Atumelnant for Congenital Adrenal Hyperplasia

Recruiting at 8 trial locations

Overseen By Crinetics Clinical Trials Crinetics Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Crinetics Pharmaceuticals Inc.

Must be taking: Glucocorticoids

Disqualifiers: Alcohol or drug abuse, Cancer, others

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

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

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 2 years

150 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called atumelnant (CRN04894) for individuals with congenital adrenal hyperplasia (CAH), a condition affecting hormone production. The goal is to determine if the treatment is safe, well-tolerated, and effective over the long term. It is open to those who have already participated in a related study and are managing their CAH with a stable glucocorticoid (steroid hormone) regimen. Participants should have completed a prior trial with atumelnant and shown benefits from ongoing treatment. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

The trial requires participants to continue their current glucocorticoid replacement therapy (like hydrocortisone) as part of the study. There is no mention of stopping other medications, so it's best to discuss your specific situation with the study team.

Is there any evidence suggesting that atumelnant (CRN04894) is likely to be safe for humans?

Research has shown that atumelnant (CRN04894) is generally well-tolerated. In earlier studies, participants did not experience any severe or serious side effects. While some side effects might occur, they are usually not serious or long-lasting. These positive safety results support further testing in future studies, indicating that the treatment is considered safe for continued research. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for Congenital Adrenal Hyperplasia (CAH) focus on replacing deficient hormones with glucocorticoids and mineralocorticoids. But atumelnant stands out because it targets the condition differently. It uses a new active ingredient, CRN04894, which acts by modulating the adrenal glands directly to balance hormone levels more naturally. Researchers are excited about atumelnant because it has the potential to stabilize hormone levels without the side effects often associated with long-term steroid use, offering a more precise and potentially safer option for managing CAH.

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

Research has shown that atumelnant, the investigational treatment in this trial, may help treat Congenital Adrenal Hyperplasia (CAH). In clinical studies, atumelnant reduced key hormones linked to CAH by over 90%. Participants tolerated it well, experiencing no serious side effects. These studies suggest that atumelnant could effectively manage CAH symptoms. ^{2 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with Congenital Adrenal Hyperplasia who have been stable on glucocorticoid replacement therapy. Women must be nonchildbearing or use contraception, and men must use condoms or abstain. Participants should not have uncontrolled diabetes, recent major surgery, mental conditions affecting study comprehension, known allergies to test materials, substance abuse issues, significant abnormal lab tests or certain heart conditions.

Inclusion Criteria

I am consistently taking my prescribed glucocorticoid medication as directed.

I am either not able to have children, use effective birth control, or use condoms.

Participants with CAH who have completed a Crinetics CRN04894 study or completed treatment in a Crinetics CRN04894 study, and in the opinion of the Investigator had an acceptable benefit-risk assessment in the completed study and would benefit from continued dosing in this extension study.

Exclusion Criteria

Participant is an employee or immediate family member of an employee of Crinetics.

I have a condition that requires me to take steroids regularly.

My diabetes is not well-managed, with an HbA1c level of 8.5% or higher.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive open-label treatment with atumelnant (CRN04894) for up to 2 years

Up to 2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• atumelnant (CRN04894)

Trial Overview The trial is testing the long-term safety and effectiveness of a drug called atumelnant (CRN04894) for treating Congenital Adrenal Hyperplasia. It's an extension study which means it continues from previous research where this medication was used.

How Is the Trial Designed?

Group I: Treatment Experimental Treatment 1 Intervention

Open-label treatment period (up to 2 years). The maximum atumelnant dose permitted is not to exceed the highest atumelnant dose explored in a parent study for the indication.

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 ... ... Congenital Adrenal Hyperplasia: Initial Results From A ... This ongoing study will explore further the safety and efficacy of various doses of atumelnant.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05907291

Study Details | NCT05907291 | Evaluate the Safety, ... This Phase 2, open-label, sequential dose cohort study will evaluate the efficacy, safety, PK, and PD of atumelnant (CRN04894) when administered for 12 weeks in ...

4. academic.oup.com academic.oup.com/jes/article/9/Supplement_1/bvaf149.272/8298490

OR07-06 Once-Daily Oral Atumelnant (CRN04894) Induces ... We report results from 3 of 4 cohorts of a 12-week, Phase 2, open-label, dose-finding study of atumelnant in patients with CAH (NCT05907291).

5. crinetics.com crinetics.com/crinetics-announces-positive-initial-findings-at-endo-2024-for-atumelnant-in-two-ongoing-open-label-studies-for-the-treatment-of-congenital-adrenal-hyperplasia-cah-and-acth-dependent-cushing/

News & Events Atumelnant (80 mg) CAH participants achieved more than a 90% reduction of A4 and 97% reduction of 17-OHP on atumelnant (80 mg), beginning at two weeks and ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06712823

NCT06712823 | An Extension Study to Evaluate Safety and ... This single-arm, long-term, open-label, study is designed to evaluate the safety, tolerability, and efficacy of atumelnant (CRN04894) in participants with ...

7. ctv.veeva.com ctv.veeva.com/study/evaluate-the-safety-efficacy-and-pharmacokinetics-of-crn04894-in-participants-with-congenital-adre

Evaluate the Safety, Efficacy, and Pharmacokinetics of ... Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn). C. Crinetics ...

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

FDA Grants Atumelnant Orphan Drug Designation for ... Atumelnant was generally well-tolerated with a positive safety profile. ... Atumelnant in Congenital Adrenal Hyperplasia (CAH). Crinectics ...