A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

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

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.

Study Overview

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

• Study Type: Interventional
• Enrollment (Estimated): 42
• Phase: Phase 2; Phase 1

Contacts and Locations

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

Study Locations

• Denmark
  • Copenhagen, Denmark, 2100
    • Recruiting
    • Rigshospitalet
• France
  • Angers, France, 49933
    • Recruiting
    • CHU Angers
  • Lille, France, 59000
    • Recruiting
    • CHU de Lille
  • Paris, France, 75013
    • Recruiting
    • GH Pitié-Salpêtrière
  • Strasbourg, France, 67091
    • Recruiting
    • CHRU Strasbourg
• Georgia
  • Tbilisi, Georgia, 0144
    • Recruiting
    • David Metreveli Medical Centre, Tbilisi
• Ireland
  • Dublin, Ireland, D02 YN77
    • Recruiting
    • Beaumont Hospital Royal College of Surgeons in Ireland (RCSI), Dublin
• Italy
  • Bologna, Italy, 40138
    • Recruiting
    • Azienda Ospedaliero Universitaria di Bologna
  • Roma, Italy, 00161
    • Recruiting
    • Azienda Ospedaliero-Universitaria Policlinico Umberto I, Roma
• Poland
  • Dobry Lekarz, Poland, 60-324
    • Recruiting
    • Centrum Nowoczesnych Terapii, Dobry Lekarz
• Sweden
  • Gothenburg, Sweden, 413 45
    • Recruiting
    • Sahlgrenska University Hospital
  • Stockholm, Sweden, 17174
    • Recruiting
    • Karolinska University Hospital
• United Kingdom
  • Birmingham, United Kingdom, B15 2TH
    • Recruiting
    • NIHR/Wellcome Trust Clinical Research Facility
  • Cambridge, United Kingdom, CB2 0SL
    • Recruiting
    • Cambridge Clinical Research Centre
  • London, United Kingdom, SE1 9RT
    • Recruiting
    • NIHR Clinical Research Facility
  • London, United Kingdom, W1T 7HA
    • Recruiting
    • University College London Hospital - NIHR
• United States
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Recruiting
      • University Hospital-University of Michigan

Participation Criteria

Eligibility Criteria

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

Description

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 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. 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.

Drug: Lu AG13909; Solution for infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: H. Lundbeck A/S
• Investigators: Study Director: Email contact via H. Lundbeck A/S, HQ_Medinfo@Lundbeck.com

Study record dates

Study Major Dates

• Study Start (Actual): December 19, 2022
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): December 31, 2027

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): March 9, 2026
• Last Update Submitted That Met QC Criteria: March 6, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

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

Drug and device information, study documents

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