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NCT Number: NCT05669950

A Trial 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.

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Key information

Conditions

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

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Rigshospitalet, Copenhagen, Denmark

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Lu AG13909

Drug

Solution for infusion

Primary outcomes

  1. Parts A and B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Time frame: Up to Day 161

  2. Parts A and B: Number of Participants With Anti-Drug Antibodies (ADAs)

    Time frame: Day 1 up to Day 161

  3. Parts A and B: Cmax: Maximum Observed Serum Concentration of Lu AG13909

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  4. Parts A and B: Tmax: Nominal Time Corresponding to the Occurrence of Cmax

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  5. Parts A and B: Ctrough: Minimum Observed Serum Concentration of Lu AG13909

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  6. Parts A and B: t½: Apparent Elimination Half-life of Lu AG13909

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  7. Parts A and B: AUC0-infinity: Area under the plasma concentration curve of x from zero to infinity of Lu AG13909

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  8. Parts A and B: CL: Apparent Total Serum Clearance of Lu AG13909

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  9. Parts A and B: Vz: Volume of Distribution During the Terminal Elimination Phase After IV Administration of Lu AG13909

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  10. Parts A and B: Change From Baseline After Each Dose of Lu AG13909 in Blood Concentrations of 17-hydroxyprogesterone (17-OHP) and Androstenedione (A4)

    Time frame: Baseline up to Day 85

  11. Parts A and B: AUC0-tau: Area under the curve over a dosing interval

    Time frame: 0 (predose) up to 24 hours postdose on Day 1 to Day 161

  12. Part C: Morning Concentration of A4 in Blood <Upper Limit of Normal (ULN)

    Time frame: Day 169

Secondary outcomes

  1. Part C: Lu AG13909 Serum Concentrations Following Multiple IV Doses

    Time frame: Baseline up to Day 352

  2. Part C: Change From Baseline of Lu AG13909 in Blood Concentrations of 17-OHP and A4

    Time frame: Baseline up to Day 169

  3. Part C: Number of Participants With TEAEs

    Time frame: Baseline up to Day 352

  4. Part C: Number of Participants With ADAs

    Time frame: Baseline up to Day 352

Study contacts

Contact information is provided by the study sponsor or research team.

Email contact via H. Lundbeck A/S

CONTACT

[email protected]

+45 36301311

Sponsors and collaborators

Lead sponsor

H. Lundbeck A/S

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2026
Study completion
2027
First posted
Jan 3, 2023
Registry last updated
Mar 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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