 |
In this Section |
|
|
|
Clinical Trials conducted in the INMR
Your participation in Clinical Trials conducted within the INMR
For more information about the Clinical trials conducted with the INMR please call Kristy Rose on 61 2 9845 1229.
Clinical trials are research studies conducted with people who volunteer to take part. Each study answers specific questions and tries to find better ways to prevent, screen for, diagnose or treat disease. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people and ways to improve health.
There are a many different types of clinical trials. Click here to read about clinical trials conducted within the INMR.
How are participants in clinical trials protected?
Our research is conducted according to strict scientific and ethical principles. Every clinical trial has a protocol (an action plan) which is like a “recipe” for conducting the trial. The protocol describes what will be done in the study, how it will be conducted, and why each part of the study is necessary. The same protocol is used by every doctor or research centre taking part in the trial. All patients entering into clinical trials (and their parents, where relevant) are given information which describes in detail the trial protocol. All research undertaken at the INMR has Ethics Committee approval from The Children’s Hospital at Westmead.
The Ethics Committee, which includes doctors, researchers, community leaders, and other members of the community, reviews all protocols to make sure the study is well-designed and that participants are not likely to be harmed. The Ethics Committee will stop a clinical trial if the trial appears to be causing unexpected harm to the participants, or if there is clear evidence that the new intervention is effective, in order to make it more widely available.
In some cases trials of new medications (or of new applications for recognised medications) are also conducted with a Data and Safety Monitoring Board (DSMB). The DSMB is an independent committee made up of statisticians, physicians, and other expert scientists. The DSMB ensures that the risks of participation are as small as possible, makes sure the data are complete, and stops a trial if safety concerns arise or when the trial’s objectives have been met. If trial participants experience severe or unexpected side effects, or if there is other evidence that the risks outweigh the benefits, the Ethics Committee and DSMB will recommend that the trial be stopped early.
What are eligibility criteria for trials, and why are they important?
For each study, the protocol will have guidelines. Each study’s protocol has guidelines for who can or cannot participate in the study. These guidelines, called eligibility criteria, describe characteristics that must be shared by all participants. The criteria differ from study to study. They may include age, gender, medical history, and current health status. Eligibility criteria for treatment studies often require that patients have a particular disorder, and possibility that they have certain complications or clinical features of that disorder.
Enrolling participants with similar characteristics in trials ensures that the results will be due to what is under study and not other factors. In this way, eligibility criteria help researchers achieve accurate and meaningful results.
What is informed consent?
Informed consent is a process by which people learn the important facts about a clinical trial to help them decide whether to participate. This information includes details about what is involved, such as the purpose of the study, the tests and other procedures used in the study, and the possible risks and benefits. In addition to talking with the researchers, parents and children participating in trials will receive a written information sheet and consent form explaining the study. People who agree to take part in the study are asked to sign the informed consent form. However, signing the form does not mean people must stay in the study. People can leave the study at any time—either before the study starts, or at any time during the study or the follow-up period.
If new benefits, risks, or side effects are discovered during the study, the researchers must inform all participants immediately.
Where do clinical trials take place?
Clinical trials take place in doctors’ offices, research units, and other departments within the hospital. Patients enrolled in the INMR’s clinical trials will generally be followed up in the Neurogenetics clinic or at specially scheduled clinic appointments. Depending on exactly who is conducting each trial, at these review appointments enrolled patients may be seen by a research nurse, physiotherapist or occupational therapist, or one of the doctors associated with the Clinical Trials program. Strength testing assessments will be carried out by an accredited physiotherapist using the INMR’s equipment in the Physiotherapy department.
Some of the trials conducted by the INMR will include patients from The Children’s Hospital at Westmead only. Others will be conducted in association with our collaborators in other research units in Australia and overseas, in which case patients may be enrolled in the studies from a number of different countries around the world. In such cases there are always very strict guidelines and agreements on data sharing and confidentiality.
How are clinical trials conducted?
Clinical trials are usually conducted in a series of steps, called phases. Screening, prevention, diagnostic, and supportive care studies do not always have a phase.
Phase I trials are the first step in testing a new approach in humans. In these studies, researchers evaluate what dose of a drug is safe, how it should be given (by mouth, injected into a vein, or injected into muscle), and how often. Researchers watch closely for any harmful side effects. Phase I trials usually enrol a small number of patients and take place at only a few locations. The patients are divided into smaller groups, called cohorts. Each cohort is treated with an increased dose of the new drug or technique. The highest dose with an acceptable level of side effects is determined to be appropriate for further testing.
Phase II trials study the safety and effectiveness of an agent or intervention, and evaluate how it affects the human body. Phase II studies usually focus on a particular type of nerve or muscle disorder, and include fewer than 100 patients.
Phase III trials compare a new agent or intervention (or new use of a standard one) with the best current (or standard) therapy. Participants are randomly assigned (‘randomised’) to the standard treatment group or the new treatment group. This method, called randomisation, helps to avoid bias in the study. In most cases, studies move into phase III testing only after they have shown promise in phases I and II. Phase III trials may include hundreds of people across the country.
Phase IV trials are conducted to further evaluate the long-term safety and effectiveness of a treatment. They usually take place after the treatment has been approved for standard use. Several hundred to several thousand people may take part in a phase IV study. These studies are less common than phase I, II, or III trials.
Who undertakes clinical trials?
People who participate in a clinical trial work with a research team. Team members may include doctors, nurses, physiotherapists and occupational therapists, psychologists, social workers, dieticians, and other health professionals. The health care team provides care, monitors participants’ health, and offers specific instructions about the study. So that the trial results are as reliable as possible, it is important for participants to follow the research team’s instructions. The instructions may include keeping logs or answering questionnaires.
What happens when a clinical trial is over?
After a clinical trial is completed, the researchers look carefully at the data collected during the trial before making decisions about the meaning of the findings and further testing. After a phase I or II trial, the researchers decide whether to move on to the next phase, or stop testing the agent or intervention because it was not safe or effective. When a phase III trial is completed, the researchers look at the data and decide whether the results have medical importance.
The results of clinical trials are usually published in peer-reviewed, scientific journals. Peer review is a process by which experts review the report before it is published to make sure the analysis and conclusions are sound. If the results are particularly important, they may be featured by the media and discussed at scientific meetings and by patient advocacy groups before they are published. Once a new approach has been proven safe and effective in a clinical trial, it may become standard practice. (Standard practice is a currently accepted and widely used approach.)
The published results of previous studies can be accessed by searching the (US) National Library of Medicine’s PubMed® database. PubMed is an easy-to-use search tool for finding journal articles in the health and medical sciences. PubMed is available at http://www.ncbi.nlm.nih.gov/PubMed on the Internet. .
Patients involved in studies conducted by the INMR will be given copies of publications arising from the clinical trials in which they are involved.
Why would I want to enrol my child in a clinical trial?
The benefits of participating in a clinical trial include the following:
- Participants have access to promising new treatments which are often not available outside the clinical trial setting.
- The approach being studied may be more effective than the standard approach.
- Participants receive regular and careful medical attention from a research team that includes doctors and other health professionals.
- Participants may be the first to benefit from the new method under study.
- Results from the study may help others in the future.
What are some of the possible risks of my child taking part in a clinical trial?
The possible risks of participating in a clinical trial include the following:
- New drugs or procedures under study are not always better than the standard care to which they are being compared.
- New treatments may have side effects or risks that doctors do not expect or that are worse than standard care.
- Participants in randomised trials will not be able to choose the approach they receive, and in ‘blinded’ trials, neither you nor your doctor will know which treatment your child has received until the end of the study.
- Participants may be required to make more visits to the doctor than they would if they were not in the clinical trial.
Who pays for the patient care costs associated with a clinical trial?
The patient care costs associated with clinical trials are not covered by private health insurance. Where the studies involve investigations or tests that would usually constitute part of routine clinical care, the cost of these tests or assessments will be covered by the hospital through Medicare funding. Where additional tests are required, or trials of new medications, the additional costs are covered by the INMR. We cover these costs by external funding from donors and sponsors, and from governmental and non-governmental grant and other funding support. In some instances we may receive financial support from drug companies. In such cases we, and the Ethics Committee and DSMB of the Children’s Hospital, are very careful to ensure that our research findings will not be influenced by that financial support and that we retain control of our research methods and data.
Involvement in a study sometimes involves travel and accommodation costs. We will attempt to cover these costs as much as is possible for families participating in clinical trials conducted by the INMR.
What are some questions you might like to ask us before enrolling in a clinical trial?
It is important for children and their parents to fully understand the nature of their commitment when they enrol in a clinical trial. Some questions you might like to ask us are listed below.
The Study
- What is the purpose of the study?
- Why do the researchers think the approach being tested may be effective? Has it been tested before?
- Who is sponsoring the study?
- Who has reviewed and approved the study?
- What are the medical credentials and experience of the researchers and other study personnel?
- How are the study results and safety of participants being monitored?
- How long will the study last?
- How will the results be shared?
- Possible Risks and Benefits
- What are the possible short-term benefits?
- What are the possible long-term benefits?
- What are the short-term risks, such as side effects?
- What are the possible long-term risks?
- What other treatment options are available?
- How do the possible risks and benefits of the trial compare with those of other options?
Participation and Care
- What kinds of treatment, medical tests, or procedures will children undergo during the study?
- How often will they receive the treatments, tests, or procedures?
- Will treatments, tests, or procedures be painful? If so, how can the pain be controlled?
- How do the tests in the study compare with what people might receive outside the study?
- Will participants be able to take their regular medications while in the clinical trial?
- Where will the participants receive their medical care? Will they be in hospital? If so, for how long?
- Who will be in charge of the participants’ care? Will they be able to see their own doctors?
- How long will participants need to stay in the study? Will there be follow up visits after the study?
Personal Issues
- How could being in the study affect the child’s daily life?
- What support is available for participants and their families?
- Can potential participants talk with people already enrolled in the study?
Where can people find more information about clinical trials?
More information regarding neuromuscular disorders is available at:
http://www.mdausa.org
http://www.mda.org.au/
http://www.mdansw.org/
http://www.parentprojectmd.org
More information regarding clinical trials is available at :
www.clinicaltrials.gov
http://www.ctc.usyd.edu.au/ |
| |
|
>> Click here to return to Research Activities |
|
|
|
Read about how our medical research helps real people lead better lives. Find more information about our current research and download papers to help you learn about our work.
To read information about the conduct of clinical trials in the INMR and around the world click here. Please take a moment to get to know our highly skilled research team. |
