A Phase 2 interventional study of atumelnant (CRN04894) in Congenital Adrenal Hyperplasia and Classic Congenital Adrenal Hyperplasia, sponsored by Crinetics Pharmaceuticals Inc.. Completed at 27 sites in 7 countries. Open to participants aged 16 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-10.
Sponsored by Crinetics Pharmaceuticals Inc. (part of Vertex Pharmaceuticals Incorporated) · Phase 2, Interventional, and Treatment
The purpose of this Phase 2, open-label, sequential dose cohort study is to evaluate the safety, efficacy, and pharmacokinetics (PK) of atumelnant (CRN04894) in participants with classic congenital adrenal hyperplasia (CAH) caused by 21-hydroxylase deficiency.
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 participants with CAH caused by 21-hydroxylase deficiency. Up to 42 participants will be enrolled in the study.
101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.
This study's enrollment of 38 is close to the median of 37 across 50 interventional studies indexed under Adrenal Hyperplasia, Congenital.
Browse Adrenal Hyperplasia, Congenital studies →Crinetics Pharmaceuticals Inc. is the lead sponsor of 19 studies on the registry; 3 are open to participants now.
Of its 12 completed or terminated interventional studies of FDA-regulated products, 6 (50%) have results posted.
First submitted before Crinetics Pharmaceuticals Inc. became part of Vertex Pharmaceuticals Incorporated.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Sequential, open-label, 12-week fixed-dose cohorts.
Drug: atumelnant (CRN04894)
Atumelnant is an orally active nonpeptide melanocortin 2 receptor (MC2R) or adrenocorticotropic hormone (ACTH) antagonist.
Change From Baseline in Morning (Before 11:00) Serum Androstenedione (A4) PM Dosing
A4 is the principal biochemical measure of disease activity in adult CAH. Blood samples were collected for the measurement of serum concentration of A4 prior to glucocorticoid administration. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was obtained by subtracting post-baseline visit value minus baseline value.
Time frame: Baseline and Week 12
Change From Baseline in Morning (Before 11:00) Serum Androstenedione (A4) AM Dosing
A4 is the principal biochemical measure of disease activity in adult CAH. Blood samples were collected for the measurement of serum concentration of A4 prior to glucocorticoid administration. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was obtained by subtracting post-baseline visit value minus baseline value.
Time frame: Baseline and Week 12
Number of Participants Reporting of Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, and Adverse Events Leading to Discontinuation Throughout the Study
An adverse event (AE) is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether considered related to the study intervention or not. TEAEs were collected in the full analysis set which consisted of all participants who received at least one dose of study drug. A serious AE is defined by its severe clinical consequences: it is any event that results in death, is life-threatening, requires inpatient hospitalization, results in persistent disability, or requires medical intervention to prevent these outcomes.
Time frame: From Day 1 to Up to Week 16 (End of study)
Change From Baseline in Morning (Before 11:00) Serum 17-hydroxyprogesterone (17-OHP) PM Dosing
Elevated levels of the immediate enzyme substrate 17-OHP are indicative of CAH. The secondary efficacy endpoint was change from baseline in morning serum 17-OHP at Week 12. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was analyzed using the mean at baseline and at each visit, for all participants with both baseline and the corresponding visit values.
Time frame: Baseline and Week 12
Change From Baseline in Morning (Before 11:00) Serum 17-hydroxyprogesterone (17-OHP) AM Dosing
Elevated levels of the immediate enzyme substrate 17-OHP are indicative of CAH. The secondary efficacy endpoint was change from baseline in morning serum 17-OHP at Week 12. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was analyzed using the mean at baseline and at each visit, for all participants with both baseline and the corresponding visit values.
Time frame: Baseline and Week 12
This was a Phase 2, open-label, sequential dose cohort study that evaluated the safety, efficacy, and pharmacokinetics of atumelnant when administered for 12 weeks in adult participants with congenital adrenal hyperplasia (CAH) caused by 21-hydroxylase deficiency (21-OHD). This study included 4 dose cohorts.
| Milestone | Cohort 2: Atumelnant 40 mg QD PM | Cohort 1: Atumelnant 80 mg QD PM | Cohort 3: Atumelnant 120 mg QD PM | Cohort 4: Atumelnant 80 mg QD AM |
|---|---|---|---|---|
| Started | 11 | 11 | 6 | 10 |
| Completed | 11 | 11 | 6 | 8 |
| Not completed | 0 | 0 | 0 | 2 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 2 |
A4 is the principal biochemical measure of disease activity in adult CAH. Blood samples were collected for the measurement of serum concentration of A4 prior to glucocorticoid administration. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was obtained by subtracting post-baseline visit value minus baseline value.
| nanograms per deciliter (ng/dL) | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM |
|---|---|---|---|
| Change From Baseline in Morning (Before 11:00) Serum Androstenedione (A4) PM Dosing | -601.2 ± 143.07 | -771.2 ± 148.22 | -953.4 ± 191.02 |
A4 is the principal biochemical measure of disease activity in adult CAH. Blood samples were collected for the measurement of serum concentration of A4 prior to glucocorticoid administration. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was obtained by subtracting post-baseline visit value minus baseline value.
| ng/dL | Atumelnant 80 mg QD AM |
|---|---|
| Change From Baseline in Morning (Before 11:00) Serum Androstenedione (A4) AM Dosing | -738.4 ± 566.01 |
An adverse event (AE) is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether considered related to the study intervention or not. TEAEs were collected in the full analysis set which consisted of all participants who received at least one dose of study drug. A serious AE is defined by its severe clinical consequences: it is any event that results in death, is life-threatening, requires inpatient hospitalization, results in persistent disability, or requires medical intervention to prevent these outcomes.
| Participants | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM | Atumelnant 80 mg QD AM |
|---|---|---|---|---|
| Any TEAE | 8 | 8 | 5 | 10 |
| Any Serious TEAE | 0 | 0 | 0 | 0 |
| Adverse events leading to study discontinuation | 0 | 0 | 0 | 0 |
Elevated levels of the immediate enzyme substrate 17-OHP are indicative of CAH. The secondary efficacy endpoint was change from baseline in morning serum 17-OHP at Week 12. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was analyzed using the mean at baseline and at each visit, for all participants with both baseline and the corresponding visit values.
| ng/dL | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM |
|---|---|---|---|
| Change From Baseline in Morning (Before 11:00) Serum 17-hydroxyprogesterone (17-OHP) PM Dosing | -7714.5 ± 2173.28 | -8773.8 ± 2288.82 | -9611.0 ± 2942.82 |
Elevated levels of the immediate enzyme substrate 17-OHP are indicative of CAH. The secondary efficacy endpoint was change from baseline in morning serum 17-OHP at Week 12. Baseline is defined as the last non-missing morning window value prior to or on the same day as the first dose date of atumelnant. Change from baseline was analyzed using the mean at baseline and at each visit, for all participants with both baseline and the corresponding visit values.
| ng/dL | Atumelnant 80 mg QD AM |
|---|---|
| Change From Baseline in Morning (Before 11:00) Serum 17-hydroxyprogesterone (17-OHP) AM Dosing | -7893.8 ± 5414.95 |
Collected over From Day 1 to Up to Week 16 (End of study). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Atumelnant 40 mg QD PM | 0/11 (0%) | 0/11 (0%) | 8/11 (72.7%) |
| Atumelnant 80 mg QD PM | 0/11 (0%) | 0/11 (0%) | 8/11 (72.7%) |
| Atumelnant 120 mg QD PM | 0/6 (0%) | 0/6 (0%) | 5/6 (83.3%) |
| Atumelnant 80 mg QD AM | 0/10 (0%) | 0/10 (0%) | 10/10 (100%) |
| Event | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM | Atumelnant 80 mg QD AM |
|---|---|---|---|---|
| HeadacheNervous system disorders | 2/11 | 4/11 | 2/6 | 5/10 |
| FatigueGeneral disorders | 3/11 | 1/11 | 1/6 | 1/10 |
| DiarrhoeaGastrointestinal disorders | 1/11 | 1/11 | 0/6 | 2/10 |
| Glucocorticoid deficiencyEndocrine disorders | 0/11 | 0/11 | 0/6 | 2/10 |
| Upper respiratory tract infectionInfections and infestations | 0/11 | 2/11 | 0/6 | 0/10 |
| Decreased appetiteMetabolism and nutrition disorders | 2/11 | 0/11 | 0/6 | 0/10 |
| DizzinessNervous system disorders | 0/11 | 0/11 | 1/6 | 0/10 |
| ParaesthesiaNervous system disorders | 0/11 | 0/11 | 1/6 | 0/10 |
| NauseaGastrointestinal disorders | 1/11 | 0/11 | 1/6 | 1/10 |
| Abdominal painGastrointestinal disorders | 0/11 | 0/11 | 1/6 | 1/10 |
The Full Analysis Set consisted of all participants who received at least one dose of Atumelnant.
| Age, Continuous(years) | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM | Atumelnant 80 mg QD AM | Total |
|---|---|---|---|---|---|
| Mean | 28.0 ± 7.67 | 33.0 ± 6.20 | 34.0 ± 10.02 | 38.6 ± 13.79 | 33.2 ± 10.10 |
| Sex: Female, Male(Participants) | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM | Atumelnant 80 mg QD AM | Total |
|---|---|---|---|---|---|
| Female | 4 | 8 | 3 | 6 | 21 |
| Male | 7 | 3 | 3 | 4 | 17 |
| Ethnicity (NIH/OMB)(Participants) | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM | Atumelnant 80 mg QD AM | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 7 | 8 | 3 | 3 | 21 |
| Not Hispanic or Latino | 4 | 3 | 1 | 7 | 15 |
| Unknown or Not Reported | 0 | 0 | 2 | 0 | 2 |
| Race/Ethnicity, Customized(Participants) | Atumelnant 40 mg QD PM | Atumelnant 80 mg QD PM | Atumelnant 120 mg QD PM | Atumelnant 80 mg QD AM | Total |
|---|---|---|---|---|---|
| Asian | 1 | 0 | 0 | 0 | 1 |
| Black or African American | 1 | 1 | 0 | 0 | 2 |
| White | 6 | 8 | 4 | 7 | 25 |
| Multiple | 1 | 1 | 0 | 0 | 2 |
| Other | 2 | 1 | 2 | 3 | 8 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Atumelnant
Adrenal Hyperplasia, Congenital →
Crinetics Pharmaceuticals Inc. →