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Label indication CRENESSITY is indicated as adjunctive treatment to glucocorticoid replacement to control androgens in adults and pediatric patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH) . CRENESSITY is a corticotropin-releasing factor type 1 receptor antagonist indicated as adjunctive treatment to glucocorticoid replacement to control androgens in adults and pediatric patients 4 years of age and… FDA label
Also known as Crinecerfont, Crenessity
NBI-74788 · 6 trials · 2 indications
Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
Percent changes in 17-OHP were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The 2 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Percent changes in 17-OHP were assessed through the collection of samples from 0600 hours to 1000 hours both prior to study drug administration (i.e., at baseline) and after 14 days of study drug dosing. The 3 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
| Arm | Type | Description |
|---|---|---|
| Crinecerfont | EXPERIMENTAL | Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks. |
| Placebo | PLACEBO_COMPARATOR | Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks. |
| Crinecerfont 50 milligrams (mg) Twice Daily (BID) | EXPERIMENTAL | Crinecerfont administered orally for 14 consecutive days. |
| Cohort 1 (50 mg QHS) | EXPERIMENTAL | NBI-74788 50 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 2 (100 mg QHS) | EXPERIMENTAL | NBI-74788 100 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 3 (100 mg QPM) | EXPERIMENTAL | NBI-74788 100 mg once daily in the evening (QPM) administered orally for 14 consecutive days. |
| Cohort 4 (100 mg BID) | EXPERIMENTAL | NBI-74788 100 mg twice daily (BID) administered orally for 14 consecutive days. |
| Name | Type | Description |
|---|---|---|
| Crinecerfont | DRUG | CRF type 1 receptor antagonist |
| Placebo | DRUG | Non-active dosage form |
| NBI-74788 | DRUG | Capsule, administered daily. |
Inclusion Criteria: * Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit. * Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency. * Be on a stable steroid regimen. * Have elevated ...
NBI-74788 is an investigational small molecule developed by Neurocrine Biosciences for congenital adrenal hyperplasia (CAH), an endocrine disorder. It is designed to act on 21-hydroxylase, an enzyme involved in steroid hormone production. The drug has been studied in a Phase 2 clinical trial in adults with CAH.
NBI-74788 is being developed as a treatment for congenital adrenal hyperplasia (CAH), a condition in which the adrenal glands produce abnormal levels of steroid hormones. It is intended for adults with CAH and has been evaluated in a Phase 2 study measuring safety, tolerability, pharmacokinetics, and pharmacodynamics.
NBI-74788 targets 21-hydroxylase, an enzyme that plays a central role in cortisol and aldosterone synthesis. By acting on this enzyme, the drug is intended to modulate the steroidogenic pathway that is dysregulated in congenital adrenal hyperplasia. The target class is an enzyme.
NBI-74788 is being developed by Neurocrine Biosciences, Inc., which trades on the Nasdaq under the ticker NBIX. The company is the sponsor of the clinical program evaluating the drug in congenital adrenal hyperplasia.
NBI-74788 is in Phase 2 development. It is an investigational drug and has not been approved by the FDA. The Phase 2 trial in adults with congenital adrenal hyperplasia has been completed, and no trials are currently active.
NBI-74788 has been studied in one clinical trial, NCT03525886, titled "Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia." The Phase 2 trial enrolled 18 adults in the United States and is completed. It was a controlled study without randomization or double blinding.