Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04457713
Recruitment Status : Recruiting
First Posted : July 7, 2020
Last Update Posted : September 9, 2021
Sponsor:
Information provided by (Responsible Party):
Knut Halvor Bjøro Smeland, Oslo University Hospital

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
Cancer

Layout table for study information
Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 2 Years
Official Title: Off-label Use of Anti-cancer Drugs in Norway -a Prospective Cohort Study
Actual Study Start Date : June 1, 2020
Estimated Primary Completion Date : December 31, 2027
Estimated Study Completion Date : December 31, 2027



Primary Outcome Measures :
  1. 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

  2. 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


Secondary Outcome Measures :
  1. Objective tumor response rate (ORR) [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: Assed through study completion, an average of 1 year ]
    Time from inclusion to institution og next therapy

  4. Overall survival (OS) [ Time Frame: Assessed up to 2 years after end of inclusion ]
    Time from date of inclusion until the date of death from any cause

  5. Fatigue [ Time Frame: From inclusion until 2 years after end of treatment ]
    Assessment of patient reported outcomes, as measured by the Chalder Fatigue Questionnaire (FQ)

  6. Depression [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: From inclusion until 2 years after end of treatment ]
    Patient self reported EQ-5D



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04457713


Contacts
Layout table for location contacts
Contact: Knut Smeland, PhD/MD +4722934000 knusme@ous-hf.no
Contact: Tormod Guren, PhD/MD +4722934000 uxtour@ous-hf.no

Locations
Layout table for location information
Norway
Oslo University Hospital Recruiting
Oslo, Norway, 0379
Contact: Knut Smeland, PhD/MD    +4722934000    knusme@ous-hf.no   
Sponsors and Collaborators
Oslo University Hospital
Investigators
Layout table for investigator information
Principal Investigator: Knut Smeland, PhD/MD Oslo University Hospital
Layout table for additonal information
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: July 7, 2020    Key Record Dates
Last Update Posted: September 9, 2021
Last Verified: September 2021

Layout table for additional information
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, Oslo University Hospital:
Off Label Use