Clinical trials play a major role in the advancement of cancer treatment and care. New approaches may include surgery, chemotherapy, radiotherapy and nanotechnology (Debela et al., 2021). Clinical trials range from phase I to phase IV.
Phase 1 trials test the introduction of drugs for human use, and examine the tolerated dose, adverse effects and the way the body metabolises the drug. This does not necessarily have to test the drug on a specific disease. These studies usually have 6 experimental test subjects and two placebos (duds), where the drug is randomized digitally. Furthermore, they are double-blinded, which means that no one knows which drug any one particular participant is using unless the trial is unblinded due to safety issues. The trial usually tests a small number of people, and monitor the pre-dose and post-dose vital signs, pre and post dose pharmacokinetic (how the body metabolises the drug) and pharmacodynamnic (how the drug affects the body) markers and other safety blood parameters to assess drug viability and ensure participant safety (Memorial Sloan Kettering Cancer Centre [MSKCC]; Ursino et al., 2017). To keep the study safe and valid, there are stringent procedures around drug delivery, storage, administration and disposal. This phase is imperative to ensure participant safety.
Phase 2 trials moves the trial to a larger group of specific people. So, for those with cancer, the drug is testing the specific cancer, whether that is a solid tumour or haematologic cancers. Like phase I studies, these studies are randomized and also depend heavily on blood parameters and biomarkers. This phase tests how well a particular drug works, where one group of people receive the experimental drug and the other standard treatment (MSKCC, 2024). Phase 2 studies are critical in cost determination and drug development progression (van Norman, 2019).
Phase 3 and 4 trials move to even larger groups where the trial may be both national or international, involve multiple study centres and thousands of people (MSKCC, 2024). Furthermore, these trials usually test for long-term drug effects and effectiveness. Furthermore, these trials compare experimental drugs with the standard treatments, and may examine efficacy and/or cost-effectiveness of a particular drug or combination of treatments compared to the standard treatment. These studies may be randomized but do not use placebos, as the novel and standard treatments are compared. Tissue samples may be taken from these studies and biomarkers developed to diagnose or prognose disease form personalised medicine (Hu & Dignam, 2019; Zhou et al., 2024).
Ongoing ethical approval and informed consent play a major role in clinical trials. An outcome of the Nuremburg trials in Germany produced the Nuremburg Code, which basically states that all subjects participating in a clinical trial must consent voluntarily. This is because during the Second World War many individuals were experimented on against their will to the determent of their wellbeing. Clinical trials must not harm and should always keep the wellbeing of the individual in mind. For this reason, clinical trials are closely regulated and monitored where accountability is determined through rigorous documentation (NHMRC, 2023). Furthermore, clinical trials must be conducted with qualified personal in facilitates that are suitable for optimum care (Stewart, 2024). For this reason, clinical research has a comprehensive set of guidelines for practice, the ICH GCP guidelines for good clinical practice (ICH, 2023). Finally, ethically approved research also ensures that all have the right and ability to participate in safe research (Smith et al., 2021). If you are interested in participating in a clinical trial, you can look up the Australian Clinical trial Registry (Australian Clinical Trials, 2024).
References
Australian Clinical Trials. (2024). Australian Clinical Trials. Australian Government. Retrieved September 20, 2024, from https://www.australianclinicaltrials.gov.au/
Debela, D. T., Muzazu, S. G., Heraro, K. D., Ndalama, M. T., Mesele, B. W., Haile, D. C., Kitui, S. K., & Manyazewal, T. (2021). New approaches and procedures for cancer treatment: Current perspectives. SAGE open medicine, 9, 20503121211034366. https://doi.org/10.1177/20503121211034366
Hu, C., & Dignam, J. J. (2019). Biomarker-Driven Oncology Clinical Trials: Key Design Elements, Types, Features, and Practical Considerations. JCO precision oncology, 3, PO.19.00086. https://doi.org/10.1200/PO.19.00086
Memorial Sloan Kettering Cancer Centre [MSKCC] (2024). What does Phase 1, 2, and 3 of a clinical trial mean? MSKCC, Retrieved September 17, 2024, from https://www.mskcc.org/cancer-care/clinical-trials/what-does-phase-clinical-trial-mean
National Health and Medical Research Council [NHMRC] (2023). National Statement on Ethical Conduct in Human Research 2023. Australian Government. https://www.nhmrc.gov.au/about-us/publications/national-statement-ethical-conduct-human-research-2023#block-views-block-file-attachments-content-block-1
Smith, S. M., Wachter, K., Burris, H. A., Schilsky, R. L., George, D. J., Peterson, D. E., Johnson, M. L., Markham, M. J., Mileham, K. F., Beg, M. S., Bendell, J. C., Drelcer, R., Keedy, V. L., Kimple, R. J., Knoll, M. A., LoConte, N., MacKay, H., Meisel, J. L., Moynlhan, Mulvey, T. M., Odeilke, O., Pennell, N. A., Redder-Hayes, K., Smith, C., Sullivan, R. J., & Uzzo, R. (2021). Clinical Cancer Advances 2021: ASCO’s Report on Progress Against Cancer. Journal of Clinical Oncology, 39(10), 1165-1184. https://ascopubs.org/doi/pdfdirect/10.1200/JCO.20.03420
Stewart, K. (2024). Nuremberg Code. Britannica. Retrieved September 19, 2024, from https://www.britannica.com/topic/Nuremberg-Code
Ursino, M., Zohar, S., Lentz, F., Alberti, C., Friede, T., Stallard, N., & Comets, E. (2017). Dose-finding methods for Phase I clinical trials using pharmacokinetics in small populations. Biometrical journal. Biometrische Zeitschrift, 59(4), 804–825. https://doi.org/10.1002/bimj.201600084
van Norman, G. A. (2019). Phase II trials in drug development and adaptive trial design. JACC Basic Transl Sci, 4(3), 428—437. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6609997/
Zhou, Y., Tao, L., Qiu, J., Xu. J., Yang, X., Zhang, Y., Tian, X., Guan, X., Cen, X., & Zhao, Y. (2024). Tumour biomarkers for diagnosis, prognosis and targeted therapy. Signal transduction and targeted therapy, 9(123), 1—86. https://www.nature.com/articles/s41392-024-01823-2
Please contact our Nurses on 07 5445 5794 or [email protected] if you have any questions.
All information provided by Bloomhill is mainly based on research from the Qld Cancer Council and best practice guidelines. Our model of care utilizes the Clinical Oncology Society of Australia (COSA) domains of wellness along with available clinical evidence. Always consult your care team regarding matters that affect your health. This is a guide intended for information only.
