Health TrialsbyHealthPanel

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

ConditionsCongenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age1-17
SponsorCrinetics Pharmaceuticals Inc.

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

Diagnosis of any form of CAH other than classic 21-OHD.

Participants treated with other GCs within 30 days of Screening.

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.

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • Atumelnant

    Drug

    Atumelnant, tablets, once daily by mouth, weight-based dosing

  • Placebo

    Drug

    Placebo, tablets, once daily by mouth, weight-based dosing

Treatment groups

153 Participants
are divided into 4 treatment groups
Group A: Treatment (Part A)Experimental treatment 1 intervention
Group B: Active Treatment (Part B)Experimental treatment 1 intervention
Group C: Placebo (Part B)Placebo comparator 1 intervention
Group D: Open-Label Treatment (Part C)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Change from baseline in morning serum androstenedione (A4) (Part A)

Time frame
Week 8
2

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
3

Change from baseline in serum early morning A4 over time (Part C)

Time frame
Up to Week 260

Secondary outcomes

1

Change from baseline in morning serum 17-hydroxyprogesterone (17-OHP) (Part A)

Time frame
Week 8
2

Plasma and/or blood concentrations of atumelnant (Part A)

Time frame
Up to Week 8
3

Change from baseline in serum early morning A4 (Part B)

Time frame
Week 4
4

Change from baseline in serum early morning 17-OHP (Part B)

Time frame
Week 4

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Crinetics Pharmaceuticals Inc.

Lead sponsor

Source ClinicalTrials.gov
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