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

Congenital Adrenal Hyperplasia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05128942
• Other study ID #: SPR001-205
• Status: Recruiting
• Phase: Phase 2
• Start date: December 10, 2021
• Est. completion date: December 2024

Study information

• Verified date: June 2024
• Source: Spruce Biosciences
• Contact: Clinical Trials
• Phone: 415-655-4169
• Email: CAHptain[@]sprucebiosciences.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 55
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Drug:
• Tildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.

Locations

Country	Name	City	State
United States	Spruce Study Site	Buffalo	New York
United States	Spruce Study Site	Charlottesville	Virginia
United States	Spruce Study Site	Chicago	Illinois
United States	Spruce Study Site	Columbia	South Carolina
United States	Spruce Study Site	Fort Worth	Texas
United States	Spruce Study Site	Indianapolis	Indiana
United States	Spruce Study Site	Minneapolis	Minnesota
United States	Spruce Study Site	Plano	Texas
United States	Spruce Study Site	Richmond	Virginia
United States	Spruce Study Site	Sacramento	California
United States	Spruce Study Site	Salt Lake City	Utah
United States	Spruce Study Site	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Spruce Biosciences

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	12 weeks
Secondary	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	4 weeks or 12 weeks
Secondary	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	4 weeks
Secondary	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	4 weeks or 12 weeks
Secondary	Tildacerfont pharmacokinetics (PK)	To determine the consistency of preliminary tildacerfont PK in participants with those simulated in a PBPK model measured by comparing tildacerfont plasma concentrations with current PBPK simulation for consistency	4 weeks or 12 weeks