A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children with CAH

Spruce Biosciences

Status and phase: Terminated; Phase 2

Conditions: Congenital Adrenal Hyperplasia; 21-OHD

Treatments: Drug: Tildacerfont

Study type: Interventional

Funder types: Industry

Identifiers

NCT05128942

SPR001-205

Details and patient eligibility

About

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

Full description

This is a study to evaluate the safety and efficacy of tildacerfont in participants with Congenital Adrenal Hyperplasia (CAH). Treatment will consist of 4 or 12 weeks of continuous dosing followed by safety follow up.

Enrollment: 65 patients

Sex: All

Ages: 2 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male and female subjects aged 2+
• Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis
• Stable dose of GC replacement for at least 1 month prior to screening

Exclusion criteria

• History of bilateral adrenalectomy or hypopituitarism
• Clinically significant unstable medical conditions, illness, or chronic diseases
• History of active bleeding disorders
• Females who are pregnant or nursing

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

---

65 participants in 9 patient groups

Cohort 1: Age 11-17 Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 12 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 2: Age 11-17 Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 12 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 3: Age 2-10 Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 12 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 4: Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 5: Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 6: Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered twice daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 7: Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 8: Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 9: Treatment with Tildacerfont

Experimental group

Description:

Oral Tildacerfont administered daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont