In short
Clinical trials are investigating ATUMELNANT in people with congenital adrenal hyperplasia, including adults and pediatric participants. These studies are looking at safety, tolerability, and how well the treatment may control adrenal androgen levels while helping reduce glucocorticoid use. The trials are in Phase 2, Phase 3, and Phase 4.
Key points
- ATUMELNANT is being studied in clinical trials for congenital adrenal hyperplasia, including classic congenital adrenal hyperplasia. The listed studies include adults and pediatric participants. The trials are in Phase 2, Phase 3, and Phase 4, so they cover earlier and later stages of research. The main goals are to check safety, tolerability, and whether the treatment can improve morning A4 levels. Some trials also look at whether daily glucocorticoid dose can be reduced while keeping hormone control.
Clinical trial overview
The available studies of ATUMELNANT are focused on congenital adrenal hyperplasia, including classic congenital adrenal hyperplasia. These are interventional studies, which means participants receive a study treatment so researchers can measure its effects.
All three listed trials are currently Authorised. The studies are looking at safety, tolerability, and whether ATUMELNANT can help control adrenal hormone levels while lowering the need for glucocorticoid treatment.
Who the trials include
The trial set includes both adult participants and pediatric participants. The pediatric study is designed for children and adolescents with classic congenital adrenal hyperplasia, while the adult study is for adults with the same disease.
The Phase 2 trial is broader in its condition wording and includes participants with congenital adrenal hyperplasia. The trial records do not provide more detailed age or eligibility rules, so only the population named in each study can be stated here.
Trial phases and study design
ATUMELNANT is being studied across Phase 2, Phase 3, and Phase 4 trials. Phase 2 usually helps researchers learn more about safety and early effectiveness, Phase 3 is a larger confirmatory stage, and Phase 4 is a later study stage that can add more information after earlier testing.
The Phase 2 study plans to enroll 150 participants, the Phase 4 pediatric study plans to enroll 156 participants, and the Phase 3 adult study plans to enroll 140 participants. This range shows that the program includes both mid-size and larger studies.
Main endpoints being measured
The studies mainly measure safety and efficacy, which means how well the treatment works. Safety outcomes include treatment-emergent adverse events, serious adverse events, and adverse events that lead to stopping treatment.
Some safety endpoints also look at glucocorticoid deficiency, adrenal insufficiency, adrenal crisis, and hospitalizations related to congenital adrenal hyperplasia. These terms describe important medical problems that can happen when the body does not have enough adrenal hormone support or when the disease becomes severe.
The main efficacy measure in the Phase 2 trial is the change from baseline in morning serum A4 over time. In the pediatric Phase 4 study, one part looks at the change from baseline in morning A4 at Week 8, another part looks at the percent change in daily glucocorticoid dose at Week 28 while serum early morning A4 stays at or below the upper limit of normal, and another part again measures change in morning A4 over time.
The adult Phase 3 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. In simple terms, this asks how many participants can keep hormone levels controlled while using a replacement dose of glucocorticoid that is intended to be close to normal body needs.
Key trial details
The Phase 2 study is titled “A study to learn about the safety and effectiveness of CRN04894 on participants,” and it is listed under congenital adrenal hyperplasia. Its brief summary says the main safety objective is to evaluate safety and tolerability, and the main efficacy objective is to evaluate change from baseline in serum androstenedione (A4).
The pediatric Phase 4 study is titled “A Study in Pediatric Participants with Congenital Adrenal Hyperplasia,” and it focuses on classic congenital adrenal hyperplasia. Its brief summary says the study wants to evaluate safety and tolerability in pediatric participants, and also whether ATUMELNANT can reduce daily glucocorticoid dose while keeping adrenal androgen levels normalized.
The adult Phase 3 study is titled “A Study to Evaluate Atumelnant in Adults with Congenital Adrenal Hyperplasia,” and it also focuses on classic congenital adrenal hyperplasia. Its brief summary says the study compares ATUMELNANT with placebo to see whether it can reduce daily glucocorticoid dosage while maintaining adrenal androgen control at the end of the 32-week treatment period.
The intervention records show oral tablet forms for ATUMELNANT in the pediatric and adult studies, with tablet strengths listed in the source data. The source data also names a 120 mg oral dose in the Phase 2 study.
Summary of the listed trials
| Trial ID | Title | Phase | Condition | Status | Enrollment | Main focus |
|---|---|---|---|---|---|---|
| 2024-514846-35-00 | A study to learn about the safety and effectiveness of CRN04894 on participants | Phase 2 | Congenital adrenal hyperplasia | Authorised | 150 | Safety, tolerability, and change in morning serum A4 |
| 2024-519578-38-00 | A Study in Pediatric Participants with Congenital Adrenal Hyperplasia | Phase 4 | Classic congenital adrenal hyperplasia | Authorised | 156 | Safety, A4 change, and glucocorticoid dose reduction |
| 2024-519579-24-00 | A Study to Evaluate Atumelnant in Adults with Congenital Adrenal Hyperplasia | Phase 3 | Classic congenital adrenal hyperplasia | Authorised | 140 | Placebo comparison and control of adrenal androgen levels |