Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)

Active study and enrolling

Purpose of study

The main purpose of the CAHtalyst study is to assess if crinecerfont is effective in lowering the daily glucocorticoid dose that CAH patients need to control their symptoms. CAHtalyst is a Phase 3 study.

Participants

Ages Eligible 18 years and older

Qualifications for participation

Criteria

Inclusion Criteria

  1. Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
  2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
  3. Be on a stable regimen of steroidal treatment for CAH.
  4. Patients of childbearing potential must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) or other highly effective contraception during the study.

Exclusion Criteria

  1. Have a diagnosis of any of the other known forms of classic CAH.
  2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
  3. Have a clinically significant unstable medical condition or chronic disease other than CAH.
  4. Have a history of cancer unless considered cured.
  5. Are pregnant.
  6. Have a known history of clinically significant arrhythmia or abnormalities on ECG.
  7. Have a known hypersensitivity to any corticotropin releasing hormone antagonists.
  8. Have received any other investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
  9. Have current substance dependence, or current substance (drug) or alcohol abuse.
  10. Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.

Study information

CAHtalyst 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 adult study participants who have classic Congenital adrenal hyperplasia (CAH). CAH is an inherited disorder that affects the adrenal glands, which produce important hormones in the body. People with CAH have very low levels of the hormone cortisol and high levels of male hormones in their blood. The low cortisol levels can cause symptoms such as nausea, vomiting, dehydration, low blood sugar levels, and shock while the high male hormone levels can lead to acne, excessive body hair, menstrual problems, infertility, metabolic disturbances, and obesity.

The main purpose of the CAHtalyst study is to assess if crinecerfont is effective in lowering the daily glucocorticoid dose that CAH patients need to control their symptoms. CAHtalyst is a Phase 3 study.

The CAHtalyst study will take you approximately 20 months to complete and will consist of the following periods:

# A 1-month Screening period  (2 visits)

# A 6-month Blinded Placebo-Controlled Treatment period (9 visits) :  “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.

# A 1-year Active Treatment period (4 visits): During this period, all participants will receive the study drug crinecerfont.

# A 1-month Follow-Up period (1 visit)

All study supplies and study drug will be provided at no cost to the participant or participant’s insurance provider. Compensation of up to approximately $1500 for completion of all study visits.  Participants may be eligible to receive reimbursements to assist with travel expenses.  This could be in the form of arranging travel or reimbursing the cost of reasonable travel expense.

I’m interested, where do I get more information?

Contact Information

Mary Jane Clifton , Sr. Clinical Research Coordinator 
(314) 362-8681  mclifton@wustl.edu     

Or

Traci Bell, RN, Research Nurse Coordinator
(314) 747-5371 traci.bell@wustl.edu

Investigator

Maamoun Salam, MD

ClinicalTrials.gov Identifier

NCT04490915

CAHtalyst Study