A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension

Status: Recruiting
Location: See all (6) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
SUMMARY

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 17
Healthy Volunteers: f
View:

⁃ Part A and B participants are eligible to be included in the study only if all of the following criteria apply:

• Male or female at birth, between 1 to \<18 years of chronological age at the time of signing the Informed Consent Form (ICF).

• Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency (21-OHD) based on standard medically accepted criteria such as elevated 17-OHP level, confirmed CYP21A2 genetic testing, positive newborn screening with confirmatory second tier testing, or cosyntropin stimulation.

• Participants must have an elevated morning serum A4 level \>ULN during Screening obtained prior to morning glucocorticoid (GC) administration.

• Participants must be on a stable supraphysiologic GC replacement therapy for at least one month prior to Screening.

• Compliance, as judged per Investigator discretion, with GC replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period.

• Normal thyroid stimulating hormone (TSH) and thyroxine (T4) within 3 months of Screening per age-appropriate range.

⁃ Part C inclusion criteria require participants to complete treatment in either Part A or Part B and in the Investigator's opinion it would benefit the participant to continue in Part C, regardless of age.

Locations
United States
Michigan
University of Michigan
RECRUITING
Ann Arbor
Texas
Cook Children's Health Care System
RECRUITING
Fort Worth
Virginia
University of Virginia Health System
RECRUITING
Charlottesville
Other Locations
Australia
Monash Health
NOT_YET_RECRUITING
Clayton
Queensland Children's Hospital
NOT_YET_RECRUITING
South Brisbane
Institute of Endocrinology of Diabetes, The Children's Hospital at Westmead
NOT_YET_RECRUITING
Westmead
Contact Information
Primary
Crinetics Clinical Trials
clinicaltrials@crinetics.com
833-827-9741
Time Frame
Start Date: 2025-11-30
Estimated Completion Date: 2030-03
Participants
Target number of participants: 153
Treatments
Experimental: Treatment (Part A)
Open-label, semi-sequential cohorts.
Experimental: Active Treatment (Part B)
Randomized, Parallel Arms, Double-Blind
Placebo_comparator: Placebo (Part B)
Randomized, Parallel Arms, Double-Blind
Experimental: Open-Label Treatment (Part C)
Open-label treatment period for participants entering Part C from Part A and B.
Sponsors
Leads: Crinetics Pharmaceuticals Inc.

This content was sourced from clinicaltrials.gov

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