Study of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

Phase 1

Recruiting

• Conditions: Congenital Adrenal Hyperplasia
• Interventions: Drug: Lu AG13909

• Registration Number: NCT05669950
• Lead Sponsor: H. Lundbeck A/S

Brief Summary

This study 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 study 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

Study Timeline

• Study Start: 2022-12-19
• Study First Submitted: 2022-12-20
• Study First Submitted QC: 2022-12-20
• Study First Posted: 2023-01-03
• Status Verified: 2024-06
• Last Update Submitted: 2024-07-03
• Last Update Posted: 2024-07-05
• Primary Completion: 2024-12-28
• Study Completion: 2024-12-28

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• 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 ≤35 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.

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.

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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	Up to Day 161
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
Number of Participants With Anti-Drug Antibodies (ADAs)	Day 1 up to end of study (Day 161)
CL: Apparent Total Serum Clearance of Lu AG13909	0 (predose) up to 24 hours postdose on Day 1 to Day 161
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
Cmax: Maximum Observed Serum Concentration of Lu AG13909	0 (predose) up to 24 hours postdose on Day 1 to Day 161
Ctrough: Minimum Observed Serum Concentration of Lu AG13909	0 (predose) up to 24 hours postdose on Day 1 to Day 161
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
Tmax: Nominal Time Corresponding to the Occurrence of Cmax	0 (predose) up to 24 hours postdose on Day 1 to Day 161
AUC0-tau: Area under the curve over a dosing interval	0 (predose) up to 24 hours postdose on Day 1 to Day 161
t½: Apparent Elimination Half-life of Lu AG13909	0 (predose) up to 24 hours postdose on Day 1 to Day 161

Trial Locations

Locations (5)

University Hospitals Birmingham NHS Foundation Trust; 🇬🇧 Birmingham, United Kingdom

Karolinska University Hospital/ Huddinge; 🇸🇪 Huddinge, Sweden

Sahlgrenska University Hospital; 🇸🇪 Gothenburg, Sweden

University College London Hospital NHS Foundation Trust; 🇬🇧 London, United Kingdom

Guy's and St Thomas' NHS Foundation Trust; 🇬🇧 London, United Kingdom