A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
- Registration Number
- NCT05669950
- Lead Sponsor
- H. Lundbeck A/S
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 42
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Lu AG13909 Lu AG13909 Participants in Part A will receive multiple intravenous (IV) doses of Lu AG13909 per a prespecified dosing schedule. After data from Part A has shown that a pharmacologically relevant dose level is safe and tolerable, participants in Part B will then receive multiple IV doses of Lu AG13909 per a prespecified dosing schedule. After data from Part B has shown that a pharmacologically relevant dose level is safe and tolerable, participants in Part C will then receive multiple IV doses of Lu AG13909 per a prespecified dosing schedule. Participants from Part C may be eligible to continue in the optional Treatment Extension.
- Primary Outcome Measures
Name Time Method 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
- Secondary Outcome Measures
Name Time Method Part C: Lu AG13909 Serum Concentrations Following Multiple IV Doses Baseline up to Day 352 Part C: Change From Baseline of Lu AG13909 in Blood Concentrations of 17-OHP and A4 Baseline up to Day 169 Part C: Number of Participants With TEAEs Baseline up to Day 352 Part C: Number of Participants With ADAs Baseline up to Day 352
Trial Locations
- Locations (7)
Rigshospitalet
🇩🇰Copenhagen, Denmark
Karolinska University Hospital
🇸🇪Stockholm, Sweden
NIHR/Wellcome Trust Clinical Research Facility
🇬🇧Birmingham, United Kingdom
Cambridge Clinical Research Centre
🇬🇧Cambridge, United Kingdom
NIHR Clinical Research Facility
🇬🇧London, United Kingdom
University College London Hospital - NIHR
🇬🇧London, United Kingdom
Sahlgrenska University Hospital
🇸🇪Gothenburg, Sweden
Rigshospitalet🇩🇰Copenhagen, Denmark