Crinecerfont for Congenital Adrenal Hyperplasia

Recruiting in Palo Alto (17 mi)

+44 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Neurocrine Biosciences

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing a medication called crinecerfont to help children with a hormone disorder called CAH. The study will compare crinecerfont to another treatment over several months. The goal is to see if crinecerfont can safely and effectively balance their hormone levels.

Eligibility Criteria

This trial is for pediatric patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. Participants must be on stable steroidal treatment, have high androgen levels, and agree to birth control if applicable. Exclusions include unstable medical conditions, cancer history unless cured, recent investigational drug use or significant blood loss, other CAH forms, known drug hypersensitivity, certain surgeries or chronic conditions.

Inclusion Criteria

Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.

I have been diagnosed with 21-hydroxylase deficiency CAH.

Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.

+2 more

Exclusion Criteria

You are allergic to any corticotropin-releasing hormone antagonist.

Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.

I have been diagnosed with a form of classic congenital adrenal hyperplasia.

+6 more

Participant Groups

The study tests the effectiveness of Crinecerfont compared to a placebo in children with CAH over a period of 28 weeks. After this phase, all participants receive Crinecerfont for another 24 weeks. Those interested can join an open-label extension for about three years to further assess long-term effects.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: CrinecerfontExperimental Treatment1 Intervention

Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.

Group II: PlaceboPlacebo Group2 Interventions

Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.