Study of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

A Multi-site, Open-label, Sequential-group, Multiple-ascending-dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Lu AG13909 in Patients With Congenital Adrenal Hyperplasia

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.

Study Overview

• Status: Recruiting
• Conditions: Congenital Adrenal Hyperplasia
• Intervention / Treatment: Drug: Lu AG13909

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Email contact via H. Lundbeck A/S; Phone Number: +45 36301311; Email: LundbeckClinicalTrials@Lundbeck.com

Study Locations

• United Kingdom
  • London, United Kingdom, SE1 7EH
    • Recruiting
    • Guy's and St Thomas' NHS Foundation Trust
  • London, United Kingdom, NW1 2PG
    • Recruiting
    • University College London Hospital NHS Foundation Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: Lu AG13909; Solution for infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	Up to Day 161
Number of Participants With Anti-Drug Antibodies (ADAs)	Day 1 up to end of study (Day 161)
Cmax: Maximum Observed Serum Concentration 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
Ctrough: Minimum Observed Serum Concentration of Lu AG13909	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
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
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
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
AUC0-tau: Area under the curve over a dosing interval	0 (predose) up to 24 hours postdose on Day 1 to Day 161

Collaborators and Investigators

• Sponsor: H. Lundbeck A/S

Study record dates

Study Major Dates

• Study Start (Actual): December 19, 2022
• Primary Completion (Estimated): December 28, 2024
• Study Completion (Estimated): December 28, 2024

Study Registration Dates

• First Submitted: December 20, 2022
• First Submitted That Met QC Criteria: December 20, 2022
• First Posted (Actual): January 3, 2023

Study Record Updates

• Last Update Posted (Actual): June 15, 2023
• Last Update Submitted That Met QC Criteria: June 13, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Endocrine System Diseases; Gonadal Disorders; Disorders of Sex Development; Urogenital Abnormalities; Congenital Abnormalities; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Adrenal Gland Diseases; Steroid Metabolism, Inborn Errors; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Hyperplasia; Adrenal Hyperplasia, Congenital; Adrenogenital Syndrome; Adrenocortical Hyperfunction
• Other Study ID Numbers: 19873A

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No