A Guide to Clinical Trials: Understanding How New Treatments Are Developed

Mar 12, 2025

What Is a Clinical Trial?

A clinical trial is a research study that tests a new treatment or drug. The goal is to see if the drug or treatment is safe and effective. Clinical trials are necessary for developing new ways of managing or treating diseases. They help scientists and doctors see if a new drug works. For example, researchers are running clinical trials for a new drug for multiple system atrophy (MSA). It targets specific cells in the body and may help ease some of the symptoms of MSA.

Clinical trials are not only for new medications. They can also test ways to improve quality of life by managing symptoms or diagnosing conditions earlier. For example, a clinical trial for progressive supranuclear palsy (PSP) is testing whether specific sleep medications can improve sleep, symptoms or quality of life.

How Drugs and Treatments Make It to Clinical Trials

Bringing a new treatment to a clinical trial is a long and expensive journey that starts in a lab. Scientists first study the disease, looking for its causes and how it progresses. Then, they spend a long time (years, even) in the lab identifying a potential treatment.

Once they find a promising treatment, they test it in the lab on animals or cells from humans. Researchers may also test the drug using computer programs or devices that mimic human organs.

Then, researchers apply to the government to start clinical trials. In the United States, the Food and Drug Administration (FDA) oversees these trials. These organizations ensure safety and trustworthy results. They also ensure the results are reliable and provide a true representation of the effects of the treatment. It’s important to note that not every drug presented makes it to clinical trials.

How Do Clinical Trials Work?

Clinical trials always follow a strict process. This ensures safety and accurate data collection. It starts with a scientific question: “What is the problem or unmet medical need that we need to address?” Then, further questions are identified, such as:

What is known about the disease or condition we are addressing?
What is the target population for this research?
Is there a scientific rationale for testing this treatment or intervention?

By answering these questions, researchers can make sure the trial has a clear purpose and a meaningful goal.

Then, they can design a clinical trial to answer the following question: “Can this new treatment improve outcomes or meet patient needs?”

They follow a series of steps:

Screening and Enrollment: Researchers find and enroll eligible participants who fit the criteria. Criteria may include diagnosis, symptoms, symptom start date, age, gender, current and past medications or other diagnoses.
Treatment: Researchers give participants a new treatment, an existing one or a placebo. A placebo looks just like real medicine but has no active ingredients. A placebo helps researchers see if the drug works by comparing the drug’s effects to those of a placebo. Participants, and sometimes researchers, are often unaware of who gets a placebo in a trial until the end. This approach is either a single-blind or double-blind study.
Monitoring and Data Collection: Participants are closely monitored for results and side effects. Side effects may include dizziness, a rash or more serious events, such as heart problems. Researchers also monitor cold or flu symptoms to understand correlation and causation.
Data Analysis: Researchers analyze the information to determine how well the treatment worked. They also want to see whether it is safe for the broader patient population. They submit the data to the FDA, which has strict standards to determine if a treatment is safe for patients.

What Happens in Each Phase of a Clinical Trial?

Clinical trials are typically conducted in four main phases. Depending on the type of trial, it usually takes an average of six to seven years to complete all four phases. Often, treatments don’t make it to the end of Phase 1 or 2. Researchers can start again, but trial variables must be adjusted.

Each phase has a different goal and builds on the results of the previous one.

Phase 1: Safety and Dosage

In Phase 1, a small group gets the treatment. This phase tests for any harmful effects on the body. It is several months long and helps researchers find the correct dose.

Phase 2: Efficacy and Side Effects

Once the researchers know the treatment is generally safe, they test it on even more people. Phase 2 can last several months to two years. The goal is to determine the drug’s effectiveness and further assess safety.

Phase 3: Efficacy and Monitoring of Adverse Reactions

The goal of Phase 3 is to prove the drug’s effectiveness and ensure the drug is safe for a wider group. This phase lasts from one to four years. Participants may show less common or adverse reactions during this phase.

Researchers compare the new treatment to the standard one, a placebo or the baseline disease status. When there are no current treatments, like with PSP, researchers will do one of two trial types:

Placebo-Controlled Trial: If no standard treatment exists, participants are divided into groups. One group receives the new treatment, and the other receives a placebo. This helps determine if the new treatment offers benefits beyond no treatment.

Single-Arm Trial: Sometimes, especially for rare or life-threatening conditions, all participants receive the new treatment. The results are then compared to historical data. This data is from untreated patients or those with the condition before any treatments existed.

In both cases, the goal is to measure the treatment’s effectiveness and safety without an existing treatment for comparison.

After Phase 3 is complete, clinical trial participants typically stop taking the drug until it is approved by the FDA. However, in some cases, participants can take part in open-label extension (OLE), which allows them to continue taking the drug if there are proven benefits and there are no other treatment options for the disease. Note, an OLE mostly happens after Phase 3, but in certain cases, it can happen after Phase 1 or 2.

Phase 4: Post-Market Safety Monitoring

This phase happens after a drug is available to the public. Researchers look at the long-term effects and benefits. They also look for any side effects that may occur over time.

Each phase ensures that the treatment is safe and effective.

Why Are Clinical Trials Important for PSP, CBD and MSA?

Clinical trials are especially important for rare diseases like PSP. They are often the first beacon of hope for those with untreatable diseases.

When clinical trials end, the participants typically stop taking the study drug until it receives approval from their country’s regulatory agency. But if patients are benefitting from the drug and have no other treatment options without it, they are sometimes allowed to continue taking the drug as part of an open-label extension (OLE). People are only allowed to participate in an OLE if they’ve completed the initial portion of the study.

In recent years, researchers have learned more about the causes of PSP and other rare neurodegenerative diseases. Clinical trials can take that knowledge to the next level. Researchers can learn more about the disease by testing new therapies that target the causes.

But, getting enough participants can be challenging because fewer people have these conditions. Additionally, individuals with PSP, CBD or MSA tend to endure delays in receiving a proper diagnosis. These delays make it harder to find people with these conditions. That’s why each participant is crucial in a clinical trial. They help researchers understand these diseases and develop new treatments.

Each clinical trial is given a name. Below are the names and details of several current clinical trials in PSP, CBD and MSA. Find more on our website.

The ORION Trial is a Phase 3 clinical trial for people living with PSP. The ORION study is looking to see whether an investigational drug (a potential new medication) affects the progression of PSP. It will also check for safety and tolerability, which means the body handles taking it well. The study will have two parts, also called “treatment periods.” Each treatment period will last around a year.

The CYPRESS Trial is a Phase 3 clinical trial for people living with MSA. Participants in the trial must be experiencing neurogenic orthostatic hypotension (nOH). The study is trying to determine if an investigational drug reduces nOH symptoms that interfere with daily activities.

The ALLFTD study is recruiting people with PSP and CBD. In this case, there is no clinical trial just yet. The goal is to prepare for future trials by building a cohort of patients who can be available for trials. Researchers collect data from participants through cognitive and behavioral assessments, imaging, blood and cerebral spinal fluid.

How To Take Part in a Clinical Trial

Clinical trials can be a meaningful way to contribute to the future of medicine. It’s also a way for people to access potential treatments that are otherwise unavailable. It’s important to understand what’s involved.

Here are some things to know:

Trial Coordinator: Every clinical trial has a coordinator on its website. Call or email this person to express interest and determine eligibility criteria.
Eligibility Criteria: Each trial has specific criteria outlining who can participate. There may be limitations on age, type of disease or treatment history.
Informed Consent: Before joining, participants receive detailed information about the trial. This will include the trial’s purpose, what will happen, potential risks and benefits. Participants must sign a consent form if they agree to take part in the trial.
Time Commitment and Logistics: Participants need to understand the time commitment involved. Clinical trials can require multiple clinic visits, hospital stays or regular monitoring. There are also complex logistics, such as traveling to the trial site, overnight stays and familial involvement. This can become complicated if a participant can no longer care for themselves. They may need a caretaker to be involved in the trial.

The decision to participate in clinical trials is personal. Anyone interested should always speak with their healthcare provider about the options.

How To Find Clinical Trials

There are a few ways to learn about participating in a clinical trial.

Talk To a Doctor: Physicians often know about ongoing research or trials.
Search Online Registries: Websites like ClinicalTrials.gov or CenterWatch provide listings of clinical trials worldwide.
Connect with CurePSP: Email us at info@curepsp.org.

Clinical trials are key to finding PSP, CBD and MSA treatments. They require careful planning, testing in phases and the participation of many people. When clinical trials succeed, people with life-altering diseases find hope for a better future.