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  • Research type

    Research Study

  • Full title

    A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment



  • Contact name

    Mehul Dattani

  • Contact email

  • Sponsor organisation

    Neurocrine Biosciences, Inc.

  • Eudract number


  • Duration of Study in the UK

    3 years, 0 months, 0 days

  • Research summary

    Congenital adrenal hyperplasia (CAH) is an inherited disorder in which the adrenal glands (located above each kidney) are unable (because of an enzyme deficiency) to produce normal amounts of the steroid hormones cortisol and (sometimes) aldosterone that regulate many important functions in the body including metabolism, immune function, salt, and blood pressure in the body. In addition, too much male sex hormones (androgens) are produced. This usually requires treatment with glucocorticoid (steroid medicine) at doses higher than what is needed to replace the missing cortisol, which (when given over a long period of time) can cause a number of side effects and medical complications.
    This Phase 3 randomized double-blind placebo-controlled trial in children with classic CAH involves crinecerfont, a medicine that binds to and blocks a receptor (target) on the pituitary gland (“master” hormone gland that sits below the brain). Blocking this receptor is expected to reduce hormone levels that cause the adrenal glands to produce too much androgens. By reducing and even normalizing androgen levels, it is expected that the glucocorticoid dose could be reduced closer to the replacement level needed for treating just the cortisol deficiency. The primary purpose of this study is to evaluate whether crinecerfont compared with placebo can reduce adrenal steroid levels during a glucocorticoid-stabel period. Both safety and efficacy of crinecerfont will be assessed.
    The study will last approximately 15 months: 1-month screening, 7-month blinded placebo-controlled treatment(participants receiving crinecerfont or placebo), 6-month active treatment (all participants receiving crinecerfont), 1-month follow-up period. Visits may include physical examinations, blood tests, urine samples, body scans, and questionnaires. The study will include 81 participants in approximately 40 research sites in Europe and North America. Eligible participants must have a diagnosis of classic CAH, be 2 to 17 years of age and meet other specific study criteria.

  • REC name

    London - City & East Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    21 Apr 2022

  • REC opinion

    Further Information Favourable Opinion