A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

NCT05669950 | Recruiting Phase 1 FDA Drug

• 1 other identifier: 19873A
• Study Type: interventional
• Target: 42
• Locations: 9 countries
• Sites: 17

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.

Conditions

Congenital Adrenal Hyperplasia

Outcome Measures

Primary Outcomes (12)

• 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 Outcomes (4)

• 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

Study Arms (1)

Lu AG13909

EXPERIMENTAL

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

Interventions

Lu AG13909 DRUG

Solution for infusion

Lu AG13909

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• H. Lundbeck A/S: lead

Study Sites (17)

University Hospital-University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Rigshospitalet

Copenhagen, 2100, Denmark

RECRUITING

Chu Angers

Angers, 49933, France

RECRUITING

CHU de Lille

Lille, 59000, France

RECRUITING

GH Pitié-Salpêtrière

Paris, 75013, France

RECRUITING

CHRU Strasbourg

Strasbourg, 67091, France

RECRUITING

David Metreveli Medical Centre, Tbilisi

Tbilisi, 0144, Georgia

RECRUITING

Beaumont Hospital Royal College of Surgeons in Ireland (RCSI), Dublin

Dublin, D02 YN77, Ireland

RECRUITING

Azienda Ospedaliero Universitaria di Bologna

Bologna, 40138, Italy

RECRUITING

Azienda Ospedaliero-Universitaria Policlinico Umberto I, Roma

Roma, 00161, Italy

RECRUITING

Centrum Nowoczesnych Terapii, Dobry Lekarz

Dobry Lekarz, 60-324, Poland

RECRUITING

Sahlgrenska University Hospital

Gothenburg, 413 45, Sweden

RECRUITING

Karolinska University Hospital

Stockholm, 17174, Sweden

RECRUITING

NIHR/Wellcome Trust Clinical Research Facility

Birmingham, B15 2TH, United Kingdom

RECRUITING

Cambridge Clinical Research Centre

Cambridge, CB2 0SL, United Kingdom

RECRUITING

NIHR Clinical Research Facility

London, SE1 9RT, United Kingdom

RECRUITING

University College London Hospital - NIHR

London, W1T 7HA, United Kingdom

RECRUITING

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders

Study Officials

• Email contact via H. Lundbeck A/S

  HQ_Medinfo@Lundbeck.com

  STUDY DIRECTOR

Central Study Contacts

Email contact via H. Lundbeck A/S

CONTACT

+45 36301311; HQ_Medinfo@Lundbeck.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 20, 2022
• First Posted: January 3, 2023
• Study Start: December 19, 2022
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: March 9, 2026

Record last verified: 2026-03

Locations

GE (1); IE (1); PL (1); US (1); IT (2); DK (1); SE (2); GB (4); FR (4)