Clinical trial NCT04806451: Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Congenital Adrenal Hyperplasia
Keywords: classic congenital adrenal hyperplasia (CAH)
21-hydroxylase deficiency
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
First posted: 2021-03-19
Enrollment: 103
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).


Eligibility criteria: Inclusion Criteria:

* Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.
* Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.
* Be on a stable steroid regimen.
* Have elevated androgen levels.
* Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.

Exclusion Criteria:

* Have a diagnosis of any of the other forms of classic CAH.
* Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
* Have a clinically significant unstable medical condition or chronic disease other than CAH.
* Have a history of cancer unless considered to be cured.
* Have a known history of clinically significant arrhythmia or abnormalities on electrocardiogram (ECG).
* Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.
* Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
* Have current substance dependence or substance (drug) or alcohol abuse.
* Have had a significant blood loss or donated blood or blood products within 8 weeks prior to the study.

Primary outcomes:
measure: Change From Baseline in Serum Androstenedione at Week 4
timeFrame: Baseline, Week 4
Secondary outcomes:
measure: Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
timeFrame: Baseline, Week 4
measure: Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28
timeFrame: Baseline, Week 28
measure: Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28
timeFrame: Week 28
measure: Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28
timeFrame: Baseline, Week 28
measure: Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28
timeFrame: Baseline, Week 28
measure: Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28
timeFrame: Baseline, Week 28
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Neurocrine Clinical Site
state: Alabama
city: Los Angeles
country: United States
facility: Neurocrine Clinical Site
state: California
city: Orange
country: United States
facility: Neurocrine Clinical Site
state: California
city: San Diego
country: United States
facility: Neurocrine Clinical Site
state: California
city: San Francisco
country: United States
facility: Neurocrine Clinical Site
state: California
city: Aurora
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Hartford
country: United States
facility: Neurocrine Clinical Site
state: Connecticut
city: Washington
country: United States
facility: Neurocrine Clinical Site
state: District of Columbia
city: Atlanta
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Indianapolis
country: United States
facility: Neurocrine Clinical Site
state: Indiana
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Minneapolis
country: United States
facility: Neurocrine Clinical Site
state: Minnesota
city: Saint Louis
country: United States
facility: Neurocrine Clinical site
state: Missouri
city: New Hyde Park
country: United States
facility: Neurocrine Clinical Site
state: New York
city: New York
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Oklahoma City
country: United States
facility: Neurocrine Clinical Site
state: Oklahoma
city: Tulsa
country: United States
facility: Neurocrine Clinical Site
state: Oklahoma
city: Philadelphia
country: United States
facility: Neurocrine Clinical Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Neurocrine Clinical Site
state: Pennsylvania
city: Dallas
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Seattle
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Brussels
country: Belgium
facility: Neurocrine Clinical Site
city: Ghent
country: Belgium
facility: Neurocrine Clinical Site
city: Edmonton
country: Canada
facility: Neurocrine Clinical Site
state: Alberta
city: Vancouver
country: Canada
facility: Neurocrine Clinical Site
state: British Columbia
city: Montréal
country: Canada
facility: Neurocrine Clinical Site
state: Quebec
city: Angers
country: France
facility: Neurocrine Clinical Site
city: Bordeau
country: France
facility: Neurocrine Clinical Site
city: Le Kremlin-Bicêtre
country: France
facility: Neurocrine Clinical Site
city: Paris
country: France
facility: Neurocrine Clinical Site
city: Paris
country: France
facility: Neurocrine Clinical Site
city: Berlin
country: Germany
facility: Neurocrine Clinical Site
city: Heidelberg
country: Germany
facility: Neurocrine Clinical Site
city: Magdeburg
country: Germany
facility: Neurocrine Clinical Site
city: Athens
country: Greece
facility: Neurocrine Clinical Site
city: Athens
country: Greece
facility: Neurocrine Clinical Site
city: Bologna
country: Italy
facility: Neurocrine Clinical Site
city: Milan
country: Italy
facility: Neurocrine Clinical Site
city: Napoli
country: Italy
facility: Neurocrine Clinical Site
city: Roma
country: Italy
facility: Neurocrine Clinical Site
city: Gdańsk
country: Poland
facility: Neurocrine Clinical Site
city: Rzeszów
country: Poland
facility: Neurocrine Clinical Site
city: Barcelona
country: Spain
facility: Neurocrine Clinical Site
city: Sevilla
country: Spain
facility: Neurocrine Clinical Site
city: London
country: United Kingdom
facility: Neurocrine Clinical Site
Study officials:
affiliation: Neurocrine Biosciences
name: Clinical Development Lead
role: STUDY_DIRECTOR
References: