In order to develop effective and safe medical treatment for diseases, there is a great need for research companies to hear from interested trial participants. Contrary to popular belief, clinical trials are conducted under extreme safety standards to ensure that patients enrolled are not harmed in any way. These studies follow strict scientific protocols so that patients are protected and reliable study results can be achieved at every stage of the trial.
Clinical trials often proceed in phases and the number of people recruited are increased according to the positive results in each phase. Example of each phase are,
Phase 1
- Early safety and tolerability
- Determine safe dosage
- Identify side effect
Phase 2
- Effectiveness (exploratory)
- Dose respond
- Further evaluation of safety
Phase 3
- Effectiveness (confirmation)
- Monitor side effects
- Comparison with other common treatment
- Collection of data and information surrounding safety of treatment on patients
Phase 4
- Surveillance on a long-term basis for patients
- Effectiveness in the general population
- Drug use optimisation
As a patient association, we investigate the reliability and trustworthiness of each Pharma research company wanting to start clinical trials for Amyloidosis sufferers. We do this by getting to know the company and understanding their purpose and intention. No doubt that there are involvement by our local medical specialists to ensure protocols are met for any study/ trial, our purpose at NZAPA is to safeguard the needs of patients. We strongly advocate for patients and carer to be involved in the whole study/ trial process so that patients are not deemed as ‘lab rats’. Effective clinical trials must not only be ‘for the patients’ but also ‘with the patients’.
We invite you to speak to us and be able to understand how clinical trials works and allay any fears surrounding it. Participation by patients is very important and unfortunately many promising treatment medication are unable to be fully established because of poor patient recruitment.
“Twenty years from now you will be more disappointed by the things you didn’t do than by the ones you did do. So throw off the bowlines. Sail away from the safe harbor. Catch the trade winds in your sails. Explore. Dream. Discover.” — Mark Twain
New Zealand currently have trials underway. For interest and more information please click the following,
- Intellia Therapeutics Clinical Trial (please email ‘galaxy@clinicalstudies.co.nz‘)
Australia currently have trials underway. For more details and information, please contact the centre and study coordinator directly,
- Princess Alexandra Hospital, Brisbane / Cindy Hall (07 3176 5145)
- Genesis Care, Perth / Dr Peter Purnell (cardiology.research@genesiscare.com) Royal Hobart Hospital / Dr Dave Russell (dave.russell@ths.tas.gov.au)
- The Victorian and Tasmanian Amyloidosis Service, Box Hill hospital Melbourne / Dr Simon Gibbs (simon.gibbs@monash.edu)
- Westmead Hospital, Sydney / Dr Mark Taylor (wslhd-ttrtrials@health.nsw.gov.au)
- Liverpool Hospital, Sydney / Natalia Inness (natalia.inness@health.nsw.gov.au) /Gayathri Kumarasinghe (gayathri.kumarasinghe@health.nsw.gov.au) 02 8738 3078.