Completed
A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia
What is being tested
Crinecerfont
Drug
Who is being recruted
Congenital Abnormalities+15
+ Urogenital Diseases
+ Adrenal Gland Diseases
From 14 to 17 Years
+21 Eligibility Criteria
How is the trial designed
Treatment Study
Phase 2
Interventional
Study Start: December 2019
Summary
Principal SponsorNeurocrine Biosciences
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner
Study start date: December 12, 2019
Actual date on which the first participant was enrolled.This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH).
Official TitleA Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia
Principal SponsorNeurocrine Biosciences
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.Design Details
8 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Conditions
Criteria
Any sex
Biological sex of participants that are eligible to enroll.From 14 to 17 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Congenital AbnormalitiesUrogenital DiseasesAdrenal Gland DiseasesAdrenal Hyperplasia, CongenitalEndocrine System DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsGonadal DisordersMetabolic DiseasesMetabolism, Inborn ErrorsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNutritional and Metabolic DiseasesDisorders of Sex DevelopmentUrogenital AbnormalitiesGenetic Diseases, InbornSteroid Metabolism, Inborn ErrorsAdrenogenital SyndromeFemale Urogenital DiseasesMale Urogenital Diseases
Criteria
7 inclusion criteria required to participate
Be in good general health.
Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
Be on a stable regimen of steroidal treatment for CAH that is expected to remain stable throughout the study.
Participants of childbearing potential must be instructed on the proper use of barrier methods of contraception and agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently from screening until the final study visit or a prespecified window after the last dose of study drug, whichever is longer.
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14 exclusion criteria prevent from participating
Have a clinically significant unstable medical condition or chronic disease, or malignancy.
Had a medically significant illness within 30 days of screening.
Have a known or suspected differential diagnosis of any of the other known forms of classic CAH.
Have a history that includes bilateral adrenalectomy, hypopituitarism, or other condition requiring daily therapy with orally administered glucocorticoids.
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Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.Treatment Groups
Study Objectives
One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalCrinecerfont administered orally for 14 consecutive days.
Study Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 6 locations
Suspended
Suspended
Neurocrine Clinical Site
Aurora, United StatesSuspended
Neurocrine Clinical Site
Ann Arbor, United StatesSuspended
Neurocrine Clinical Site
Minneapolis, United StatesCompleted6 Study Centers