Summary

Eligibility
for people ages 3 months to 47 months (full criteria)
Location
at UCSF
Dates
study started
study ends around
Principal Investigator
by Ayca Erkin-Cakmak (ucsf)
Headshot of Ayca Erkin-Cakmak
Ayca Erkin-Cakmak

Description

Summary

The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.

Official Title

A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Crinecerfont in Pediatric Participants 3 Months to <4 Years of Age With Classic Congenital Adrenal Hyperplasia

Keywords

Congenital Adrenal Hyperplasia, CAH, Crinecerfont, Pediatric

Eligibility

You can join if…

Open to people ages 3 months to 47 months

  • Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD)
  • Have received at least 3 months of hydrocortisone treatment before Day 1, with a stable dose for at least 4 weeks before screening.
  • Have a body weight of at least 4.5 kilograms (kg) at screening.
  • Have a newborn screen that is otherwise normal except for elevated 17-OHP or any other abnormality on newborn screen that was cleared upon evaluation by a pediatric specialist.

You CAN'T join if...

  • Have a known or suspected diagnosis of any of the other forms of classic CAH.
  • Have any condition besides CAH that requires chronic daily therapy with orally administered steroids.
  • Have any other clinically significant medical condition or chronic disease.

Note: Other protocol-defined inclusion and exclusion criteria may apply.

Locations

  • Neurocrine Clinical Site accepting new patients
    San Francisco California 94143 United States
  • Neurocrine Clinical Site accepting new patients
    Seattle Washington 98101 United States

Lead Scientist at University of California Health

  • Ayca Erkin-Cakmak (ucsf)
    Dr. Ayca Erkin-Cakmak is a pediatric endocrinologist who cares for children with conditions related to hormonal issues.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Neurocrine Switzerland GmbH
ID
NCT07536269
Phase
Phase 2 Congenital Adrenal Hyperplasia Research Study
Study Type
Interventional
Participants
Expecting 20 study participants
Last Updated