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

Phase 2TerminatedNCT05128942

Spruce Biosciences67 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Congenital Adrenal Hyperplasia 21-OHD

Interventions

TildacerfontDRUG

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

Eligibility

Sex: ALLMin age: 2 Years

Medical Language ↔ Plain English

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

Locations (13)

Spruce Study Site

Sacramento, California, United States

Spruce Study Site

San Diego, California, United States

Spruce Study Site

Chicago, Illinois, United States

Outcomes

Primary Outcomes

Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0

To evaluate safety of tildacerfont in participants with CAH as measured by number of subjects with adverse events following dosing by CTCAE version 5.0

Time frame: 12 weeks

Secondary Outcomes

Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing

To determine the efficacy of tildacerfont on disease control or reduction of GC use in participants with classic CAH as measured by number of subjects who achieve a reduction in A4 or reduction in GC dosing during treatment period

Time frame: 12 weeks

Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4

To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4

Time frame: 4 weeks

Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization

To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4 or week 12

Time frame: 4 weeks or 12 weeks

Data from ClinicalTrials.gov

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