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The Retinoblastoma Clinical Trial
Much research is carried out to identify new and better treatments for cancer. When research involves patients, it is called a Cancer Clinical Trial.
Clinical trials may be funded by the country’s government or National Cancer Institute, by pharmaceutical companies or by charities such as Daisy’s Eye Cancer Fund.
Cancer clinical trials are used to help improve understanding of available treatments (their effectiveness and side effects). This is usually done by comparing a standard treatment to a new therapy or a modified version of the standard treatment.
Clinical trials are designed by experts to minimise risks and maximise benefits to all participants. Participants are monitored carefully during and after the study, so that any side effects can be picked up and responded to quickly.
The researchers should clearly identify the information they need to collect to assess the effectiveness or safety of the drug or treatment approach which is being trialled.
A reliable clinical trial which is testing a new treatment may require hundreds or thousands of participants. This is because the researchers must find small, but important, differences between the new and standard treatment being compared in the trial.
For example, to show a 10% (1:10) improvement in effectiveness of a therapy, the trial must contain 1,000 patients. To show a 5% (1:20) improvement, 4,000 patients would be needed.
Clinical trials are conducted in phases. Each phase has specific aims and objectives which help to answer different questions or hypotheses posed by the researchers.
Phase I: preliminary testing of an experimental drug or treatment approach to evaluate its safety, establish a safe dose range, and identify side effects. The number of patients in a phase I study is usually limited to under 100.
Phase II: the number of participants is increased to between 100-300 to evaluate its effectiveness and to further assess safety and side effects.
Phase III: With between 1,000-3,000 participants, further evaluation of the protocol is carried out to validate its effectiveness, track side effects, assess safety and evaluate it against other commonly used therapies.
Phase IV: continuing observation and assessment provides further information on risks, benefits, and optimal use of the therapy.
Conducting a clinical trial is the only certain way of measuring the success of a certain cancer treatment. When a clinical trial has shown that a particular treatment is effective, further research trials are usually carried out to find better ways of using it, to reduce side effects or to establish different uses.
Clinical trial protocols must include guidelines setting out who can participate in the research. Establishing and observing eligibility criteria helps to produce reliable results by ensuring the researchers will be able to answer their questions. and this is therefore a vital element of any medical research study.
Eligibility criteria include factors such as age, type, location and stage of disease, previous treatment and other medical conditions. Participants must meet these criteria before joining the study.
All types of clinical research are carried out by a research team, including doctors and specialist research nurses. Clinical trials often involve more tests and clinic visits than might be expected with non-research therapies. However, this type of research is most effective when the protocol is carried out precisely and the participant or their parents have regular, frequent contact with the research team.
Before enrolling in a clinical trial, participants or their patents must be given enough information to enable informed decision about participation. The research team must provide clear details about the research, including its purpose, duration, required procedures, potential side effects, risks and benefits, key contacts and alternative treatment options. Translation of the information must be offered for speakers of a language other than that in which the research is being carried out.
An Informed Consent Document, laying out details about the study, must be signed by the participant or their parent, and a copy must be provided to them. This document is not a legal contract, and the participant or their parent may opt to withdraw from the trial at any time during the trial.
Retinoblastoma is a relatively rare cancer. Due to its rarity, not enough children are diagnosed with the same level of disease in well resourced countries to enable reliable clinical trial research.
Many valuable centre-based research studies are being carried out around the world, but these involve very small groups of children, and these studies are therefore limited in their ability to effictively evaluate therapies.
For this reason, multi-centre clinical trial research is the only effective way of testing existing and new retinoblastoma treatments. Currently, only two multi-centre trials exist to investigate treatment for intraocular retinoblastoma: one in the USA (coordinated by the Childern's Oncology Group - COG), and one international based trial (coordinated by Toronto's Hospital for Sick Children and DECF).
Participation in a clinical trial or centre-based research is completely voluntary. Parents should be given plenty of information to help them decide if the trial is right for their child.
Doctors should clearly explain to parents the nature of the research including official clinical trial status, purpose of the research, eligibility criteria, treatment protocol, potential side effects and monitoring procedures. A full copy of the research protocol can be obtained on request from the treating physician.
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