A clinical research study is a medical research study involving human participants. It tests the use of a medical procedure, medical device, drug, or potential treatment, and is led by researchers and study doctors. Clinical studies provide valuable information, and may help researchers to understand a condition better and learn how best to treat it, or may lead to new or better treatments being made available.

A study drug must pass three phases of clinical testing before it is approved by a country’s regulatory authority and made available to the public. CAHtalyst is a Phase 3 study.

Phase 1: The study drug is given to a small group of healthy volunteers or patients. Researchers look to see how much is safe to take and how the human body responds when it receives the drug.

Phase 2: The study drug is given to a small group of people with the target disease or condition it is intended to treat. In this phase, researchers evaluate what dose is appropriate and begin to learn more about how safe the drug is and how well it works.

Phase 3: Researchers test the safety and effectiveness of the study drug in a larger group of participants over a longer period of time. Sometimes, comparisons are made between the study drug and other medications that are already approved for the same purpose.

Phase 4: A Phase 4 study is conducted after a study drug has been approved for public use. The approved drug may be compared with other drugs on the market or researchers may monitor the drug’s long-term effectiveness and impact on patients’ quality of life.

Before you take part in a clinical study, it is important that you are aware of all aspects of the study, including what will happen at the study visits, how you will take the study drug, what is expected of you when you participate, any potential risks and benefits involved, and many other things. Before you agree to participate, the study doctor will meet with you to tell you everything you need to know. You will be given written information to read that you can discuss with your family and friends and you will be given time to ask any questions you may have.

When you understand what is involved in the study and you decide to take part, you will be given an Informed Consent form to sign, which documents that you voluntarily consent to taking part in the study. Even if you provide informed consent, you are free to leave the study at any time and for any reason.

After the 4-week Screening period, your participation in the CAHtalyst study will last for approximately 19 months. However, participation is voluntary and you are free to leave the study at any time.

All medications, both those that are approved and those undergoing clinical studies, may potentially cause side effects – some of these are known, but there could be others that have not previously been observed. Study procedures may also involve risks. You will be informed about all of the known potential side effects of the study drug and study procedures before you decide to take part in this clinical study. If any further risks are discovered while the study is ongoing, you will be informed immediately.

If your condition gets worse, your study doctor will discuss additional or alternative treatment options with you, if necessary. These options may include: continuing with the study treatment; withdrawing from the study; switching to a different type of treatment; or taking medication to relieve your symptoms.

Your health and well-being are of utmost importance during the study, and any decisions regarding the study treatment or any alternative treatments will be made with that in mind.

If you are enrolled in the study, you will be randomly assigned by a computer to receive either crinecerfont or placebo for the first 24 weeks. The placebo looks like crinecerfont but does not contain any active drug. Placebos are commonly used in clinical studies to help understand if the drug being researched is effective. In this study, one of every three participants will take placebo. Neither you nor the study doctor or nurses will know if you are receiving crinecerfont or placebo. In the case of an emergency, it is possible for the study doctor to obtain this information.

You are free to leave the study at any time and this will not affect your future care in any way. There are a number of possible alternative treatment options available to you for your condition. Your doctor will continue to look after you and advise you about alternative treatment options.

If you complete the questionnaire in the ‘Am I Eligible?’ section and are potentially eligible for CAHtalyst, you will be asked to enter your contact details (i.e. name, email, telephone number) and to choose the study center nearest to you. The contact details you provide will be securely transmitted to your chosen study center. This allows staff at your chosen study center to contact you about the study. Your contact details will only be visible to staff at your chosen study center and to a limited number of Longboat staff for technical support and study management reasons. Your contact details will never be visible to the sponsor – the company who is running this clinical study.

Records of your participation in this study will be held confidential except when disclosure is required by law or as described in the Informed Consent document. The study site, study doctor, the sponsor and their designees, and certain regulatory agencies (e.g. the US Food and Drug Administration) will be able to inspect and copy confidential study records that could identify you. Therefore, absolute confidentiality cannot be guaranteed during the study. If the results of this study are published or presented at scientific meetings, it will not be possible to identify individual participants.