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Corticosteroid
Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst Trial)
Phase 3
Waitlist Available
Research Sponsored by Neurocrine Biosciences
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be on a stable regimen of steroidal treatment for CAH.
Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
Must not have
Have a history of cancer unless considered cured.
Have a known history of clinically significant arrhythmia or abnormalities on ECG.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial
Summary
This trial is testing a new medication called crinecerfont in adults with a genetic condition that affects hormone production. The study aims to see if crinecerfont can help balance their hormone levels and improve their symptoms. Participants will take the medication for several months, with an option to continue longer.
Who is the study for?
Adults with classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency can join. They must be on stable CAH medication, agree to use contraception if they can have children, and follow study rules. Excluded are those with unstable conditions, hypersensitivity to similar drugs, certain medical histories like cancer or chronic glucocorticoid therapy needs.
What is being tested?
The trial is testing Crinecerfont against a placebo for CAH over 24 weeks in a double-blind setup where neither the participants nor the researchers know who gets what. After this phase, there's an open-label period where everyone receives Crinecerfont for up to three years.
What are the potential side effects?
While specific side effects of Crinecerfont aren't listed here, common ones may include reactions at the injection site, hormonal imbalances leading to mood changes or skin issues, and potential risks associated with long-term hormone therapy.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am on a consistent dose of steroids for congenital adrenal hyperplasia.
Select...
I have been diagnosed with classic 21-hydroxylase deficiency CAH.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have had cancer in the past but am considered cured.
Select...
I have a history of heart rhythm problems.
Select...
I have a serious health condition that is not CAH.
Select...
I have lost or donated more than 550 mL of blood in the last 8 weeks.
Select...
I need long-term steroid treatment due to a specific medical condition.
Select...
I have been diagnosed with a form of classic congenital adrenal hyperplasia.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Side effects data
From 2021 Phase 2 trial • 8 Patients • NCT0404514525%
Headache
13%
Myalgia
13%
Dermatitis contact
13%
Gastritis
13%
Frequent bowel movements
13%
Pyrexia
13%
Arthropod sting
13%
Nasopharyngitis
13%
Dizziness
13%
Blepharospasm
13%
Vomiting
100%
80%
60%
40%
20%
0%
Study treatment Arm
Crinecerfont 50 mg
Awards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: CrinecerfontExperimental Treatment1 Intervention
Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.
Group II: PlaceboPlacebo Group2 Interventions
Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Crinecerfont
2019
Completed Phase 2
~10
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Congenital Adrenal Hyperplasia (CAH) include glucocorticoids and CRF1 receptor antagonists like Crinecerfont. Glucocorticoids replace deficient cortisol and suppress excess adrenocorticotropic hormone (ACTH) production, thereby reducing the overproduction of androgens.
CRF1 receptor antagonists block the corticotropin-releasing factor 1 receptor, reducing ACTH release from the pituitary gland. This helps manage the hormonal imbalances in CAH patients, which is essential for controlling symptoms and improving long-term health outcomes.
Find a Location
Who is running the clinical trial?
Neurocrine BiosciencesLead Sponsor
75 Previous Clinical Trials
6,147 Total Patients Enrolled
Clinical Development LeadStudy DirectorNeurocrine Biosciences
24 Previous Clinical Trials
2,000 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You are allergic to any corticotropin releasing hormone antagonists.I have had cancer in the past but am considered cured.I have a history of heart rhythm problems.I have a serious health condition that is not CAH.I have lost or donated more than 550 mL of blood in the last 8 weeks.I am on a consistent dose of steroids for congenital adrenal hyperplasia.I need long-term steroid treatment due to a specific medical condition.You are currently dependent on drugs or alcohol, or actively abusing them.I have been diagnosed with classic 21-hydroxylase deficiency CAH.I have been diagnosed with a form of classic congenital adrenal hyperplasia.
Research Study Groups:
This trial has the following groups:- Group 1: Crinecerfont
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.