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).
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
153
Atumelnant, tablets, once daily by mouth, weight-based dosing
Placebo, tablets, once daily by mouth, weight-based dosing
University of Michigan
Ann Arbor, Michigan, United States
RECRUITINGUniversity of Minnesota
Minneapolis, Minnesota, United States
Change from baseline in morning serum androstenedione (A4) (Part A)
Time frame: Week 8
Percent change from baseline in glucocorticoid (GC) daily dose while serum early morning A4 ≤Upper Limit of Normal (ULN) (Part B)
Time frame: Week 28
Change from baseline in serum early morning A4 over time (Part C)
Time frame: Up to Week 260
Change from baseline in morning serum 17-hydroxyprogesterone (17-OHP) (Part A)
Time frame: Week 8
Plasma and/or blood concentrations of atumelnant (Part A)
Time frame: Up to Week 8
Change from baseline in serum early morning A4 (Part B)
Time frame: Week 4
Change from baseline in serum early morning 17-OHP (Part B)
Time frame: Week 4
Proportion of participants with physiologic GC dose while serum early morning A4 <ULN (Part B)
Time frame: Week 28
Change from baseline in serum early morning 17-OHP over time (Part C)
Time frame: Up to Week 260
Percent change from baseline in GC daily dose over time (Part C)
Time frame: Up to Week 260
Proportion of participants with physiologic GC dose while serum early morning A4 <ULN over time (Part C)
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Cook Children's Health Care System
Fort Worth, Texas, United States
RECRUITINGUniversity of Virginia Health System
Charlottesville, Virginia, United States
RECRUITINGHospital Italiano de Buenos Aires
Buenos Aires, Argentina
RECRUITINGInstituto Médico Especializado (IME)
Buenos Aires, Argentina
RECRUITINGInstitute of Endocrinology of Diabetes, The Children's Hospital at Westmead
Westmead, New South Wales, Australia
NOT_YET_RECRUITINGQueensland Children's Hospital
South Brisbane, Queensland, Australia
NOT_YET_RECRUITINGMonash Health
Clayton, Victoria, Australia
NOT_YET_RECRUITINGHopital Robert Debre
Paris, France
RECRUITING...and 2 more locations
Time frame: Up to Week 260