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About CAHtalyst Pediatric Study

About CAHtalyst Pediatric Study

CAHtalyst Pediatric Study is a clinical research study evaluating a study drug called crinecerfont. The study is sponsored by Neurocrine Biosciences.

The study drug will be evaluated in children and adolescents between 2 and 17 years of age who have classic CAH. Classic CAH is a genetic disorder that affects the balance of hormones made by the adrenal glands. For children, this hormone imbalance, as well as the glucocorticoid medication used to treat the imbalance, can cause problems with growth and development.

The primary purpose of this clinical research study is to evaluate the safety and effectiveness of crinecerfont in children and adolescents with classic CAH. CAHtalyst Pediatric Study is a Phase 3 study.

Up to approximately 60 weeks

Approximately 81 participants

Approximately 35 study centers around the world


The CAHtalyst Pediatric Study Journey

CAHtalyst Pediatric Study will last up to approximately 60 weeks and consists of the following:

  • Screening Period (4 weeks)
  • Study Period 1 - Blinded Placebo-Controlled Treatment (28 weeks)"Blinded placebo-controlled treatment" means neither the participant nor the study team will know if the participant is receiving the study drug or a placebo. The placebo looks like crinecerfont but it does not contain any active drug. Participants will be assigned to crinecerfont or placebo at random. There is a 67% chance of receiving crinercerfont.
  • Study Period 2 - Open-Label Treatment (24 weeks)During this period, all participants will receive the study drug crinecerfont.
  • Follow-Up Period (4 weeks)

Those who qualify to participate in this clinical research study will receive study-related medical exams and study-related laboratory tests at no cost. Compensation for time and travel may also be available.

Study Visits

During CAHtalyst Pediatric Study, participants will attend regular visits at the study center to complete tests and procedures, which will be assessed by study doctors. If participants prefer, it may be possible for some visits to take place in their own home to minimize traveling.

Some examples of procedures that will take place are physical examinations, blood tests, and urine samples. Participants will also be asked to complete different questionnaires. Most visits will take 1-4 hours but some may last up to 7 hours. Prior to some visits, participants may be asked to fast for 8 hours. All of these tests and procedures will help the study doctors to find out more about crinecerfont and its effect on the participants’ health and symptoms.

Check Eligibility

Answer the following questions and click Submit to check if your child may be eligible for CAHtalyst Pediatric Study.

Please note that clicking Submit does not mean that you (or your child) agree to participate in the study.


Are they between 2 and 17 years of age?


Do they weigh at least 10 kg?


Do they have a medically confirmed diagnosis of classic congenital adrenal hyperplasia (CAH)?


Are they currently taking any glucocorticoid medication for their condition?


Do they have a known hypersensitivity or allergy to any corticotropin-releasing hormone (CRH) receptor antagonist?


Are they currently pregnant or lactating?

Your child may be eligible

Based on your answers, your child is potentially eligible for the study. If you would like to be contacted by your nearest site in relation to this study, please enter your contact details below:

Thank you

Thank you. Your information has been sent.



There has been an error submitting your information. Please resubmit the form.


Your child is not eligible

Based on your answers, your child is currently not eligible for the study. If your answers to any of these questions change in future, please check back again to see if your child is eligible. Thank you for your interest in this study.


Find a Study Center

Please use the map below to find a study center near you. Study centers will be added to the map when they begin enrolling participants. If there is no center that is enrolling in your location at the moment, please check back at a later time.


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 Pediatric Study 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 a participant takes part in a clinical study, it is important that they (and their parents/caregivers) are aware of all aspects of the study, including what will happen at the study visits, how they will take the study drug, what is expected of them when they participate, any potential risks and benefits involved, and many other things. Before participants agree to participate, the study doctor will meet with them to tell them everything they need to know. Participants (and their parents/caregivers) will be given written information to read that they can discuss with their family and friends, and they will be given time to ask any questions they may have.

When the participant (and their parents/caregivers) understand what is involved in the study and they decide to take part, they will be given an Informed Consent form to sign, which documents that they voluntarily consent to taking part in the study. Even if a participant (or their parents/caregivers) provide informed consent, they are free to leave the study at any time and for any reason.

Participation in CAHtalyst Pediatric Study will last for up to approximately 60 weeks. However, participation is voluntary and participants 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. Participants will be informed about all of the known potential side effects of the study drug and study procedures before they decide to take part in this clinical study. If any further risks are discovered while the study is ongoing, participants will be informed immediately.

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

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

Participants who are enrolled in the study will be randomly assigned by a computer to receive either crinecerfont or placebo during the first 28 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 the participants nor the study doctor or nurses will know if a participant is receiving crinecerfont or placebo. In the case of an emergency, it is possible for the study doctor to obtain this information.

Participants are free to leave the study at any time, and this will not affect their future care in any way. There are a number of possible alternative treatment options available to participants for their condition. The participant’s doctor will continue to look after them and advise them about alternative treatment options.

If participants (or their parents/caregivers) complete the questionnaire in the ‘Check Eligibility’ section and are potentially eligible for CAHtalyst Pediatric Study, they will be asked to enter their contact details (i.e. name, email, telephone number) and to choose the study center nearest to them. The contact details provided will be securely transmitted to the chosen study center. This allows staff at the chosen study center to contact the participant (or their parents/caregivers) about the study. The provided contact details will only be visible to staff at the chosen study center and to a limited number of Longboat staff for technical support and study management reasons. The provided contact details will never be visible to the sponsor – the company that is running this clinical study.