Clinical Trials

Without clinical studies, the development of new KRD treatments and therapies would be impossible. Patients like you play a key role in the research and development process by volunteering to participate.

Unfortunately, many clinical trials fail to recruit enough volunteers. This is especially true of the development of KCNQ2 treatments.

By participating in a clinical trial, you can help researchers discover new treatments for KRDs, gain access to care from medical experts, and potentially benefit from a medical breakthrough before it’s released to the public.

The U.S. Food and Drug Administration (FDA) defines the four phases of clinical trials. A new drug must pass through each phase before the FDA will review it for approval. 

• Phase I trials: Researchers test a drug or treatment in a small group of people (20–80) for the first time. The purpose is to study the drug or treatment to assess safety and identify potential side effects.
• Phase II trials: The new drug or treatment is given to a larger group of people (100–300) to determine its effectiveness and further assess its safety.
• Phase III trials: The new drug or treatment is given to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments, and collect information to ensure its safe use.
• Phase IV trials: After a drug is approved by the FDA and made available to the public, researchers track its safety in the general population, seeking more information about a drug or treatment’s benefits and optimal use.

Before taking part in a clinical trial, you must meet the eligibility criteria and then move to informed consent, a process where you and the study doctor will discuss what participating in the study will be like.

Eligibility Criteria

Every study has different guidelines for who can or cannot participate. These guidelines, called eligibility criteria, describe characteristics that must be shared by all participants. The criteria differ from study to study.

Specific eligibility criteria help control for certain variables, enabling researchers to achieve accurate and meaningful results. It also helps protect vulnerable individuals from increased risks.

Ethics review boards approve eligibility criteria, and no exceptions can be made.

Informed Consent

The informed consent form will outline the purpose of the research, its duration, and a list of the procedures you will undergo.

The form should also address participation risks, how your privacy will be protected, and the study team's contact information.

Remember, by signing an informed consent form, you are not signing a contract. You can leave the study at any time, and the people who monitor the trial can also remove you if they feel your health is declining.

Time Commitment

Time is one of the biggest commitments you’ll make when you participate in a clinical trial.

You’ll likely need to attend frequent checkups and undergo tests, like blood and urine tests, to monitor the safety and effectiveness of the treatment.

For a list of KCNQ2 trials, see: here.

In clinical trials that compare a new product or therapy with another that already exists, researchers try to determine if the new one is as good, or better than, the existing one. In some studies, you may be assigned to receive a placebo (an inactive product that resembles the test product, but without its treatment value).

Comparing a new product with a placebo can be the fastest and most reliable way to show the new product’s effectiveness. However, placebos are not used if you would be put at risk — particularly in the study of treatments for serious illnesses — by not having effective therapy. You will be told if placebos are used in the study before entering a trial.

Randomization is the process by which treatments are assigned to participants by chance rather than by choice. This is done to avoid any bias in assigning volunteers to get one treatment or another. The effects of each treatment are compared at specific points during a trial. If one treatment is found superior, the trial is stopped so that the most volunteers receive the more beneficial treatment. This video helps explain randomization for all clinical trials.

"Blinded" (or "masked") studies are designed to prevent members of the research team and study participants from influencing the results. Blinding allows the collection of scientifically accurate data. In single-blind ("single-masked") studies, you are not told what is being given, but the research team knows. In a double-blind study, neither you nor the research team are told what you are given; only the pharmacist knows. Members of the research team are not told which participants are receiving which treatment, in order to reduce bias. If medically necessary, however, it is always possible to find out which treatment you are receiving.

Off-Label Use

When a drug is used in a way that is different from that described in the FDA-approved drug label, it’s said to be an “off-label” use. This can mean that the drug is:

• Used for a different disease or medical condition
• Given in a different way (such as by a different route)
• Given in a different dose than in the approved label

Off-label is also called non-approved or unapproved use of a drug. The off-label use of FDA-approved drugs is legal in the United States and many other countries. Off-label drug use is common in the treatment of rare diseases.

The biggest problem with off-label drug use is obtaining approval from a payer (insurance company) to reimburse for the off-label drug use. Many insurance companies will not pay for an expensive drug that’s used in a way that’s not listed in the approved drug label. They do this on the grounds that its use is “experimental” or “investigative.” This financial burden can be an enormous strain on patients and families, especially when the cost of off-label treatments runs very high.

The health insurance coverage laws and regulations are complex. If your doctor is thinking about off-label drug use, you and your doctor should carefully check your health plan’s coverage. If you are denied coverage, it might help if the doctor sends the insurer copies of peer-reviewed journal articles or other respected sources that support the off-label use.