This trial will evaluate the effects of different doses of Lu AG13909 in adult participants with congenital adrenal hyperplasia, also called CAH. CAH is a rare genetic disorder that affects a person's ability to produce certain hormones. The main goals of this trial are to learn about the safety and tolerability of Lu AG13909, how Lu AG13909 behaves in the body, and how the body responds to Lu AG13909.
Eligibility
Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Parts A and B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — Up to Day 161
Parts A and B: Number of Participants With Anti-Drug Antibodies (ADAs) — Day 1 up to Day 161
Parts A and B: Cmax: Maximum Observed Serum Concentration of Lu AG13909 — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Parts A and B: Tmax: Nominal Time Corresponding to the Occurrence of Cmax — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Parts A and B: Ctrough: Minimum Observed Serum Concentration of Lu AG13909 — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Parts A and B: t½: Apparent Elimination Half-life of Lu AG13909 — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Parts A and B: AUC0-infinity: Area under the plasma concentration curve of x from zero to infinity of Lu AG13909 — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Parts A and B: CL: Apparent Total Serum Clearance of Lu AG13909 — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Parts A and B: Vz: Volume of Distribution During the Terminal Elimination Phase After IV Administration of Lu AG13909 — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Parts A and B: Change From Baseline After Each Dose of Lu AG13909 in Blood Concentrations of 17-hydroxyprogesterone (17-OHP) and Androstenedione (A4) — Baseline up to Day 85
Parts A and B: AUC0-tau: Area under the curve over a dosing interval — 0 (predose) up to 24 hours postdose on Day 1 to Day 161
Part C: Morning Concentration of A4 in Blood <Upper Limit of Normal (ULN) — Day 169
Trial sites (17)
Facility
City
Region
Status
University Hospital-University of Michigan
Ann Arbor
Michigan
Recruiting
Rigshospitalet
Copenhagen
Denmark
Recruiting
Chu Angers
Angers
France
Recruiting
CHU de Lille
Lille
France
Recruiting
GH Pitié-Salpêtrière
Paris
France
Recruiting
CHRU Strasbourg
Strasbourg
France
Recruiting
David Metreveli Medical Centre, Tbilisi
Tbilisi
Georgia
Recruiting
Beaumont Hospital Royal College of Surgeons in Ireland (RCSI), Dublin
Dublin
Ireland
Recruiting
Azienda Ospedaliero Universitaria di Bologna
Bologna
Italy
Recruiting
Azienda Ospedaliero-Universitaria Policlinico Umberto I, Roma
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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