugs in Norway, including prevalence estimation and health care related cost analyses.
9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.
This study's planned enrollment of 200 is close to the median of 204 across 1,683 observational studies indexed under Neoplasms.
Browse Neoplasms studies →Oslo University Hospital is the lead sponsor of 810 studies on the registry; 148 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Any patient accepted by the leadership in the Department of Oncology at OUH to receive anti-cancer treatment off-label from the date of start of the study is eligible for inclusion. Of note, this does not include off-label use of drugs used for supportive care.
Exclusion Criteria:
Progression free survival (PFS).
Time from date of inclusion until the date of first documented progression or date of death from any cause, whichever come first, according to RECIST v1.1
Time frame: Assessed up to 2 years after end of inclusion
Patients questionnaire EORTC QLQ-C30
Assessment of patients reported quality of life, as measured by EORTC QLC30
Time frame: Assessed from inclusion until 2 years after end of treatment
Objective tumor response rate (ORR)
Defined as the proportion of patients with an objective tumor response (either partial response \[PR\] or complete response \[CR\] using RECIST v1.1) response (DR), time to next treatment and overall survival (OS)
Time frame: Assed through study completion, an average of 1 year
Duration of response (DR)
Duration of response among patients with an objective response, according to RECIST v1.1
Time frame: Assed through study completion, an average of 1 year
Time to next treatment (TTNT)
Time from inclusion to institution og next therapy
Time frame: Assed through study completion, an average of 1 year
Overall survival (OS)
Time from date of inclusion until the date of death from any cause
Time frame: Assessed up to 2 years after end of inclusion
Fatigue
Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ)
Time frame: From inclusion until 2 years after end of treatment
Depression
Assessment of patient reported outcomes, as measured by the patient health questionnaire (PHQ-9)
Time frame: From inclusion until 2 years after end of treatment
Pain intensity
Assessment of patient reported outcomes, as measured by an 11 point Numerical Rating Scale (NRS) for pain intensity
Time frame: From inclusion until 2 years after end of treatment
Adverse event
Patients files and self-report. Classified according to CTCAE v 5.0 and MedDRA
Time frame: From inclusion until 2 years after end of treatment
Quality adjusted life years (QALYs)
Patient self reported EQ-5D
Time frame: From inclusion until 2 years after end of treatment
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Oslo University Hospital