A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

Launched by H. LUNDBECK A/S · Dec 20, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Lu AG13909 in adults who have a condition known as congenital adrenal hyperplasia (CAH). CAH is a rare genetic disorder that affects how the body produces important hormones. The main goals of this study are to check if Lu AG13909 is safe to use, how well it is tolerated by the body, and how the body processes this medication. Participants will receive different doses of Lu AG13909, and researchers will closely monitor their health and responses.

To be eligible for this trial, participants must be adults aged 18 to 65 who have been diagnosed with a specific type of CAH (21-hydroxylase deficiency). They should have stable hormone replacement therapy for at least one month before joining the study and be generally healthy apart from their CAH. It’s important to note that pregnant or breastfeeding individuals cannot participate, and those with certain health conditions may also be excluded. Participants can expect regular check-ups and tests throughout the study to ensure their safety and to gather information about how the medication works.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• Parts A and B:
• • Confirmed diagnosis of 21-hydroxylase deficiency CAH (based on a pathogenic CYP21A2 variant and/or elevated 17-OHP).
• • Morning (pre-glucocorticoid \[GC\] replacement dose) blood concentrations of 17-OHP \>4-times upper limit of normal (ULN).
• • Body mass index (BMI) ≥18.5 kilograms (kg)/square meter (m\^2) (minimum 50 kg) and ≤40 kg/m\^2.
• • Stable GC replacement therapy for ≥1 month prior to the Screening Visit.
• • For the salt-wasting form of CAH, the participant must have been on a stable dose of mineralocorticoid replacement for ≥3 months prior to the Screening Visit.
• • Apart from CAH, the participant is generally healthy in the opinion of the investigator and based on medical history, physical examination, vital signs, ECGs, and the results of the safety laboratory tests.
• Part C:
• • Confirmed diagnosis of 21-hydroxylase deficiency CAH (based on a pathogenic CYP21A2 variant and/or elevated 17-OHP).
• • For Cohort C1 only: Morning (pre-GC replacement dose) blood concentrations of androgens (A4) \> ULN for age and sex.
• • For Cohort C2 only: Morning (pre-GC replacement dose) blood concentrations of androgens (A4) ≤ ULN for age and sex and the participant is treated with high doses of GC.
• • Stable GC replacement therapy for ≥1 month prior to the Screening Visit.
• • For the salt-wasting form of CAH, the participant must have been on a stable dose of mineralocorticoid replacement for ≥1 month prior to the Screening Visit.
• Exclusion Criteria:
• • The participant is pregnant or breastfeeding.
• • The participant has a clinically significant abnormal laboratory value, electrocardiogram (ECG) parameter, or vital signs value, or other safety findings at the Screening Visit that indicate a potential risk for the participant if enrolled, in the opinion of the investigator.
• • The participant has a history of known hypersensitivity or intolerance to Lu AG13909 or its excipients.
• Part C Only:
• • The participant has received at least one dose of Lu AG13909 in Part A or Part B.
• • Other inclusion and exclusion criteria may apply.