Mesoblast

Clinical trials are research studies that test medical therapies for human use. Each study answers a particular medical question to prevent, screen for, diagnose or treat a disease. Before any product can be approved for use in the general public, a number of clinical trials must be conducted to show that the treatment is safe and effective. The information gained from clinical trials helps to improve medical care and contributes to the understanding of diseases and conditions (for example, how a disease progresses or how it affects different systems in the body).

That depends on the trial in question. Every clinical trial is conducted according to an approved protocol, which includes specific eligibility criteria that people must meet in order to be eligible for the trial. Eligibility criteria are also known as inclusion and exclusion criteria. Examples of eligibility criteria include:

• Generally, people must have been diagnosed with a specific medical condition or injury to be eligible for a trial, and there will often be restrictions related to the severity of the condition or injury.
• Normally only people within a specified age group will be eligible. Children and elderly people are often excluded from trials, especially in the early stages of research.
• There are normally restrictions related to other diseases that people may have, and other medications they may be taking.
• Pregnant and breastfeeding women are normally excluded, and women of child bearing potential are generally required to use a reliable method of contraception for a specified period of time.

There is a limit to the number of people who can enroll in any trial. This varies depending on the nature of the trial – for example in the early stages of research, trials may only require 12 to 24 participants, whereas in later stages, trials can involve many hundreds of participants. Once the specified number of participants has been enrolled in a trial, it is normally not possible to enroll anyone else.

Before someone can participate in a clinical trial, they must provide informed consent. They are given information about the trial and an opportunity to discuss it with medical staff, and then they are asked to sign a consent form if they wish to participate.

After giving informed consent, participants must attend one or more screening visits at a clinic or hospital, at which they will undergo a number of tests and answer questions on their condition and medical history. This will help establish if they are eligible for the trial.

If a person is eligible for a trial and agrees to participate, they will receive treatment according to the trial protocol and attend a clinic or hospital for follow-up tests at defined times over the duration of the trial. Each clinical trial can only take place at specifically approved clinics or hospitals, so it may not be possible for people to participate in a trial in their local area.

In many cases, clinical trials are “randomized” and compare at least two different treatment options. Most of Mesoblast’s clinical trials compare adult allogeneic Mesenchymal Precursor Cell (MPC) treatments with a “control” treatment, which is generally the treatment that someone would receive if they did not participate in the trial. This means that people who participate in our clinical trials are not guaranteed to receive treatment with MPCs. This is necessary to ensure that independent medical investigators can evaluate the safety and effectiveness of our treatments in a fair and unbiased way.

Possible benefits:

• You might gain access to new research treatments before they are widely available.
• You will receive extensive medical care associated with the clinical trial.
• You will help others by contributing to medical research.

Possible risks:

• There may be unpleasant, serious or even life-threatening side effects to experimental treatment.
• The experimental treatment may not work.
• You may be placed in the control group and not receive the experimental treatment.
• You may need to visit the hospital or doctor’s room more frequently, stay there longer and undergo more tests.

Only people who meet all of the eligibility criteria for the trial can be enrolled. There is a doctor in charge of each trial, who is known as the Principal Investigator. The Principal Investigators in charge of our trials are not Mesoblast employees; they are independent experts.

The Principal Investigator is responsible for deciding whether people are suitable for a trial, and Mesoblast has no influence over those decisions. Therefore, Mesoblast cannot enroll people directly into our clinical trials.

People who are interested in enrolling in clinical trials should generally discuss it with their doctors, who can then refer them to the Principal Investigator of a trial if appropriate.

Please note that we are unable to respond to enquiries from the public about participation in our clinical trials or treatment with our products.

Enrollment in clinical trials is overseen by the doctors in charge of the clinical trial sites, not Mesoblast. Patients should discuss their medical condition and treatment options with their doctor.

Mesoblast is currently running a number of clinical trials using allogeneic or universal donor Mesenchymal Precursor Cells.
Details of all Mesoblast trials can be found by clicking here.