Atumelnant for Congenital Adrenal Hyperplasia
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.
Will I have to stop taking my current medications?
The trial requires participants to be on a stable glucocorticoid (GC) therapy for at least one month before screening, so you won't need to stop this medication. However, you must not have used other GCs within 30 days of screening or had a stress dose of GC therapy within 2 weeks of screening.
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.12345
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
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part A
Open-label, semi-sequential cohorts for ages 12 to <18 years, followed by a cohort for ages 1 to 11 years
Treatment Part B
Double-blind, randomized, placebo-controlled confirmatory portion
Open-label Extension (Part C)
Open-label treatment period for participants entering from Part A and B
Follow-up
Participants are monitored for safety and effectiveness after treatment
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?
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Treatment groups
Experimental Treatment
Placebo Group
Open-label, semi-sequential cohorts.
Open-label treatment period for participants entering Part C from Part A and B.
Randomized, Parallel Arms, Double-Blind
Randomized, Parallel Arms, Double-Blind
Find a Clinic Near You
Who Is Running the Clinical Trial?
Crinetics Pharmaceuticals Inc.
Lead Sponsor
Citations
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.
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 ...
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-hyperplasiaFDA 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 ...
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