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

Enrollment: 67 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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67 participants in 9 patient groups

Cohort 1: Age 11-17 Treatment with Tildacerfont

Experimental group

Description:

50 mg daily for 12 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 2: Age 11-17 Treatment with Tildacerfont

Experimental group

Description:

200 mg daily for 12 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 3: Age 2-10 Treatment with Tildacerfont

Experimental group

Description:

50, 100, or 200 mg daily for 12 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 4: Age >/= 18 Treatment with Tildacerfont

Experimental group

Description:

200 mg twice daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 5: Age >/= 18 Treatment with Tildacerfont

Experimental group

Description:

300 or 400 mg twice daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 6: Age 11-17 Treatment with Tildacerfont

Experimental group

Description:

200 mg twice daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 7: Age 2-10 Treatment with Tildacerfont

Experimental group

Description:

200 mg twice daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 8: Age 11-17 Treatment with Tildacerfont

Experimental group

Description:

300 or 400 mg twice daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont

Cohort 9: Age 2-10 Treatment with Tildacerfont

Experimental group

Description:

300 or 400 mg twice daily for 4 consecutive weeks.

Treatment:

Drug: Tildacerfont