Until there's a cure ... there's care

A clinical trial is a research study using human volunteers to study the safety and effectiveness of a drug, treatment, or device in changing the course of health outcomes.

Read more about Upcoming Clinical Trials in Australia on the MND Australia website.

Clinical trials follow basic scientific research that has been conducted to better understand disease pathways and pre-clinical studies that test treatment effects in the laboratory. Trials can be conducted to prevent (e.g. immunization trials) or treat disease (e.g. drug and therapeutic device trials).

In MND research, most trials have been drug trials. Drug trials study whether experimental (new) treatments or new ways of using known therapies are safe and effective under controlled conditions outlined in the study protocol.

What is a study protocol?

A protocol is an action plan based on strict scientific and ethical principles that describes how the study will be conducted and how the patients' safety will be protected. The protocol will outline the number of participants needed, eligibility criteria (patient characteristics required for inclusion) such as age, gender, medical history, tests that will be performed, length of the trial, treatment details, and side effects that may occur.

Why are clinical trials important?

Until a cure for a disease is found, it is necessary to continue to search for better, safer, and faster acting treatments to halt or slow down the progression of the disease. Since riluzole, the only currently approved drug for MND, has a very modest effect on extending life, other treatments still need to be developed and tested to more effectively treat MND.

Understanding Clinical Trial Phases

Treatments under study must go through several phases of testing before conclusions about safety and effectiveness can be drawn. It can take up to five years or longer to develop drugs before human testing begins.

Phase I - Researchers test a new drug or treatment in a small group of people to assess its safety, determine a safe dosage range, and identify side effects.
Phase II - The drug or treatment is given to a larger group of people to see if it is effective to further evaluate its safety.
Phase III - The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the treatment to be used safely. Participants are randomized into either the treatment or standard care group for comparison.
Phase IV - After a drug or treatment has already been on the market, researchers gather information on the drug's effect in various populations and further study possible side-effects with long-term use.

Source: http://clinicaltrials.gov/

Who sponsors clinical trials and where do they take place?

Trials are usually sponsored by government agencies, pharmaceutical, biotechnology and medical device companies, private organizations, healthcare institutions, or individual researchers who are searching for ways to improve the health of persons living with a chronic or life-threatening illness. Clinical studies often take place in hospitals, universities, doctors' offices, and community health clinics. Trials may be conducted in one site or at multiple sites at the same time.

What are the risks and benefits?

What are the risks and benefits of participation in a trial?

  • Access to superior healthcare by leading MND physician researchers and care teams
  • Access to new treatments before they become generally available
  • Opportunity to make a valuable contribution to clinical MND research and help others living with the disease now and in the future


  • Unknown side effects
  • Treatment may be found to be of lesser value than standard treatment, or not effective for some individuals
  • Potential time involved with traveling to the study site, receiving treatment, tests, and possible hospital stays
  • Potential costs associated with travel to study site

How safe are clinical trials?

Clinical trials are generally regulated by organisations similar in function to the Food and Drug Administration (FDA) in the United States. All federally regulated trials must be approved and monitored by an independent committee of doctors, scientists, advocates and others to ensure safety. These committees are generally called Institutional Review Boards (IRBs) or Ethics Review Boards (ERBs). The review boards study and approve all study-related documents such as protocols, Informed Consent forms, physician credentials and eligibility, and patient recruitment methods. Data and Safety Monitoring Committees are also common to clinical trials. These independent groups of experts carefully monitor data to detect benefit or harm, and validity of results.

What is Informed Consent?

Being given all the facts about a clinical trial before deciding to take part is known as informed consent. A printed consent form which outlines key details of the study including those related to tests, treatments, potential risks, benefits, or side effects, and participant rights and responsibilities is provided. Before signing, a member of the research team should also discuss the study with you and answer any questions. When you decide to join a trial, you are required to first sign the consent form. You have the right to continue to ask questions throughout the study period and withdraw your consent at any time.

Questions to ask your doctor if you are considering participating in a trial

  • What is the purpose of the study? Does this support progress toward a better treatment and cure for MND and how? Who has reviewed and approved the study?
  • Who are the sponsors? Who will be paying for the treatments and tests? How does the money get spent?
  • What are the risks and benefits and how does this compare with other treatments available?
  • What kinds of tests and procedures will be required? Where will they take place?
  • How long will I receive the treatment? Will it be available after the trial is over? Will there be follow-up after the study is over?
  • Will the study require extra time, effort, or expenses on my part?
  • What does my family need to know?
  • Where can I learn more about the study and the researchers?Are there any previous publications by the researcher related to this study?

To learn more about clinical trials in general, conduct a search on the Internet using the key words: clinical trials.

Information about Australian clinical trials, can be found at http://www.australianclinicaltrials.gov.au/trial-websites

See also The International Alliance of ALS/MND Association's publication about good practice in drug trials: Drug Trials in ALS/MND: A Statement of good Practice,  http://www.alsmndalliance.org/about-us/policies/good-practice-drug-trials/