Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Congenital Adrenal Hyperplasia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04806451
• Other study ID #: NBI-74788-CAH2006
• Secondary ID: 2020-004381-19
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: June 25, 2021
• Est. completion date: August 2027

Study information

• Verified date: May 2024
• Source: Neurocrine Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 103
• Est. completion date: August 2027
• Est. primary completion date: March 10, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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 21-hydroxylase deficiency CAH.

• Be on a stable regimen of steroidal treatment for CAH.

• 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 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.

Related Conditions & MeSH terms

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

Intervention

Drug:
• Crinecerfont
CRF1-receptor antagonist
• Placebo
Non-active dosage form

Locations

Country	Name	City	State
Belgium	Neurocrine Clinical Site	Brussels
Belgium	Neurocrine Clinical Site	Ghent
Canada	Neurocrine Clinical Site	Edmonton	Alberta
Canada	Neurocrine Clinical Site	Montréal	Quebec
Canada	Neurocrine Clinical Site	Vancouver	British Columbia
France	Neurocrine Clinical Site	Angers
France	Neurocrine Clinical Site	Bordeau
France	Neurocrine Clinical Site	Le Kremlin-Bicêtre
France	Neurocrine Clinical Site	Paris
France	Neurocrine Clinical Site	Paris
Germany	Neurocrine Clinical Site	Berlin
Germany	Neurocrine Clinical Site	Heidelberg
Germany	Neurocrine Clinical Site	Magdeburg
Greece	Neurocrine Clinical Site	Athens
Greece	Neurocrine Clinical Site	Athens
Italy	Neurocrine Clinical Site	Bologna
Italy	Neurocrine Clinical Site	Milan
Italy	Neurocrine Clinical Site	Napoli
Italy	Neurocrine Clinical Site	Roma
Poland	Neurocrine Clinical Site	Gdansk
Poland	Neurocrine Clinical Site	Rzeszów
Spain	Neurocrine Clinical Site	Barcelona
Spain	Neurocrine Clinical Site	Sevilla
United Kingdom	Neurocrine Clinical Site	London
United States	Neurocrine Clinical Site	Ann Arbor	Michigan
United States	Neurocrine Clinical Site	Atlanta	Georgia
United States	Neurocrine Clinical Site	Aurora	Colorado
United States	Neurocrine Clinical Site	Birmingham	Alabama
United States	Neurocrine Clinical Site	Boston	Massachusetts
United States	Neurocrine Clinical Site	Dallas	Texas
United States	Neurocrine Clinical Site	Hartford	Connecticut
United States	Neurocrine Clinical Site	Indianapolis	Indiana
United States	Neurocrine Clinical Site	Los Angeles	California
United States	Neurocrine Clinical Site	Minneapolis	Minnesota
United States	Neurocrine Clinical Site	New Hyde Park	New York
United States	Neurocrine Clinical Site	New York	New York
United States	Neurocrine Clinical Site	Oklahoma City	Oklahoma
United States	Neurocrine Clinical Site	Orange	California
United States	Neurocrine Clinical Site	Philadelphia	Pennsylvania
United States	Neurocrine Clinical Site	Pittsburgh	Pennsylvania
United States	Neurocrine Clinical site	Saint Louis	Missouri
United States	Neurocrine Clinical Site	San Diego	California
United States	Neurocrine Clinical Site	San Francisco	California
United States	Neurocrine Clinical Site	Seattle	Washington
United States	Neurocrine Clinical Site	Tulsa	Oklahoma
United States	Neurocrine Clinical Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Neurocrine Biosciences

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Germany,  Greece,  Italy,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in Serum Androstenedione (A4) at Week 4		Baseline to Week 4
Secondary	Change from Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4		Baseline to Week 4
Secondary	Percent Change from Baseline in Glucocorticoid Daily Dose at Week 28		Baseline to Week 28
Secondary	Achievement of a reduction in glucocorticoid daily dose to physiologic levels at Week 28		Baseline to Week 28
Secondary	Change from baseline in body mass index at Week 28		Baseline to Week 28
Secondary	Change from baseline in salivary 17-OHP at Week 28		Baseline to Week 28
Secondary	Change in bone age advancement at Week 28		Baseline to Week 28
Secondary	Change from baseline in predicted adult height at Week 52		Baseline to Week 52