Evaluating Lu AG13909 for treating congenital adrenal hyperplasia
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 study is testing different doses of a new medication called Lu AG13909 to see if it can safely help adults with congenital adrenal hyperplasia manage their hormone levels better.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 42 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | H. Lundbeck A/S Industry-sponsored |
| Locations | 17 sites (Ann Arbor, Michigan and 16 other locations) |
| Trial ID | NCT05669950 on ClinicalTrials.gov |
What this trial studies
This trial evaluates the effects of varying doses of Lu AG13909 in adults diagnosed with congenital adrenal hyperplasia (CAH), a rare genetic disorder affecting hormone production. The study aims to assess the safety, tolerability, and pharmacokinetics of Lu AG13909, as well as the body's response to the treatment. Participants will be monitored for their hormone levels and overall health during the trial. The study is divided into phases to ensure thorough evaluation of the drug's effects.
Who should consider this trial
Good fit: Ideal candidates include adults with a confirmed diagnosis of 21-hydroxylase deficiency CAH who are stable on glucocorticoid and mineralocorticoid replacement therapies.
Not a fit: Patients without a confirmed diagnosis of CAH or those with other significant health issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve hormone regulation and quality of life for patients with congenital adrenal hyperplasia.
How similar studies have performed: While there have been studies on CAH treatments, the specific approach with Lu AG13909 is novel and has not been extensively tested in prior trials.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Ann Arbor, Michigan and 16 other locations
- University Hospital-University of Michigan — Ann Arbor, Michigan, United States (Recruiting)
- Rigshospitalet — Copenhagen, Denmark (Recruiting)
- Chu Angers — Angers, France (Recruiting)
- CHU de Lille — Lille, France (Recruiting)
- GH Pitié-Salpêtrière — Paris, France (Recruiting)
- CHRU Strasbourg — Strasbourg, France (Recruiting)
- David Metreveli Medical Centre, Tbilisi — Tbilisi, Georgia (Recruiting)
- Beaumont Hospital Royal College of Surgeons in Ireland (RCSI), Dublin — Dublin, Ireland (Recruiting)
- Azienda Ospedaliero Universitaria di Bologna — Bologna, Italy (Recruiting)
- Azienda Ospedaliero-Universitaria Policlinico Umberto I, Roma — Roma, Italy (Recruiting)
- Centrum Nowoczesnych Terapii, Dobry Lekarz — Dobry Lekarz, Poland (Recruiting)
- Sahlgrenska University Hospital — Gothenburg, Sweden (Recruiting)
- Karolinska University Hospital — Stockholm, Sweden (Recruiting)
- NIHR/Wellcome Trust Clinical Research Facility — Birmingham, United Kingdom (Recruiting)
- Cambridge Clinical Research Centre — Cambridge, United Kingdom (Recruiting)
- NIHR Clinical Research Facility — London, United Kingdom (Recruiting)
- University College London Hospital - NIHR — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Email contact via H. Lundbeck A/S
- Email: HQ_Medinfo@Lundbeck.com
- Phone: +45 36301311
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.