Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
Purpose
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.
Condition
- Congenital Adrenal Hyperplasia
Eligibility
- Eligible Ages
- Between 3 Months and 47 Months
- Eligible 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.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Crinecerfont |
Participants with CAH will receive crinecerfont. |
|
Recruiting Locations
Neurocrine Clinical Site
San Francisco, California 94143
San Francisco, California 94143
Neurocrine Clinical Site
Hartford, Connecticut 06106
Hartford, Connecticut 06106
Neurocrine Clinical Site
Atlanta, Georgia 30329
Atlanta, Georgia 30329
Neurocrine Clinical Site
Chicago, Illinois 60618
Chicago, Illinois 60618
Neurocrine Clinical Site
Indianapolis, Indiana 46202
Indianapolis, Indiana 46202
Neurocrine Clinical Site
Ann Arbor, Michigan 48109
Ann Arbor, Michigan 48109
Neurocrine Clinical Site
Minneapolis, Minnesota 55454
Minneapolis, Minnesota 55454
Neurocrine Clinical Site
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
Neurocrine Clinical Site
Dallas, Texas 75235
Dallas, Texas 75235
Neurocrine Clinical Site
Seattle, Washington 98101
Seattle, Washington 98101
More Details
- Status
- Recruiting
- Sponsor
- Neurocrine Switzerland GmbH