NCT05128942

A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH

A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics of SPR001 (Tildacerfont) in Children Aged 2 to 17 Years With Congenital Adrenal Hyperplasia (CAH)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 67 (actual)
• Sponsor: Spruce Biosciences · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

An investigation of the safety and efficacy of tildacerfont in participants with CAH.

Detailed description

This is a Phase 2 open-label study with up to 10 cohorts that will evaluate the safety, efficacy, and PK of different tildacerfont dosing regimens potentially up to 200mg QD for 12 weeks in children with classic CAH, and up to 400mg BID for 4 weeks in children and adults with classic CAH.

Conditions

• Congenital Adrenal Hyperplasia
• 21-OHD

Interventions

Type	Name	Description
DRUG	Tildacerfont	Oral tablet formulation taken once daily in combination with glucocorticoid therapy.

Timeline

• Start date: 2021-12-10
• Primary completion: 2025-01-31
• Completion: 2025-01-31
• First posted: 2021-11-22
• Last updated: 2025-12-24
• Results posted: 2025-12-24

Locations

13 sites across 1 country: United States

Regulatory

• FDA-regulated drug study