Clincosm LogoSign in Get a demo

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

Sponsor
Oslo University Hospital (Other)
Overall Status
Recruiting
CT.gov ID
NCT04457713
Collaborator
(none)
200
Enrollment
1
Location
91
Anticipated Duration (Months)
2.2
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational [Patient Registry]
    Anticipated Enrollment :
    200 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
    Actual Study Start Date :
    Jun 1, 2020
    Anticipated Primary Completion Date :
    Dec 31, 2027
    Anticipated Study Completion Date :
    Dec 31, 2027

    Outcome Measures

    Primary Outcome Measures

    1. Progression free survival (PFS). [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

    2. Patients questionnaire EORTC QLQ-C30 [Assessed from inclusion until 2 years after end of treatment]

      Assessment of patients reported quality of life, as measured by EORTC QLC30

    Secondary Outcome Measures

    1. Objective tumor response rate (ORR) [Assed through study completion, an average of 1 year]

      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)

    2. Duration of response (DR) [Assed through study completion, an average of 1 year]

      Duration of response among patients with an objective response, according to RECIST v1.1

    3. Time to next treatment (TTNT) [Assed through study completion, an average of 1 year]

      Time from inclusion to institution og next therapy

    4. Overall survival (OS) [Assessed up to 2 years after end of inclusion]

      Time from date of inclusion until the date of death from any cause

    5. Fatigue [From inclusion until 2 years after end of treatment]

      Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ)

    6. Depression [From inclusion until 2 years after end of treatment]

      Assessment of patient reported outcomes, as measured by the patient health questionnaire (PHQ-9)

    7. Pain intensity [From inclusion until 2 years after end of treatment]

      Assessment of patient reported outcomes, as measured by an 11 point Numerical Rating Scale (NRS) for pain intensity

    8. Adverse event [From inclusion until 2 years after end of treatment]

      Patients files and self-report. Classified according to CTCAE v 5.0 and MedDRA

    9. Quality adjusted life years (QALYs) [From inclusion until 2 years after end of treatment]

      Patient self reported EQ-5D

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Oslo University Hospital Oslo Norway 0379

    Sponsors and Collaborators

    • Oslo University Hospital

    Investigators

    • Principal Investigator: Knut Smeland, PhD/MD, Oslo University Hospital

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Knut Halvor Bjøro Smeland, Chief Attending Physician, Oslo University Hospital
    ClinicalTrials.gov Identifier:
    NCT04457713
    Other Study ID Numbers:
    • Off Label Study protocol
    First Posted:
    Jul 7, 2020
    Last Update Posted:
    Sep 9, 2021
    Last Verified:
    Sep 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Knut Halvor Bjøro Smeland, Chief Attending Physician, Oslo University Hospital
    Off Label Use

    Study Results

    No Results Posted as of Sep 9, 2021