Purpose

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).

Conditions

Eligibility

Eligible Ages
Between 1 Year and 17 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

Part A and B participants are eligible to be included in the study only if all of the following criteria apply: 1. Male or female at birth, between 1 to <18 years of chronological age at the time of signing the Informed Consent Form (ICF). 2. 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. 3. Participants must have an elevated morning serum A4 level >ULN during Screening obtained prior to morning glucocorticoid (GC) administration. 4. Participants must be on a stable supraphysiologic GC replacement therapy for at least one month prior to Screening. 5. Compliance, as judged per Investigator discretion, with GC replacement and mineralocorticoid replacement (if applicable) regimen documented during the Screening Period. 6. 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.

Exclusion Criteria

Part A and Part B: Individuals in Part A and Part B who meet any of the following criteria will be excluded from participation in this study: 1. Diagnosis of any form of CAH other than classic 21-OHD. 2. Participants treated with other GCs within 30 days of Screening. 3. Stress dose of GC therapy within 2 weeks of start of Screening, defined as any dose above the normal maintenance dose, including but not limited to intravenous (IV) or intramuscular (IM) hydrocortisone. 4. Use of growth hormones within 1 week of start of Screening for short acting, or within 6 weeks of start of Screening for long acting. 5. Use of a corticotropin-releasing factor receptor antagonist within 14 days of Screening. 6. History of cancer excluding cured/treated dermal squamous or basal cell carcinoma or cervical carcinoma in situ. 7. Abnormal sleep/wake cycles (as determined by the Investigator). 8. Female participants who are pregnant or lactating. 9. Participants who have been dosed with an investigational drug (other than atumelnant) in any prior clinical study within 60 days or 5 half-lives (whichever is longer) prior to the first dose. Part C: 10. Individuals in Part C who do not meet the Part C Inclusion Criteria.

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Treatment (Part A)
Open-label, semi-sequential cohorts.
  • Drug: Atumelnant
    Atumelnant, tablets, once daily by mouth, weight-based dosing
    Other names:
    • CRN04894
Experimental
Active Treatment (Part B)
Randomized, Parallel Arms, Double-Blind
  • Drug: Atumelnant
    Atumelnant, tablets, once daily by mouth, weight-based dosing
    Other names:
    • CRN04894
Placebo Comparator
Placebo (Part B)
Randomized, Parallel Arms, Double-Blind
  • Drug: Placebo
    Placebo, tablets, once daily by mouth, weight-based dosing
Experimental
Open-Label Treatment (Part C)
Open-label treatment period for participants entering Part C from Part A and B.
  • Drug: Atumelnant
    Atumelnant, tablets, once daily by mouth, weight-based dosing
    Other names:
    • CRN04894

Recruiting Locations

University of Michigan
Ann Arbor 4984247, Michigan 5001836 48109
Contact:
Thiara Blanco Varela
734-647-5661
tbv@med.umich.edu

University of Minnesota
Minneapolis 5037649, Minnesota 5037779 55454
Contact:
Elizabeth Tepozteco
tepoz001@umn.edu

Cook Children's Health Care System
Fort Worth 4691930, Texas 4736286 76104
Contact:
Tiffany Skrodzki
682-885-1951
tiffany.skrodzki@cookchildrens.org

University of Virginia Health System
Charlottesville 4752031, Virginia 6254928 22903
Contact:
Gabrielle Laskey
(434) 243-8236
gal5u@uvahealth.org

More Details

Status
Recruiting
Sponsor
Crinetics Pharmaceuticals Inc.

Study Contact

Crinetics Clinical Trials
833-827-9741
clinicaltrials@crinetics.com

Detailed Description

This Phase 2/3 plus open-label extension study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic CAH. Part A is a Phase 2, open-label, semi-sequential cohorts portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is the open-label extension (OLE) portion of the study. Participants in Part A and B are eligible to enroll in Part C (OLE). A total of approximately 153 participants may be enrolled in the study (planned and optional cohorts) ages 1 to < 18 years old. The first 3 cohorts in Part A are for ages 12 to <18 years and will be sequential, and Safety Review Committee (SRC) review of data and approval to proceed is required prior to enrolling each subsequent cohort. The fourth cohort in Part A is for ages 1 to 11 years old and will begin after Cohorts 1 and 2 have been completed, additional requirements are fulfilled, and following SRC review of Cohorts 1 and 2 data.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.