NBI-74788-CAH3003
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
IRAS ID
282848
Contact name
Umasuthan Srirangalingam
Contact email
Sponsor organisation
Neurocrine Biosciences, Inc.
Eudract number
2019-004873-17
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 8 months, 30 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 adults 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 to placebo can reduce glucocorticoid doses while keeping androgen levels in control. Both safety and efficacy of crinecerfont will be assessed.
The study will last approximately 20 months: 1-month screening, 6-month blinded placebo-controlled treatment (participants receiving crinecerfont or placebo), 1-year 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 165 participants in approximately 65 research sites in Europe and North America. Eligible participants must have a diagnosis of classic CAH, be 18 years of age or older, and meet other specific study criteria.REC name
London - City & East Research Ethics Committee
REC reference
20/LO/1005
Date of REC Opinion
15 Oct 2020
REC opinion
Further Information Favourable Opinion