Clinical trial

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

NCT05128942

Name

SPR001-205

Description

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

Trial arms

Trial start

2021-12-10

Estimated PCD

2024-12-01

Trial end

2024-12-01

Status

Recruiting

Phase

Early phase I

Treatment

Tildacerfont

Oral tablet formulation taken once daily in combination with glucocorticoid therapy.

Arms:

Cohort 1: Age 11-17 Treatment with Tildacerfont, Cohort 2: Age 11-17 Treatment with Tildacerfont, Cohort 3: Age 2-10 Treatment with Tildacerfont, Cohort 4: Treatment with Tildacerfont, Cohort 5: Treatment with Tildacerfont, Cohort 6: Treatment with Tildacerfont, Cohort 7: Treatment with Tildacerfont, Cohort 8: Treatment with Tildacerfont, Cohort 9: Treatment with Tildacerfont

Other names:

SPR001

Size

Primary endpoint

Number of participants with Treatment-emergent adverse event (TEAE) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

12 weeks

Eligibility criteria

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Subjects will be assigned to treatment cohorts by age.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 55, 'type': 'ESTIMATED'}}

Updated at

2024-04-02

1 organization

1 product

2 indications

Organization

Spruce Biosciences

Product

Tildacerfont

Indication

Congenital Adrenal Hyperplasia

Indication

21-Hydroxylase Deficiency