A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

Официальное название

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

Обзор

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.

Вмешательства

• Препарат Lu AG13909
  Solution for infusion

Первичные конечные точки

• Parts A and B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [Срок оценки: Up to Day 161]
• Parts A and B: Number of Participants With Anti-Drug Antibodies (ADAs) [Срок оценки: Day 1 up to Day 161]
• Parts A and B: Cmax: Maximum Observed Serum Concentration of Lu AG13909 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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) [Срок оценки: Baseline up to Day 85]

Вторичные конечные точки (4)

• Part C: Lu AG13909 Serum Concentrations Following Multiple IV Doses [Срок оценки: Baseline up to Day 352]
• Part C: Change From Baseline of Lu AG13909 in Blood Concentrations of 17-OHP and A4 [Срок оценки: Baseline up to Day 169]
• Part C: Number of Participants With TEAEs [Срок оценки: Baseline up to Day 352]
• Part C: Number of Participants With ADAs [Срок оценки: Baseline up to Day 352]

Критерии участия

Критерии включения

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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Франция · 4 центра

• Chu Angers — Angers
• CHU de Lille — Lille
• GH Pitié-Salpêtrière — Paris
• CHRU Strasbourg — Strasbourg

Великобритания · 4 центра

• NIHR/Wellcome Trust Clinical Research Facility — Birmingham
• Cambridge Clinical Research Centre — Cambridge
• NIHR Clinical Research Facility — London
• University College London Hospital - NIHR — London

Италия · 2 центра

• Azienda Ospedaliero Universitaria di Bologna — Bologna
• Azienda Ospedaliero-Universitaria Policlinico Umberto I, Roma — Roma

Швеция · 2 центра

• Sahlgrenska University Hospital — Gothenburg
• Karolinska University Hospital — Stockholm

США · 1 центр

• University Hospital-University of Michigan — Ann Arbor

Дания · 1 центр

• Rigshospitalet — Copenhagen

Грузия · 1 центр

• David Metreveli Medical Centre, Tbilisi — Tbilisi

Ирландия · 1 центр

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

Польша · 1 центр

• Centrum Nowoczesnych Terapii, Dobry Lekarz — Dobry Lekarz

Идентификаторы

NCT: NCT05669950 · 19873A

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗