Table of contents

• Trial overview
• Who is being studied
• Trial phases and study design
• Main endpoints and what they mean
• Summary of the available trials
• What these studies may mean for patients

Trial overview

The available clinical trials are studying ATUMELNANT in people with congenital adrenal hyperplasia (CAH), including the classic form of the disease.^[1][2][3]

All three trials are listed as Authorised and are interventional studies, which means participants receive a study treatment and researchers measure its effects.^[1][2][3]

The main goals across the studies are to assess safety, tolerability, and effectiveness, especially control of morning androstenedione (A4) and the possibility of reducing daily glucocorticoid dose.^[1][2][3]

Who is being studied

One Phase 2 trial includes participants with CAH and plans to enroll 150 people.^[1]

One Phase 4 trial is for pediatric participants with classic congenital adrenal hyperplasia and plans to enroll 156 people.^[2]

One Phase 3 trial is for adults with classic congenital adrenal hyperplasia and plans to enroll 140 people.^[3]

So, the study program covers both children and adults, but only in the disease group listed in the trial records.^[1][2][3]

Trial phases and study design

The Phase 2 study is an earlier-stage trial that looks at safety and early signs of benefit in participants with CAH.^[1]

The Phase 3 study is a later-stage trial in adults and compares ATUMELNANT with placebo while checking whether adrenal androgen control is maintained during the 32-week treatment period.^[3]

The Phase 4 study is in pediatric participants and focuses on safety, effectiveness, and glucocorticoid dose reduction across different parts of the study.^[2]

All three studies are interventional, meaning the research team gives the study treatment and then checks the results over time.^[1][2][3]

Main endpoints and what they mean

The Phase 2 study measures treatment-emergent adverse events (new medical problems that start after treatment begins), serious adverse events, and adverse events that lead to stopping treatment.^[1]

It also measures glucocorticoid deficiency, adrenal insufficiency, adrenal crisis, and hospitalizations related to CAH.^[1]

For effectiveness, this study looks at the change from baseline in morning serum A4 before 11:00 AM over time.^[1]

The Phase 4 pediatric study also measures treatment-emergent adverse events, serious adverse events, and events leading to discontinuation in Part A.^[2]

In Part A of that study, researchers measure the change from baseline in morning A4 at Week 8.^[2]

In Part B, the study measures the percent change from baseline in daily glucocorticoid dose at Week 28 while morning A4 remains at or below the upper limit of normal.^[2]

In Part C, the study again measures change from baseline in morning A4 over time.^[2]

The Phase 3 adult study measures the proportion of participants with morning post-glucocorticoid A4 at or below the upper limit of normal who are on physiologic glucocorticoid replacement at Week 32.^[3]

Its brief summary says the study compares ATUMELNANT with placebo to see whether daily glucocorticoid dose can be reduced while adrenal androgen control is maintained.^[3]

Summary of the available trials

What these studies may mean for patients

These trials are trying to find out whether ATUMELNANT can help people with CAH keep adrenal hormones under control while using less glucocorticoid medicine.^[1][2][3]

They also check important safety outcomes, such as serious side effects, adrenal crisis, and hospital stays linked to CAH.^[1]

Because the studies include both pediatric participants and adults, the research program is looking at different age groups with the same disease.^[2][3]