Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study

Cancer Clinical Trial

Official title:

Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04457713
• Other study ID #: Off Label Study protocol
• Status: Recruiting
• Start date: June 1, 2020
• Est. completion date: December 31, 2027

Study information

• Verified date: October 2023
• Source: Oslo University Hospital
• Contact: Knut Smeland, PhD/MD
• Phone: +4722934000
• Email: knusme[@]ous-hf.no
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical 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. 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: December 31, 2027
• Est. primary completion date: December 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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.

Exclusion Criteria:

• None

Related Conditions & MeSH terms

• Cancer

Locations

Country	Name	City	State
Norway	Oslo University Hospital	Oslo

Sponsors (1)

Lead Sponsor	Collaborator
Oslo University Hospital

Country where clinical trial is conducted

Norway, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Assessed up to 2 years after end of inclusion
Primary	Patients questionnaire EORTC QLQ-C30	Assessment of patients reported quality of life, as measured by EORTC QLC30	Assessed from inclusion until 2 years after end of treatment
Secondary	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)	Assed through study completion, an average of 1 year
Secondary	Duration of response (DR)	Duration of response among patients with an objective response, according to RECIST v1.1	Assed through study completion, an average of 1 year
Secondary	Time to next treatment (TTNT)	Time from inclusion to institution og next therapy	Assed through study completion, an average of 1 year
Secondary	Overall survival (OS)	Time from date of inclusion until the date of death from any cause	Assessed up to 2 years after end of inclusion
Secondary	Fatigue	Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ)	From inclusion until 2 years after end of treatment
Secondary	Depression	Assessment of patient reported outcomes, as measured by the patient health questionnaire (PHQ-9)	From inclusion until 2 years after end of treatment
Secondary	Pain intensity	Assessment of patient reported outcomes, as measured by an 11 point Numerical Rating Scale (NRS) for pain intensity	From inclusion until 2 years after end of treatment
Secondary	Adverse event	Patients files and self-report. Classified according to CTCAE v 5.0 and MedDRA	From inclusion until 2 years after end of treatment
Secondary	Quality adjusted life years (QALYs)	Patient self reported EQ-5D	From inclusion until 2 years after end of treatment