A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
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
- Congenital Adrenal Hyperplasia
- Classic Congenital Adrenal Hyperplasia
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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
Recruiting Locations
Ann Arbor 4984247, Michigan 5001836 48109
Minneapolis 5037649, Minnesota 5037779 55454
Fort Worth 4691930, Texas 4736286 76104
Charlottesville 4752031, Virginia 6254928 22903
More Details
- Status
- Recruiting
- Sponsor
- Crinetics Pharmaceuticals Inc.
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.