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CLINICAL TRIALSUNDERSTANDING

Clinical trials are research studies that help doctors and scientists learn more about a disease and new

ways to improve treatment.

They are essential to studying drugs with the goal of approving only the safest and most effective ones.

This book, Understanding Clinical Trials, explains why clinical trials are needed and what might be involved

if your child or loved one were to join one.

EVER WONDER WHAT A CLINICAL TRIAL IS?

As parents, we want to do what is best for our children. So when our daughter’s doctor reached out to us about a clinical trial that could benefit her, we wanted to learn more.

We had never considered a clinical trial for Sara before, and we had so many questions.

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What exactly is a clinical trial?We had only heard about them.

So we decided to look up the basics first before speaking with Sara’s doctor.

Here are some of the things we learned...

Doctors and researchers are always working hard to discover new ways

to help manage illness.To do this, they follow a number of steps.

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First, researchers come up with an idea for a new or better way to treat a disease.

That discovery could be a new medicine or a new medical device that can be used to

help prevent people from getting sick, or to help those who are sick feel better.

Researchers carry out preclinical experiments, which are done in test tubes and on animals to make sure the new discoveries work and are safe.

The most promising discoveries move on to be

tested in clinical trials.

A clinical trial is a research study that involves people. Clinical trials are also sometimes

called clinical studies. They are an important step for getting medical breakthroughs to

the people who need them.

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Clinical trials help doctors and researchers answer specific questions about a new treatment.

After reading about the basics, we were interested in learning how participatingin the clinical trial could help find better medical treatments.

So we decided to set up an appointment with Dr. Tai to discuss the clinical trial for Sara.

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Here’s what Dr. Tai explained to us about clinical trials...

Researchers and doctors spend countless hours designing a clinical trial before it starts.

During this time, they work out a plan for all the details of the study.

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Every clinical trial is carried out on a group of similar people.

Although many clinical trials are done in adults, some trials are done with children and teens like Sara.

This is because children’s and teens’ bodies are different than adults’ bodies. Doctors need to make sure that new treatments work and are safe for them too.

People all around the world participate in clinical trials to help researchers and doctors discover the

best ways to improve people’s health.

Some trials involve healthy individuals, while others involve people who have the same medical condition.

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A clinical trial may take place at a hospital, clinic, university, or at the doctor’s office.

Phase 1 trials test a new treatment in a small group of people for the first time to see if it’s safe

and to see what it does once inside the body.

Dr. Tai then explained that there are four different clinical trial phases.Each phase helps researchers and doctors learn more about a new treatment.

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Phase 2 trials happen in a larger group of people to see how well the new treatment works and to monitor for side effects.

Phase 3 trials involve even more people to confirm that the new treatment works and is safe.

Finally, Phase 4 trials keep track of how safe and effective a new treatment is after doctors can prescribe it to everyone who needs it.

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A placebo is an inactive substance that contains

no medicine.

We were curious to know how doctors can tell whether the new treatment works.

Dr. Tai told us that some clinical trials compare a new treatment with a control treatment.

The control treatment may be a treatment that doctors already know works and is safe, or it may be a placebo.

Participants are picked at random to receive either the new treatment or the control treatment.

You and the doctors wouldn’t know whether Sara was given

the new treatment or the control. This makes sure that

the trial is completely fair.

In some clinical trials, all participants will receive the new treatment. This

can happen when no other effective treatment is available for people

with serious illnesses.9

Sara was interested in knowing how long trials usually last.

Some trials are short and last only a few months, while others are

longer and last a few years.

Before deciding to join a trial, the doctor will explain all the details to you.

You’ll also learn the purpose of the trial, how long it will last, and the potential benefits and dangers.

Dr. Tai told us that it’s normal to feel hopeful about a new treatment but also concerned for Sara’s safety. That’s why it’s important to ask any questions we may have to understand what the clinical trial would mean for Sara.

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He then told us to take the time we need to decide together as a family whether or not

Sara would like to join the trial.

If Sara agrees to participate, this is called giving assent.

Dr. Tai then explained to us what would happen if we decided to have Sara participate in a clinical trial.

Since Sara is under the age of 18, as parents you will also need to give your permission.

This is called informed consent.

Assent and consent aren’t a contract. They just show that you agree to join a trial.

The thought of having a family meeting excited Sara because they always involve ice cream!

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PHYSICAL EXAM

BREATHINGRATE

BLOOD PRESSURE

HEIGHT &WEIGHT

HEARTRATE

Before starting a trial, Saramay have a physical exam and some

tests, like blood tests and scans.

These tests are needed to see if she meets the eligibility criteria required to participate in the trial.

If Sara decides to participate, she will be part of the research team.

Doctors, nurses, and other health care professionals would keep a close eye on Sara during the trial to see how she’s doing.

It made us feel better knowing that Sarawould be monitored closely during the trial.

Sara may be asked to help out with the research by filling out questionnaires about her health or by

keeping a diary about how she feels.

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GLOSSARY

You will take both eliglustat by mouth and imiglucerase into your vein during the study treatment period. If you feel better after a year (52 weeks), you may just take the eliglustat capsules.

Study MedicineThese include your heart rate, blood pressure, and body temperature.

Vital SignsUsing a blood test, the doctors will check on what might have caused your Gaucher disease. This test can help tell how much medicine you will receive during the study.

Genetic Test

You will be asked to pee into a clean cup. The hospital team can then test your urine (pee) for things like sugar and protein.

Urine TestThe doctor will do a check-up and may listen to your heart and lungs, and check things like your belly, eyes, and joints.

Check-up

You will be asked to answer a few questions to help see how your brain is working. The test looks at your prob-lem-solving skills and how you learn.

Brain Function TestYou and your parent or guardian will fill out a form to answer questions about your health, like how much pain you are in and how tired you are.

Questionnaire

After the doctor explains the study, you and/or your parent or guardian will sign a form to give your permission to join the study.

Permission

You will have an x-ray of your chest to look at your lungs.

Chest X-Ray

Your doctor will ask about your bone pain and any difficulties you might have in moving.

Gaucher Disease Rating

These are scans that look at the inside of your body and your bones.

MRI and DXAThe doctor will test if you can hear sounds of different volume.

Hearing Test

Blood tests are used to measure lots of things, like the number of different blood cells you have. They can also measure how much medicine is in your blood.

Blood Test

An ECG is a painless test that measures your heart’s activity.

Electrocardiogram

You will have x-rays of your left hand, fingers, and wrist.

Hand X-Ray

Nervous System TestThe doctor will check your senses (touch, pain, temperature, vibrations)and movement.

ELIKIDS TRIAL SCHEDULE(children) GROUP 2

EFC13738 ELIKIDS Study Schedule (Children) Cohort 2 V1.0 13/05/2018 (UK)

GLOSSARY

You will take both eliglustat by mouth and imiglucerase into your vein during the study treatment period. If you feel better after a year (52 weeks), you may just take the eliglustat capsules.

Study MedicineThese include your heart rate, blood pressure, and body temperature.

Vital SignsUsing a blood test, the doctors will check on what might have caused your Gaucher disease. This test can help tell how much medicine you will receive during the study.

Genetic Test

You will be asked to pee into a clean cup. The hospital team can then test your urine (pee) for things like sugar and protein.

Urine TestThe doctor will do a check-up and may listen to your heart and lungs, and check things like your belly, eyes, and joints.

Check-up

You will be asked to answer a few questions to help see how your brain is working. The test looks at your prob-lem-solving skills and how you learn.

Brain Function TestYou and your parent or guardian will fill out a form to answer questions about your health, like how much pain you are in and how tired you are.

Questionnaire

After the doctor explains the study, you and/or your parent or guardian will sign a form to give your permission to join the study.

Permission

You will have an x-ray of your chest to look at your lungs.

Chest X-Ray

Your doctor will ask about your bone pain and any difficulties you might have in moving.

Gaucher Disease Rating

These are scans that look at the inside of your body and your bones.

MRI and DXAThe doctor will test if you can hear sounds of different volume.

Hearing Test

Blood tests are used to measure lots of things, like the number of different blood cells you have. They can also measure how much medicine is in your blood.

Blood Test

An ECG is a painless test that measures your heart’s activity.

Electrocardiogram

You will have x-rays of your left hand, fingers, and wrist.

Hand X-Ray

Nervous System TestThe doctor will check your senses (touch, pain, temperature, vibrations)and movement.

ELIKIDS TRIAL SCHEDULE(children) GROUP 2

EFC13738 ELIKIDS Study Schedule (Children) Cohort 2 V1.0 13/05/2018 (UK)

BLOOD TESTX-RAYMRI SCAN

BLOOD PRESSURE

HEART RATE

BODY TEMPERATURE

The most important thing is that you really understand what joining the trial means for Sara and your family.

Sara may have several check-ups during the trial where she may have

more blood tests or scans.

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Something else I want you to keep in mind is that not all trials are successful.

Sometimes a trial may show that there is no benefit from the new treatment, and some trials are canceled if the new treatment has too many side effects.

Now that Dr. Tai had explained clinical trials to us, he definitely gave us a lot to think about.

If you or Sara change your mind about being in the trial for whatever reason,

you can stop any time. We just ask you to tell us the reason why.

Sara may have one last check up to make sure she is OK before leaving the trial.

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We discussed the idea ofSara joining the clinical trial,

and decided to say yes!

If you’re interested in learning more about a clinical trial for your child, talk to your child’s doctor for more information.

Later that night we had a family meeting over big bowls of ice cream!

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CONTROL TREATMENT

In a clinical trial, a new treatment might be compared to a control. A control is either a treatment that the doctors already know works or a placebo.

WHAT ARE CLINICAL TRIALS?

Clinical trials are a type of research that looks at tests and treatments in people. Before new tests and treatments become available to the public, a clinical trial is needed to check how well they work and how safe they are.

PERMISSION

A minor’s (under the age of 18) permission to join a clinical trial is called assent.

Permission to join a clinical trial given by an adult or by the parent or guardian of a child is called informed consent.

Even after giving permission to join, a participant can choose to leave the trial at any time.

GLOSSARY

PLACEBO

A placebo looks exactly like the treatment being tested in the clinical trial, but it doesn’t contain any medicine.

SIDE EFFECTS

These are unwanted and usually unpleasant reactions that may happen during the trial.

RISKS

The risks of a clinical trial include:• Serious side effects caused by a new treatment• The new treatment may not work or may not work any better than

the current treatment• Your child may not receive the new treatment but may be in the

control or placebo group

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PHASES OF A CLINICAL TRIALTesting new treatments in clinical trials follows a series of steps called phases:

PHASE 2The new treatment is then given to a larger number of people to see how well it works and how safe it is.

PHASE 1Phase 1 trials test a new treatment in a small group of people for the first time. These trials aim to find the best dose of the new treatment with the least amount of side effects.

PHASE 3Phase 3 trials compare the effectiveness and safety of a new treatment with the current standard treatment.

PHASE 4Once a new treatment becomes generally available to the public, Phase 4 trials aim to assess how well the treatment works and how safe it is over a long period of time.

GLOSSARY

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EDITOR IN CHIEF Columba Quigley, MD

WRITTEN BY Kimberley Maxwell, PhD

Shawn deLoache

PEER REVIEWERPatrick A. Zweidler-McKay, MD, PhD

ART EDITOR Tom Hunt

ARTWORK BY Dan Peterson

UNDERSTANDING CLINICAL TRIALS ISBN 978-1-64455-592-7

The health information contained herein is provided for educational purposes only and is not intended to replace discussions with your health care provider. All decisions regarding patient care must be made with a health care provider, considering the unique characteristics of the patient.

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