CancerNCT04457713
Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
An observational study of Cancer, run by Oslo University Hospital at 1 site in Norway.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cancer
- How many people
- 200 participants
- Where
- 1 siteNorway
- Study dates
- 1 Jun 2020to 31 Dec 2027
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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.
Who may be able to join
- Verified cancer diagnosis (based on radiological, histological/cytological or operative evidence).
- Treatment with off-label anti-cancer drug.
- Age ≥ 18 years
- Able to provide written informed consent.
Who cannot join
- None
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Norway1 site, 1 recruiting
- Recruiting
Oslo University Hospital
Oslo, 0379
Knut Smeland, PhD/MD, +4722934000, knusme@ous-hf.no
About this study
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.
- Conditions
- Cancer
- Keywords
- Off Label Use
- Medical subject headings
- Neoplasms
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: Assessed from inclusion until 2 years after end of treatment
Secondary outcomes (9)
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)
Measured: 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
Measured: Assed through study completion, an average of 1 year
Time to next treatment (TTNT)
Time from inclusion to institution og next therapy
Measured: 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
Measured: Assessed up to 2 years after end of inclusion
Fatigue
Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ)
Measured: From inclusion until 2 years after end of treatment
Depression
Assessment of patient reported outcomes, as measured by the patient health questionnaire (PHQ-9)
Measured: 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
Measured: 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
Measured: From inclusion until 2 years after end of treatment
Quality adjusted life years (QALYs)
Patient self reported EQ-5D
Measured: From inclusion until 2 years after end of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2020
- Study start1 Jun 2020
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CancerStudies of the same condition, open and under way.
- More trials run by Oslo University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Oslo University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Knut Halvor Bjøro Smeland, Oslo University Hospital
- Investigators
- Knut Smeland, PhD/MD, Principal investigator, Oslo University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04457713
- Sponsor's protocol ID
- Off Label Study protocol
- Status last verified
- Aug 2025
- First submitted
- 25 Jun 2020
- Last updated
- 15 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT04457713
- Acronym / sponsor ID
- Off Label Study protocol
- Conditions
- Cancer
- Keywords
- Off Label Use
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Oslo University HospitalUniversity, hospital or other
- Study start
- 1 Jun 2020 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 7 Jul 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 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.
Key inclusion criteria (4 listed)
- Verified cancer diagnosis (based on radiological, histological/cytological or operative evidence).
- Treatment with off-label anti-cancer drug.
- Age ≥ 18 years
- Able to provide written informed consent.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Progression free survival (PFS).
Time frame: Assessed up to 2 years after end of inclusion
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
Patients questionnaire EORTC QLQ-C30
Time frame: Assessed from inclusion until 2 years after end of treatment
Assessment of patients reported quality of life, as measured by EORTC QLC30
9 secondary outcomes
Objective tumor response rate (ORR)
Time frame: Assed through study completion, an average of 1 year
Duration of response (DR)
Time frame: Assed through study completion, an average of 1 year
Time to next treatment (TTNT)
Time frame: Assed through study completion, an average of 1 year
Overall survival (OS)
Time frame: Assessed up to 2 years after end of inclusion
Fatigue
Time frame: From inclusion until 2 years after end of treatment
Depression
Time frame: From inclusion until 2 years after end of treatment
Pain intensity
Time frame: From inclusion until 2 years after end of treatment
Adverse event
Time frame: From inclusion until 2 years after end of treatment
Quality adjusted life years (QALYs)
Time frame: From inclusion until 2 years after end of treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Knut Smeland, PhD/MD principal investigator
Oslo University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cancer, run by Oslo University Hospital at 1 site in Norway.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Norway. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Knut Smeland, PhD/MD (+4722934000, knusme@ous-hf.no), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression free survival (PFS). (time frame: Assessed up to 2 years after end of inclusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2025.