Clinical Trials

Congenital Adrenal Hyperplasia

Neurocrine Biosciences completed a Phase II study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of crinecerfont in adult patients with classic congenital adrenal hyperplasia (CAH). The final results from this open-label, multiple-dose, dose-escalation study were reported in June 2020. Neurocrine Biosciences plans to initiate a single, global registrational study of crinecerfont in adult patients with classic CAH in the second half of 2020.

Neurocrine Biosciences is conducting a Phase II, open-label, multiple-dose, dose-escalation study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of crinecerfont in pediatric female and male patients (14 to 17 years of age) with classic 21-hydroxylase deficiency CAH. For more information on this Phase II study of crinecerfont in pediatric patients with CAH, please visit or

Parkinson’s Disease

NBIb-1817 is currently being evaluated in the Phase II RESTORE-1 trial in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day, as measured by a validated self-reported patient diary. For more information about the RESTORE-1 clinical trial, please visit

NBIb-1817 is being developed by Neurocrine Biosciences and Voyager Therapeutics as part of a strategic collaboration announced in January 2019.

Chorea Associated with Huntington’s Disease

Neurocrine Biosciences is conducting KINECT-HD, a multi-center randomized, double-blind, placebo-controlled Phase III study to evaluate the efficacy, safety and tolerability of valbenazine for the treatment of chorea in patients with Huntington’s disease (HD). HD is a hereditary progressive neurodegenerative disorder, in which many patients may experience chorea, a troublesome involuntary movement disorder. For more information on this Phase III study of valbenazine for the treatment of chorea associated with HD, please visit or

New VMAT2 Inhibitor

Neurocrine Biosciences filed an Investigational New Drug (IND) application and completed dosing in the single ascending dose portion of a Phase I study designed to assess initial safety, tolerability and pharmacokinetics of a novel, internally discovered VMAT2 inhibitor. This compound has the potential to be used in the treatment of several neurology and/or psychiatry disorders. The multiple dosing portion of this Phase I study is ongoing and is expected to be completed during the first half of 2019.

Compassionate Use

Policy on expanded access to investigational medicines

Individuals interested in participating in clinical trials for Neurocrine Biosciences’ products may visit the “Pipeline” section of the website for information on ongoing clinical trials. At this time, Neurocrine Biosciences does not have an expanded access program that allows patients to have access to the Company’s investigational products prior to FDA approval. Neurocrine Biosciences will update this page as the Company grows and reassesses the approach to expanded access programs. If you have any questions, please contact