Summary

Eligibility
for people ages 2-17 (full criteria)
Location
at UCSF
Dates
study started
study ends around
Principal Investigator
by Maya Lodish (ucsf)
Headshot of Maya Lodish
Maya Lodish

Description

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).

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

Keywords

Congenital Adrenal Hyperplasia, classic congenital adrenal hyperplasia (CAH), 21-hydroxylase deficiency, Congenital adrenal hyperplasia due to 21 hydroxylase deficiency, crinecerfont

Eligibility

You can join if…

Open to people ages 2-17

  • 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.

You CAN'T join if...

  • 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.

Locations

  • Neurocrine Clinical Site
    San Francisco California 94158 United States
  • Neurocrine Clinical Site
    San Diego California 92123 United States
  • Neurocrine Clinical Site
    Los Angeles California 90027 United States
  • Neurocrine Clinical Site
    Orange California 92868 United States

Lead Scientist at University of California Health

  • Maya Lodish (ucsf)
    Maya Lodish, MD, MHSc, Selna L. Kaplan Chair, Distinguished Professorship in Pediatric Endocrinology/Diabetes, Professor of Pediatrics, Division Chief of Pediatric Endocrinology Dr. Lodish completed her undergraduate degree at Dartmouth College, Hanover NH and she went on to Yale University School of Medicine.

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Neurocrine Biosciences
ID
NCT04806451
Phase
Phase 3 Congenital Adrenal Hyperplasia Research Study
Study Type
Interventional
Participants
About 103 people participating
Last Updated