Congenital Adrenal HyperplasiaPhase 1/2NCT05669950
A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
A Phase 1/2 study testing Lu AG13909 in people with Congenital Adrenal Hyperplasia, run by H. Lundbeck A/S at 17 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- What is being tested?
- Lu AG13909
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 17 sitesin 9 countries
- Study dates
- 19 Dec 2022to 31 Dec 2027
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
17 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 9 countries
France4 sites, 4 recruiting
- Recruiting
Chu Angers
Angers, 49933
- Recruiting
CHU de Lille
Lille, 59000
- Recruiting
GH Pitié-Salpêtrière
Paris, 75013
- Recruiting
CHRU Strasbourg
Strasbourg, 67091
United Kingdom4 sites, 4 recruiting
- Recruiting
NIHR/Wellcome Trust Clinical Research Facility
Birmingham, B15 2TH
- Recruiting
Cambridge Clinical Research Centre
Cambridge, CB2 0SL
- Recruiting
NIHR Clinical Research Facility
London, SE1 9RT
- Recruiting
University College London Hospital - NIHR
London, W1T 7HA
Italy2 sites, 2 recruiting
- Recruiting
Azienda Ospedaliero Universitaria di Bologna
Bologna, 40138
- Recruiting
Azienda Ospedaliero-Universitaria Policlinico Umberto I, Roma
Roma, 00161
Sweden2 sites, 2 recruiting
- Recruiting
Sahlgrenska University Hospital
Gothenburg, 413 45
- Recruiting
Karolinska University Hospital
Stockholm, 17174
United States1 site, 1 recruiting
- Recruiting
University Hospital-University of Michigan
Ann Arbor, Michigan, 48109
Georgia1 site, 1 recruiting
- Recruiting
David Metreveli Medical Centre, Tbilisi
Tbilisi, 0144
Ireland1 site, 1 recruiting
- Recruiting
Beaumont Hospital Royal College of Surgeons in Ireland (RCSI), Dublin
Dublin, D02 YN77
Poland1 site, 1 recruiting
- Recruiting
Centrum Nowoczesnych Terapii, Dobry Lekarz
Dobry Lekarz, 60-324
About this study
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
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
- Being tested
- Lu AG13909
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lu AG13909Drug
Solution for infusion
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Lu AG13909 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Parts A and B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Measured: Up to Day 161
Parts A and B: Number of Participants With Anti-Drug Antibodies (ADAs)
Measured: Day 1 up to Day 161
Parts A and B: Cmax: Maximum Observed Serum Concentration of Lu AG13909
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: Baseline up to Day 85
Parts A and B: AUC0-tau: Area under the curve over a dosing interval
Measured: 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)
Measured: Day 169
Secondary outcomes (4)
Part C: Lu AG13909 Serum Concentrations Following Multiple IV Doses
Measured: Baseline up to Day 352
Part C: Change From Baseline of Lu AG13909 in Blood Concentrations of 17-OHP and A4
Measured: Baseline up to Day 169
Part C: Number of Participants With TEAEs
Measured: Baseline up to Day 352
Part C: Number of Participants With ADAs
Measured: Baseline up to Day 352
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jan 2023
- Study start19 Dec 2022
- Primary completion30 Sep 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- More trials run by H. Lundbeck A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- H. Lundbeck A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Email contact via H. Lundbeck A/S, Study director, HQ_Medinfo@Lundbeck.com
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05669950
- Sponsor's protocol ID
- 19873A
- Status last verified
- Mar 2026
- First submitted
- 20 Dec 2022
- Last updated
- 9 Mar 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No