Crinecerfont for Congenital Adrenal Hyperplasia in Young Children

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old

Recruiting Phase 2 Interventional Study
Congenital Adrenal Hyperplasia
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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Review the details below, then apply to join this clinical trial.

At a Glance
Age
3 Months – 47 Months
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
20 (estimated)
Sponsor
Neurocrine Switzerland GmbH · Industry
Who this trial is looking for

This trial is looking for young children aged 3 months to less than 4 years with a specific type of adrenal condition called congenital adrenal hyperplasia (CAH). Participants will be given crinecerfont to see how safe it is and how well it is tolerated.

Are You a Good Fit for This Trial?
Must be taking Hydrocortisone

You may be able to join if

  • I have been diagnosed with classic CAH due to 21-hydroxylase deficiency.
  • I have been on hydrocortisone treatment for at least 3 months.
  • I weigh at least 4.5 kilograms.
  • I have normal newborn screening results except for elevated 17-OHP.

You may not be able to join if

  • I have been diagnosed with another form of classic CAH.
  • I have another condition that needs daily steroid therapy.
  • I have any other serious medical condition or chronic disease.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

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About This Trial

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.

Trial Locations
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Eligibility 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 …
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