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Understanding Clinical Trials

Without the participation of people like you, new treatments and cures will never happen.

What is a Clinical Trial?

Medical research studies involving people are called clinical trials, or sometimes clinical studies. A clinical trial explores whether a medical strategy, drug, treatment, or device is safe and effective for patients. Studies can also look at other aspects of care, such as improving quality of life for patients.

Two Types of Clinical Trials


Studies in which a treatment or other intervention is being given, and their outcomes are measured by the investigators.


Studies in which patients are observed and their outcomes are measured by the investigators.

Trial Phases

Trials are divided into different stages, called phases. Each trial phase has a specific purpose and is designed to answer certain questions.


Determines a safe dose of the treatment under study (study drug), and monitors how the new treatment affects the human body.


Determines if the new treatment is seemingly effective, and monitors any side effects.


Confirms the treatment’s effectiveness, monitors side effects, and compares the new treatment with the current standard treatment or a control arm in a randomized controlled study.


Gathers more information on a drug or treatment after it has been marketed to see its effect in various populations and any side effects associated with long-term use.

What's Involved?

Most studies are interventional studies. These will require administration of the study drug or treatment and a sequence of monitoring visits. Monitoring usually takes place under clinical conditions (in a clinic), and on rare occasions may require overnight stays.

Blood Tests

Urine Tests


Other Tests

Every Clinical Trial Featured

Outlines what’s involved for the specific trial, however we encourage you to contact the trial coordinator, or the MAP nurse, who will be happy to talk through all the details, and answer any further questions you may have

Why Participate?

Participating in a clinical trial is an invaluable way to get excellent medical care and play a part in advancing research for better treatments and a cure for amyloidosis.

Amyloidosis is a rare disease, and trials can take years to accrue which can dramatically slow down the process of evaluating a drug’s effect. The good news is more and more patients like you are becoming empowered to take part.

So MAP is designed to make it easy for you to find available trials, and – where trials do not yet exist – inspire researchers to focus on new areas of study.

Myth vs Facts

My disease is too advanced to participate in a clinical trial

Clinical trials are designed for individuals with many stages of amyloidosis. Depending on the trial, having advanced disease may not automatically exclude you from participating

Clinical trials are dangerous, and patients are treated like guinea pigs

Clinical trials exist to improve patient care. Protecting patient safety is a top priority on any trial and every trial is carefully regulated. However, clinical trials are experiments so there is always some level of risk. As with any medical treatment, there is still a chance of potentially serious side effects so it is important that you discuss a clinical trial with your doctor thoroughly before deciding to enroll

If there were a clinical trial that could help me, my doctor would have told me about it

New clinical trials are always in development, and your doctor may be unaware of all the clinical trials that are currently available in your area. By creating a profile in MAP, you will be matched to current clinical trials you may qualify for and you will also be notified as new trials come available

I can’t afford to go on a clinical trial

While each clinical trial is set-up differently, in many cases the costs associated with participating in a clinical trial or study will be covered by either your health insurance or the trial sponsor. Participants will typically not be charged for:

  • Study drug
  • Monitoring techniques (e.g. blood tests, which are carried out exclusively for research)
  • Standard of care tests
  • Treatment you receive as part of the clinical trial

In some cases, travel costs to and from the clinical trial center may also be compensated. Contact a trial center to find out more about what may or may not be covered for a specific trial.


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