United Kingdom Children’s Cancer Study Group (UKCCSG)
Reprinted with kind permission from Contact (UKCCSG)
What is a clinical trial?
Traditional medical practice entails a doctor prescribing for a patient that treatment which in his/her judgement, based on past experience of himself and others, offers the best prognosis. Since there are few conditions for which treatment is 100% effective, there is scope for potential improvements in therapy. Such improvements are generally derived by means of clinical trials.
The majority of clinical trials are concerned with evaluation of drug therapies, but they may also involve, for example, surgical procedures.
There are basically three phases of clinical trials in children’s cancer. Most UKCCSG trials are Phase III, involving comparison of a new form of treatment with the standard therapy.
The ‘early drug or new agent trials’, which are often linked to pharmaceutical companies, are aimed at determining toxicities of new drugs, and acceptable drug dosage (Phase I) and evidence of efficacy of treatment (Phase II).
The key elements of clinical trials
The Protocol - contains details of the purpose, design and conduct of the trial.
The Study Coordinator - is the clinician responsible for preparation of the protocol, medical monitoring of the study, and ultimately reporting of results.
The patients - Statistical methods are used to determine the number of patients required to answer the trial’s main objectives, but availability of patients has also to be taken into account. The estimated time period for patient recruitment will depend on the frequency of any given disease.
Randomised trials - involve comparison of two or more treatments and, to avoid the risk of biased results, a patient may be randomly assigned to one treatment arm or another. (Because of small patient numbers in particular tumour types, and the need to balance numbers in both arms, it is not always possible to run randomised trials in paediatric oncology).
Data collection, management and analysis - The need for accurate recording and processing of patient data is essential, and requires close collaboration between the treatment centres and the UKCCSG Data Centre. Clinical trials now operate to strict codes of conduct.
Ethical issues - Every clinical trial requires careful assessment of whether it is ethically acceptable for patient participation. In the UK, protocols now require approval of the new national ethical review process, as well as local approval from each participating centre.
Publication of trial results - The reporting of clinical trial results as a publication in a refereed journal, or in the form of presentations at major scientific meetings, is the most effective way to inform other investigators of the advances in current research. (Because of the need for adequate follow up information on all patients in a trial, final publication may be delayed for some time after recruitment has ended).
Participation in a clinical trial, or specific queries relating to any aspect of the trial should always be discussed with your/your child’s doctor.