Idiopathic hypersomnia is a sleep disorder that causes extreme sleepiness during the day, even after a full night’s rest. This type of tiredness is called excessive daytime sleepiness (EDS). Idiopathic hypersomnia and narcolepsy both cause EDS, but they’re different conditions. Even doctors sometimes have trouble telling them apart. “I’m now getting another sleep study to see if it's narcolepsy or idiopathic hypersomnia,” one MyNarcolepsyTeam member wrote.

Researchers are continuing to study potential treatment options for idiopathic hypersomnia. These drugs are tested in clinical trials, which are studies with volunteer participants. In these trials, researchers evaluate how well a new treatment works and whether it has fewer side effects than current options.

Before any therapy can be approved by the U.S. Food and Drug Administration (FDA), it must first go through clinical trials to show that it’s safe and effective. Clinical trials can study new medications, different drug combinations, new ways to use existing drugs, and other kinds of therapies, which are often called investigational drugs or investigational treatments.

Clinical Trials Have Led to New Treatments for Idiopathic Hypersomnia

The FDA has approved several treatments for narcolepsy but just one specifically for idiopathic hypersomnia.

In August 2021, the FDA approved the first medication for idiopathic hypersomnia — a combination of calcium, magnesium, potassium, and sodium oxybates (Xywav). This medication works by reducing the activity in the central nervous system. In clinical trials, people who took the drug had less severe EDS symptoms than those who took a placebo (a lookalike pill that doesn’t contain medicine).

In August 2021, the FDA approved the first medication for idiopathic hypersomnia.

Before 2021, doctors treated idiopathic hypersomnia with off-label medications — drugs that were approved for other conditions but had shown potential to help with idiopathic hypersomnia.

Researchers are testing several other possible therapies for idiopathic hypersomnia. The U.S. government keeps an online list of current clinical trials looking for people with idiopathic hypersomnia to join.

Read on to learn more about what it’s like to take part in a clinical trial.

You May Get Access to New Treatments

Clinical trials give people with specific conditions a chance to try investigational treatments. These new therapies aren’t yet available to the public and may work better than current options, but there’s no guarantee they’ll be effective. Like all medications, they can also cause side effects.

It’s also important to know that some people who participate in a clinical trial may receive a placebo, not the new treatment. Researchers use placebos to compare results and see how well the investigational treatment works. This is known as a placebo-controlled study. In some clinical trials, participants who first get a placebo may later receive the investigational treatment.

You’ll Be Told Whether You May Receive a Placebo

Researchers will tell potential participants if there’s a chance they might receive a placebo instead of the investigational treatment. This information may affect whether you want to participate.

To keep the results fair and avoid bias, participants are randomly assigned to different groups. This process is called randomization. Many clinical trials are also double-blind (or double-masked), which means neither the participants nor the researchers know who is getting which treatment. This information is revealed only after the trial ends or if a participant needs to switch treatments or leaves the trial.

In some cases, if the study drug shows strong results, it may be offered to all participants, including those originally in the placebo group. If you’re thinking about joining a clinical trial, ask whether the new treatment could become available to everyone in the study if it works well.

Researchers Will Prioritize Safety

Joining a clinical trial is voluntary, and you can leave at any time for any reason. In the U.S., clinical trials are closely regulated by the federal government to keep participants safe.

Before a new treatment is tested in people, researchers do many laboratory and animal tests to check its safety and effectiveness. If these early results seem promising, the treatment may move forward into human testing.

Institutional Review Boards Help Protect Participants

Most trials are reviewed by an institutional review board (IRB) — a group of scientists, doctors, and other specialists who make sure the study is as safe as possible and that potential benefits outweigh the risks. If a trial becomes unsafe, the IRB can stop it right away.

Informed Consent

Before joining a clinical trial, participants go through an informed consent process. This means the researchers will explain everything you need to know about the study — what’s involved, any risks, and what you can expect. You’ll have time to ask questions and talk with your family, doctor, or any other advisers before you decide. You’ll also receive updates and information throughout the study.

If you’re thinking about joining a clinical trial, ask your doctor what questions to consider. These might include whether you’ll need to travel and if you’ll need to pay any out-of-pocket costs.

Phases of Clinical Trials

Clinical trials are conducted in different phases, each designed to keep participants safe while testing how well a treatment works and what risks it may have. These phases allow researchers to carefully study the treatment before moving on:

• Phase 1 focuses on determining the highest dose that can be given safely. These studies usually involve 20 to 100 people.
• Phase 2 looks at whether the treatment helps with a specific condition and continues to monitor side effects. These studies have around 100 to 300 participants.
• Phase 3 and 4 trials confirm efficacy (how well the treatment works) and monitor for any rare or long-term side effects. These later phases may include several hundred or thousands of participants.

Earlier phases may last up to two years, whereas later phases can continue for four years or longer.

Some Costs May Be Covered

Joining an idiopathic hypersomnia clinical study is a personal decision. Some people participate for the chance to try a new treatment that might help their symptoms. Others are motivated by the opportunity to support research that could lead to better treatments.

There are many reasons to participate in an idiopathic hypersomnia clinical study, including the chance to receive a new therapy, the desire to contribute to research, and the opportunity for additional care and monitoring.

Financial considerations may also play a role. In some cases, the sponsor (the organization running the clinical trial) may cover the costs of the study treatment. Health insurance may also help out. Some people also appreciate the extra care, monitoring, and follow-up provided in clinical studies.

Every clinical trial has its own eligibility criteria, which are rules about who can participate. These factors may include your age, sex, and medical history and the stage of your disease.

The Hypersomnia Foundation has more information on clinical trials. You can talk to your doctor to find out if a clinical trial may be a good fit for you.

Find Your Team

MyNarcolepsyTeam is the social network for people living with narcolepsy, excessive daytime sleepiness, and idiopathic hypersomnia. On MyNarcolepsyTeam, more than 11,000 members come together to ask questions, give advice, and share their stories with others who understand life with these conditions.

Would you consider participating in a clinical study for idiopathic hypersomnia? Share your experience in the comments below, or start a conversation by posting on your Activities page.