What Are Clinical Trials and Why Are They Important?

As a fundamental component in the advancement of medical knowledge, clinical trials play an important role in the discovery of new treatments, therapies, and procedures for diseases and conditions. They also help to find new ways of reducing, diagnosing, and detecting the chance of developing those diseases or conditions.

These studies are designed to show what works and what doesn’t in humans, key information that can’t be learned in the lab or with animal models. Think about all the current medications, therapies, and devices used to treat diseases and conditions today. You may not realize that they’re only available because they’ve gone through this rigorous process and had human participants involved in their clinical trials.

The ultimate goal in clinical trials is to improve health outcomes. For people living with a rare disease like acromegaly, clinical trials can provide an opportunity to try new therapies that aren’t yet approved (these are called investigational). Participation is also a chance to engage more frequently with physicians, to contribute to research that could ultimately improve their own condition, and to help others in the future.

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The PATHFNDR studies are randomized, placebo-controlled, multi-center studies to evaluate the safety and efficacy of ONCE-DAILY, ORAL paltusotine in subjects with acromegaly. If you’d like to know more about these studies of this investigational drug, click here for more information and eligibility requirements.

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Brain power. It can mean getting good grades or having a knack for figuring things out quickly. But in more literal terms it simply means getting the brain the energy, or in this case sugar (glucose), it needs to function. This sounds simple enough, but there are many things that can go wrong with the steps involved in delivering the right amount of glucose to the brain.

When something goes wrong with those steps, the brain can’t do its job. This may be the case for patients who have congenital hyperinsulinism (HI), a very rare disease involving the levels of glucose and insulin in the body, and how the body does – or doesn’t – regulate these levels. Basically, glucose is a nutrient and insulin, which is made by the pancreas, is what the body needs to use this glucose. Glucose and insulin normally work together to keep the glucose levels “just right.” When glucose goes up (like after a meal), the pancreas secretes insulin to return it to normal levels, and when glucose levels decrease insulin secretion is suppressed. Insulin also tells the rest of the body “I’m fed” so the body stores energy in the muscle, in the liver and in fat, rather than tapping into those reserves to give energy to the brain.

Life with HI is like a roller coaster ride, sometimes you’re high; sometimes you’re low but with research, funding, and new developments you never run out of hope.

— Jilani's parent

About Clinical Trials

There are different types of clinical trials, depending on what researchers are studying. Each is crucial in modern, evidence-based medicine in finding new treatments that could help improve a person’s health.

Preventive Trials

These typically include healthy individuals as well as people who previously had a certain condition and are trying to prevent it from coming back. Testing includes new medications, vitamins, minerals, supplements,  vaccines, and/or lifestyle changes that might lower a person’s risk of certain diseases or conditions.

Screening Trials

Screening trials attempt to find the best ways to detect certain diseases or conditions before patients start to experience symptoms. The goal is often to determine if earlier detection of illnesses decreases a person’s risk of serious harm or death.


These terms describe different ways trials can be designed and conducted.

Subjects Volunteers who take part in the study (also called participants or patients)
Randomized Subjects are assigned to different groups by chance
Open-label Subjects and researchers are all aware of the intervention being given
Single-blind The subjects do not know which group they are in (e.g., whether they are in the treatment group or the placebo group)
Double-blind Neither the researchers nor the subjects know which group the subjects are in until it is revealed at the end of the study
Placebo A substance which does not contain the active ingredients of the experimental drug, but looks the same
Add-on All subjects receive an existing medical therapy, but some then receive the additional experimental drug while others do not or are given a placebo
Single-center The study is carried out at one location
Multi-center The study is carried out in several locations (i.e., different cities or even different countries)


The Principal Investigator

Every clinical trial is overseen by a principal investigator (PI) , who is typically a medical doctor. The PI has expertise in study design, regulatory compliance, data management, project management, and statistical analysis. In a multi-site trial, a PI is typically assigned for each site.

The Sponsor(s)

Sponsors prepare the protocol for the trial and oversee the study. They are often pharmaceutical companies that develop the novel drug to be tested, but may also be physicians, hospitals, government agencies, institutions, advocacy groups, or other organizations.

Every drug development program begins in a lab, where scientists develop and test new ideas. This can include drugs, vaccines, and medical devices. If the results of these tests are promising, the study advances to the next step.


Experiments are conducted with the goal of reaching a “proof-of-concept.” Researchers look at the drug’s nature, its chemistry, its effects (pharmacology), and its potential damage to the body (toxicology). Other measures include:

  • How much of a drug gets absorbed into the bloodstream
  • The potential toxicity of the product and how it’s broken down by the body
  • How quickly the drug and its metabolites (the substances that remain after a drug is broken down by the body) are excreted


There are almost as many reasons for participating in a clinical trial as there are people who do so. It’s a highly personal decision that most participants reach only after discussing it with family, friends, and their doctor(s). A few of the more common reasons people choose to do it include:

Helping to develop better treatment options

Some people are motivated to participate in a clinical trial because currently available treatment options haven’t worked, or certain side effects have a negative impact on their quality of life. Clinical trials provide a way to explore where current therapies fail or to identify areas of needed improvement.

Paying it forward

Giving back by way of contributing to the advancement of medical knowledge is another reason people participate.

The Final Word

The most important aspects in the discovery of new treatments are its safety and effectiveness. Clinical trials pave the way for the development of medical advancements and improved therapies. Patients and families affected by rare diseases like acromegaly know all too well the challenges of managing symptoms and quality of life where treatment options are scarce, inconvenient, and/or painful.

If you’re considering participating in a clinical trial, your doctor may be able to suggest an appropriate study for your condition. We also encourage you to explore the resources on this page to learn more.


The Better Bulletin, our periodic newsletter, helps you stay informed on our work to improve the lives of patients living with rare endocrine diseases.



This database of privately and publicly funded clinical studies conducted around the world is one of the best resources available. You can search by disease, country, and other key filters to find a trial that may be right for you.

The site includes a good list of questions to ask when considering participating in a clinical trial. See them here.

Why participate in clinical research?

This video features researchers and clinical trial participants talking about what it’s like to volunteer for a trial and how it promotes medical advances.

Deciding to participate in clinical trials

Animation and easy-to-understand language make this video from the Office for Human Research Protections (OHRP) a must-see for any potential trial participant.

Another OHRP video addresses randomization. Watch it here.

A participant’s first-hand account

Watch Juliana’s story about her experience participating in a trial for sickle cell anemia.

Informed consent for clinical trials

This page from the FDA website has a lot of good information on the topic.

National Institutes of Health (2015, August 20) Clinical Trials [https://www.nih.gov/research-
training/clinical-trials]. Retrieved May 2020.
U.S. Food and Drug Administration (2018, January 04) Step 3: Clinical Research
Retrieved May 2020.
ClinicalTrials.gov (2019, March) Learn About Clinical Studies
[https://clinicaltrials.gov/ct2/about-studies/learn]. Retrieved May 2020.
National Health Service (2019, May 08) Clinical trials
[https://www.nhs.uk/conditions/clinical-trials/]. Retrieved May 2020.
UptoDate. Patient education: What are clinical trials? (The Basics). Topic 16168 Version 11.0