Congenital Adrenal Hyperplasia
Conditions
Keywords
Congenital Adrenal Hyperplasia, CAH, Crinecerfont, Pediatric
Brief summary
The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to \<4 years of age with CAH.
Interventions
DRUGCrinecerfont
Oral solution
Sponsors
Neurocrine Switzerland GmbH
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Months to 47 Months
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD) * Have received at least 3 months of hydrocortisone treatment before Day 1, with a stable dose for at least 4 weeks before screening. * Have a body weight of at least 4.5 kilograms (kg) at screening. * Have a newborn screen that is otherwise normal except for elevated 17-OHP or any other abnormality on newborn screen that was cleared upon evaluation by a pediatric specialist. Key
Exclusion criteria
* Have a known or suspected diagnosis of any of the other forms of classic CAH. * Have any condition besides CAH that requires chronic daily therapy with orally administered steroids. * Have any other clinically significant medical condition or chronic disease. Note: Other protocol-defined inclusion and
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAE) | Day 1 up to 28 weeks |
Countries
United States
Contacts
CONTACTNeurocrine Medical Information Call Center
STUDY_DIRECTORClinical Development Lead
Neurocrine Biosciences
Outcome results
None listed