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Recruiting NCT05669950

A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia

Phase I / Phase II Interventional Congenital Adrenal Hyperplasia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Lu AG13909.
Who it may be relevant to
Registry conditions: Congenital Adrenal Hyperplasia. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Denmark, France, Georgia, Ireland +4
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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.

Interventions

  • Drug Lu AG13909
    Solution for infusion

Primary outcome measures

  • Parts A and B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [Time frame: Up to Day 161]
  • Parts A and B: Number of Participants With Anti-Drug Antibodies (ADAs) [Time frame: Day 1 up to Day 161]
  • 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]
  • 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]
  • 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]
  • 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]
  • 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]
  • 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]
  • 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]
  • 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]
Secondary outcome measures (4)
  • Part C: Lu AG13909 Serum Concentrations Following Multiple IV Doses [Time frame: Baseline up to Day 352]
  • Part C: Change From Baseline of Lu AG13909 in Blood Concentrations of 17-OHP and A4 [Time frame: Baseline up to Day 169]
  • Part C: Number of Participants With TEAEs [Time frame: Baseline up to Day 352]
  • Part C: Number of Participants With ADAs [Time frame: Baseline up to Day 352]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

France · 4 centers
  • Chu Angers — Angers
  • CHU de Lille — Lille
  • GH Pitié-Salpêtrière — Paris
  • CHRU Strasbourg — Strasbourg
United Kingdom · 4 centers
  • NIHR/Wellcome Trust Clinical Research Facility — Birmingham
  • Cambridge Clinical Research Centre — Cambridge
  • NIHR Clinical Research Facility — London
  • University College London Hospital - NIHR — London
Italy · 2 centers
  • Azienda Ospedaliero Universitaria di Bologna — Bologna
  • Azienda Ospedaliero-Universitaria Policlinico Umberto I, Roma — Roma
Sweden · 2 centers
  • Sahlgrenska University Hospital — Gothenburg
  • Karolinska University Hospital — Stockholm
United States · 1 center
  • University Hospital-University of Michigan — Ann Arbor
Denmark · 1 center
  • Rigshospitalet — Copenhagen
Georgia · 1 center
  • David Metreveli Medical Centre, Tbilisi — Tbilisi
Ireland · 1 center
  • Beaumont Hospital Royal College of Surgeons in Ireland (RCSI), Dublin — Dublin
Poland · 1 center
  • Centrum Nowoczesnych Terapii, Dobry Lekarz — Dobry Lekarz

Identifiers

NCT: NCT05669950 · 19873A

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗