Lu AG13909 for Congenital Adrenal Hyperplasia

Recruiting at 20 trial locations

Overseen By Email contact via H. Lundbeck A/S

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: H. Lundbeck A/S

Must be taking: Glucocorticoids, Mineralocorticoids

Disqualifiers: Pregnancy, Breastfeeding, Abnormal lab values, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

42 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines a new treatment called Lu AG13909, an experimental therapy for individuals with congenital adrenal hyperplasia (CAH), a rare genetic disorder affecting hormone production. The researchers aim to assess the safety and tolerability of various doses of Lu AG13909 and observe the body's response. Individuals diagnosed with CAH who have stable hormone replacement therapy may be suitable candidates for this trial. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in humans.

Is there any evidence suggesting that Lu AG13909 is likely to be safe for humans?

A previous study found Lu AG13909 safe for people with congenital adrenal hyperplasia (CAH), as participants did not experience serious side effects. Another study showed that Lu AG13909 could lower certain hormone levels, such as 17-hydroxyprogesterone, without causing harm. Animal studies revealed no harmful effects. These findings suggest that Lu AG13909 is generally safe for humans, but ongoing research will help confirm this. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for CAH?

Researchers are excited about Lu AG13909 for congenital adrenal hyperplasia (CAH) because it offers a new approach to treatment. Unlike traditional therapies that often focus on hormone replacement, Lu AG13909 is designed to be administered intravenously, which could allow for more precise control over hormone levels. This method might reduce the side effects associated with oral medications like glucocorticoids and mineralocorticoids, which are the current standards of care. Additionally, Lu AG13909 could provide a more consistent therapeutic effect, potentially improving quality of life for patients with CAH.

What evidence suggests that Lu AG13909 might be an effective treatment for congenital adrenal hyperplasia?

Research has shown that Lu AG13909, the investigational treatment in this trial, can help reduce symptoms of congenital adrenal hyperplasia (CAH). In earlier studies, patients tolerated Lu AG13909 well, and it helped lower adrenal androgens, hormones often overproduced in CAH. Additionally, animal studies found that Lu AG13909 significantly reduced important hormones like cortisol and aldosterone without causing harmful effects. These findings suggest that Lu AG13909 could help manage CAH by addressing the hormone imbalances typical of the condition. ^{2 4 5 6 7}

Who Is on the Research Team?

Email contact via H. Lundbeck A/S

Principal Investigator

[e-mail]

Are You a Good Fit for This Trial?

Inclusion Criteria

My morning blood test shows very high levels of 17-OHP.

I have been on a stable hormone replacement therapy for over a month.

I have been diagnosed with 21-hydroxylase deficiency CAH.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Participants receive multiple intravenous (IV) doses of Lu AG13909 per a prespecified dosing schedule

Duration not specified

Treatment Part B

Participants receive multiple IV doses of Lu AG13909 after Part A data shows safety and tolerability

Duration not specified

Treatment Part C

Participants receive multiple IV doses of Lu AG13909 after Part B data shows safety and tolerability

Duration not specified

Treatment Extension (optional)

Participants from Part C may continue in the optional Treatment Extension

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Lu AG13909

How Is the Trial Designed?

Group I: Lu AG13909 Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

H. Lundbeck A/S

Lead Sponsor

Trials

332

Recruited

78,300+

Charl van Zyl

H. Lundbeck A/S

Chief Executive Officer since 2023

Degree in Medical Biochemistry from the University of Cape Town, South Africa

Johan Luthman

H. Lundbeck A/S

Chief Medical Officer since 2019

MD from the University of Gothenburg, Sweden

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12543959/

MON-468 A Novel Anti-ACTH Antibody Lu AG13909 in ... - PMC

Conclusions: The preliminary results of this phase 1 study show that Lu AG13909 is well-tolerated and can reduce adrenal androgen production, ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05669950

NCT05669950 | A Trial of Lu AG13909 in Participants With ...

This trial will evaluate the effects of different doses of Lu AG13909 in adult participants with congenital adrenal hyperplasia, also called CAH.

3.

academic.oup.com

academic.oup.com/jes/article/9/Supplement_1/bvaf149.323/8297625

SAT-455 A Phase 2, Open-Label Trial Evaluating the Efficacy ...

A phase 1 trial (NCT05669950) in adults with classic CAH demonstrated that Lu AG13909 is well-tolerated and can reduce 17-hydroxyprogesterone ( ...

4.

prnewswire.com

prnewswire.com/news-releases/lundbeck-receives-orphan-drug-designation-in-the-us-and-eu-for-lu-ag13909-for-the-treatment-of-patients-with-congenital-adrenal-hyperplasia-302489314.html

Lundbeck receives orphan drug designation in the US and ...

In animal studies, Lu AG13909 has shown significant and durable reductions of corticosterone/cortisol and aldosterone. ... No adverse effects were ...

5.

hra.nhs.uk

hra.nhs.uk/planning-and-improving-research/application-summaries/research-summaries/study-of-lu-ag13909-in-participants-with-congenital-adrenal-hyperplasia/

* Study of Lu AG13909 in Participants with Congenital ...

Based on nonclinical data, Lu AG13909 is expected to reduce the abnormal androgen secretion from the adrenal glands, which is characteristic for CAH patients.

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11453276/

12484 Safety And Pharmacokinetics Of Anti-ACTH Antibody ...

Lu AG13909 is a novel, high-affinity, anti-ACTH monoclonal antibody that neutralizes ACTH-induced signaling. In preclinical models, Lu AG13909 dose-dependently ...

7.

lundbeck.com

lundbeck.com/us/newsroom/2025/lundbeck-receives-orphan-drug-designation-in-the-us-and-eu-for-l

Lundbeck receives orphan drug designation in the US and ...

In animal studies, Lu AG13909 has shown significant and durable reductions of corticosterone/cortisol and aldosterone.3 No adverse effects were ...