Atumelnant for Congenital Adrenal Hyperplasia

Recruiting at 5 trial locations

Overseen By Crinetics Clinical Trials

Age: < 18

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Crinetics Pharmaceuticals Inc.

Must be taking: Glucocorticoids

Must not be taking: Growth hormones

Disqualifiers: Non-classic CAH, Cancer, Pregnant, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

153 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called atumelnant, a potential drug for children with classic congenital adrenal hyperplasia (CAH), a condition that affects hormone production. The trial aims to assess the safety and effectiveness of this treatment and understand its mechanism in the body. Participants will receive the treatment, a placebo (a harmless pill with no active drug), or both at different stages. Children with a confirmed diagnosis of classic CAH who are on stable hormone therapy might be suitable for this trial. As a Phase 2, Phase 3 trial, this study is crucial for measuring the treatment's effectiveness in a smaller group and represents the final step before FDA approval. It offers participants a chance to contribute to potentially groundbreaking advancements in CAH treatment.

Is there any evidence suggesting that atumelnant is likely to be safe for pediatric participants with congenital adrenal hyperplasia?

Research has shown that atumelnant has been tested in people, with promising results for safety. In earlier studies, most participants tolerated atumelnant well. The most common side effects were mild, such as headaches and some nausea, and they usually resolved on their own without medical intervention.

The trial under consideration is in a later stage, indicating that earlier trials have already demonstrated atumelnant's safety for testing in more people. This is a positive sign for its safety.

While atumelnant is still under study, current evidence supports its use in clinical settings. Discuss any concerns with a doctor before joining a clinical trial.

Why do researchers think this study treatment might be promising?

Atumelnant is unique because it targets congenital adrenal hyperplasia (CAH) through a new mechanism of action that distinguishes it from current standard treatments like glucocorticoids and mineralocorticoids. While most existing treatments focus on replacing deficient hormones, Atumelnant aims to directly modulate the production of adrenal hormones, potentially offering a more precise approach to managing CAH. Researchers are excited about Atumelnant because it could lead to better hormonal balance with fewer side effects, improving quality of life for patients. Additionally, the varied treatment phases, including open-label and double-blind designs, allow for a comprehensive assessment of its safety and efficacy.

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

Research has shown that atumelnant, which participants in this trial may receive, offers promising results for treating congenital adrenal hyperplasia (CAH), a condition affecting hormone production. In studies, atumelnant was well-tolerated and quickly reduced high levels of adrenal androgens, hormones often elevated in people with CAH. Remarkably, key disease indicators dropped by up to 80%. Importantly, these studies reported no serious side effects. This evidence supports atumelnant's potential to help manage CAH symptoms. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for children and teens born with Congenital Adrenal Hyperplasia (CAH), aged 1 to <18, who have been on stable hormone replacement therapy for at least a month. They should be able to follow the treatment plan and have certain hormone levels above normal. Those with normal thyroid function are eligible.

Inclusion Criteria

I am under 18 years old and can sign the consent form.

I have been diagnosed with classic CAH due to 21-hydroxylase deficiency.

My morning A4 hormone levels are high before taking any steroid medication.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Open-label, semi-sequential cohorts for ages 12 to <18 years, followed by a cohort for ages 1 to 11 years

Varies by cohort

Treatment Part B

Double-blind, randomized, placebo-controlled confirmatory portion

Varies

Open-label Extension (Part C)

Open-label treatment period for participants entering from Part A and B

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Atumelnant

Trial Overview The study tests Atumelnant's safety and effectiveness in managing CAH symptoms compared to a placebo. It will also look into how the body processes the drug (pharmacokinetics) and its impact on the body's functions (pharmacodynamics).

How Is the Trial Designed?

Group I: Treatment (Part A) Experimental Treatment 1 Intervention

Open-label, semi-sequential cohorts.

Group II: Open-Label Treatment (Part C) Experimental Treatment 1 Intervention

Open-label treatment period for participants entering Part C from Part A and B.

Group III: Active Treatment (Part B) Experimental Treatment 1 Intervention

Randomized, Parallel Arms, Double-Blind

Group IV: Placebo (Part B) Placebo Group 1 Intervention

Randomized, Parallel Arms, Double-Blind

Who Is Running the Clinical Trial?

Crinetics Pharmaceuticals Inc.

Lead Sponsor

Trials

13

Recruited

760+

Citations

1. crinetics.com crinetics.com/wp-content/uploads/2025/01/3-FINAL-CAH-Ph2-Data-Release-Jan-2025.pdf

Topline Phase 2 results from Atumelnant in Congenital ... In clinical studies, atumelnant was well-tolerated with no severe or serious adverse events. Efficacy and safety support progressing to Phase 3.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11455294/

12537 Once Daily Oral Atumelnant (CRN04894) Induces ... In conclusion, these data demonstrate rapid, profound, and sustained suppression of A4 and 17-OHP with 80 mg once daily, oral atumelnant. There were no serious ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT07144163

A Study to Evaluate Atumelnant in Adults With Congenital ... The purpose of this study is to evaluate the efficacy, safety, PK, and PD of atumelnant in adults with classic CAH due to 21-OHD. Detailed ...

4. crinetics.com crinetics.com/crinetics-announces-positive-topline-results-from-phase-2-trial-of-atumelnant-in-congenital-adrenal-hyperplasia-cah/

Crinetics Announces Positive Topline Results From Phase ... This Phase 2 study demonstrated that atumelnant was well tolerated and resulted in a reduction of adrenal androgen levels so rapid and robust.

5. hcplive.com hcplive.com/view/fda-grants-atumelnant-orphan-drug-designation-for-congenital-adrenal-hyperplasia

FDA Grants Atumelnant Orphan Drug Designation for ... Atumelnant demonstrated rapid, substantial, and sustained reductions in key biomarkers across all doses, including up to an 80% mean reduction ...