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Corticosteroid
Tildacerfont for Congenital Adrenal Hyperplasia
Phase 2
Recruiting
Research Sponsored by Spruce Biosciences
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male and female subjects aged 2 to 17 years
Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis
Must not have
CAH not due to 21-OHD
History of bilateral adrenalectomy or hypopituitarism
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 4 weeks or 12 weeks
Awards & highlights
Summary
This trial is testing a medication called tildacerfont to see if it is safe and effective for people with Congenital Adrenal Hyperplasia (CAH). The treatment involves taking the medication for a period of time, followed by a safety check. Tildacerfont aims to help balance hormones that are not properly regulated in people with CAH. Tildacerfont has been studied for its effectiveness in adults with Congenital Adrenal Hyperplasia.
Who is the study for?
This trial is for children aged 2 to 17 with Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. They must have been on a stable dose of glucocorticoid replacement for at least one month. It's not suitable for those without this specific CAH type, who've had both adrenal glands removed, have unstable medical conditions, are pregnant or nursing females, or have bleeding disorders.
What is being tested?
The study tests the safety and effectiveness of Tildacerfont in treating pediatric patients with CAH. This medication could potentially help manage hormone levels in these young patients.
What are the potential side effects?
While the trial aims to establish the side effects of Tildacerfont in children with CAH, potential risks may include hormonal imbalances, growth issues, and typical drug-related reactions like nausea or rashes.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 2 and 17 years old.
Select...
I have CAH and need ongoing hormone therapy since my diagnosis.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My adrenal hyperplasia is not caused by 21-hydroxylase deficiency.
Select...
I have had surgery to remove both adrenal glands or have a condition affecting my pituitary gland.
Select...
I am not pregnant or nursing.
Select...
I have a history of bleeding disorders.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 4 weeks or 12 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~4 weeks or 12 weeks
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of participants with Treatment-emergent adverse event (TEAE) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Secondary study objectives
Proportion of participants who achieve a reduction in androstenedione (A4) or reduction in glucocorticoid (GC) dosing
Proportion of participants with elevated baseline A4 who achieve a reduction in A4
Proportion of participants with elevated baseline A4 who achieve a reduction in A4 who achieve A4 normalization
+1 moreTrial Design
9Treatment groups
Experimental Treatment
Group I: Cohort 9: Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 4 consecutive weeks.
Group II: Cohort 8: Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 4 consecutive weeks.
Group III: Cohort 7: Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 4 consecutive weeks.
Group IV: Cohort 6: Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered twice daily for 4 consecutive weeks.
Group V: Cohort 5: Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 4 consecutive weeks.
Group VI: Cohort 4: Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 4 consecutive weeks.
Group VII: Cohort 3: Age 2-10 Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 12 consecutive weeks.
Group VIII: Cohort 2: Age 11-17 Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 12 consecutive weeks.
Group IX: Cohort 1: Age 11-17 Treatment with TildacerfontExperimental Treatment1 Intervention
Oral Tildacerfont administered daily for 12 consecutive weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tildacerfont
2022
Completed Phase 2
~30
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 mineralocorticoids, which work by replacing deficient hormones and suppressing excess androgen production. Glucocorticoids, such as hydrocortisone, mimic cortisol, helping to regulate metabolism, immune response, and stress.
Mineralocorticoids, like fludrocortisone, replace aldosterone to maintain salt and water balance. Tildacerfont, a CRF1 receptor antagonist, works by inhibiting the corticotropin-releasing factor receptor 1, reducing the overproduction of adrenocorticotropic hormone (ACTH) and subsequently lowering androgen levels.
This is crucial for CAH patients as it helps manage symptoms and prevents complications associated with hormone imbalances.
Find a Location
Who is running the clinical trial?
Spruce BiosciencesLead Sponsor
5 Previous Clinical Trials
250 Total Patients Enrolled
Will Charlton, MDStudy DirectorSpruce Biosciences
6 Previous Clinical Trials
648 Total Patients Enrolled
Dennis AguilingStudy DirectorDirector of Clinical Operations
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I do not have any unstable or serious long-term health conditions.I have been on a stable dose of glucocorticoid replacement for at least 1 month.I have had surgery to remove both adrenal glands or have a condition affecting my pituitary gland.I am between 2 and 17 years old.I have CAH and need ongoing hormone therapy since my diagnosis.My adrenal hyperplasia is not caused by 21-hydroxylase deficiency.I am not pregnant or nursing.I have a history of bleeding disorders.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 5: Treatment with Tildacerfont
- Group 2: Cohort 7: Treatment with Tildacerfont
- Group 3: Cohort 4: Treatment with Tildacerfont
- Group 4: Cohort 8: Treatment with Tildacerfont
- Group 5: Cohort 6: Treatment with Tildacerfont
- Group 6: Cohort 9: Treatment with Tildacerfont
- Group 7: Cohort 1: Age 11-17 Treatment with Tildacerfont
- Group 8: Cohort 2: Age 11-17 Treatment with Tildacerfont
- Group 9: Cohort 3: Age 2-10 Treatment with Tildacerfont
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
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.
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