Study Overview: This study is focused on understanding how safe and tolerable crinecerfont is for young children under 4 years old who have a condition called Classic Congenital Adrenal Hyperplasia (CAH). CAH is a condition where the adrenal gland doesn't work properly.
Eligibility: To join, a child must be diagnosed with classic CAH due to 21-hydroxylase deficiency. They should have been on hydrocortisone treatment for at least 3 months with a stable dose for the last 4 weeks. They must weigh at least 4.5 kg and have a newborn screening with no issues except elevated 17-OHP (a hormone).
Key Points:
- This study involves taking a medication called crinecerfont to see if it's safe for young children with CAH.
- Participants should have been on a stable hydrocortisone dose for at least 4 weeks before the study.
- Children with other significant medical conditions or who need daily oral steroids for other reasons cannot join.
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