NCT07536269
Not yet recruiting
Phase 2
A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Crinecerfont in Pediatric Participants 3 Months to <4 Years of Age With Classic Congenital Adrenal Hyperplasia
Overview
- Phase
- Phase 2
- Status
- Not yet recruiting
- Sponsor
- Neurocrine Biosciences
- Enrollment
- 20
- Primary Endpoint
- Number of Participants With Treatment-emergent Adverse Events (TEAE)
Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 3 Months to 47 Months (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
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 exclusion criteria may apply.
Arms & Interventions
Crinecerfont
Experimental
Participants with CAH will receive crinecerfont.
Intervention: Crinecerfont (Drug)
Outcomes
Primary Outcomes
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Time Frame: Day 1 up to 28 weeks
Secondary Outcomes
No secondary outcomes reported
Investigators
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