Clinical trial NCT05128942: A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children with CAH
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Congenital Adrenal Hyperplasia
21-OHD
Keywords: Pediatric
CAH
Adrenal Disorder
Congenital Adrenal Hyperplasia
Lead sponsor: Spruce Biosciences
Sponsor class: INDUSTRY
First posted: 2021-11-22
Enrollment: 65
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Subjects will be assigned to treatment cohorts by age.
Primary purpose: TREATMENT

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

Detailed 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.

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

Primary outcomes:
measure: Number of participants with Treatment-emergent adverse event (TEAE) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
timeFrame: 12 weeks
Secondary outcomes:
measure: Proportion of participants who achieve a reduction in androstenedione (A4) or reduction in glucocorticoid (GC) dosing
timeFrame: 4 weeks or 12 weeks
measure: Proportion of participants with elevated baseline A4 who achieve a reduction in A4
timeFrame: 4 weeks
measure: Proportion of participants with elevated baseline A4 who achieve a reduction in A4 who achieve A4 normalization
timeFrame: 4 weeks or 12 weeks
measure: Tildacerfont pharmacokinetics (PK)
timeFrame: 4 weeks or 12 weeks
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: Spruce Study Site
state: California
city: San Diego
country: United States
facility: Spruce Study Site
state: California
city: Chicago
country: United States
facility: Spruce Study Site
state: Illinois
city: Minneapolis
country: United States
facility: Spruce Study Site
state: Minnesota
city: Buffalo
country: United States
facility: Spruce Study Site
state: New York
city: Providence
country: United States
facility: Spruce Study Site
state: Rhode Island
city: Columbia
country: United States
facility: Spruce Study Site
state: South Carolina
city: Dallas
country: United States
facility: Spruce Study Site
state: Texas
city: Edinburg
country: United States
facility: Spruce Study Site
state: Texas
city: Fort Worth
country: United States
facility: Spruce Study Site
state: Texas
city: Salt Lake City
country: United States
facility: Spruce Study Site
state: Utah
city: Charlottesville
country: United States
facility: Spruce Study Site
state: Virginia
city: Richmond
country: United States
facility: Spruce Study Site
state: Virginia
Study officials:
affiliation: Spruce Biosciences
name: Will Charlton, MD
role: STUDY_DIRECTOR
References: