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Corticosteroid

Tildacerfont for Congenital Adrenal Hyperplasia

Phase 2
Waitlist Available
Led By Kyriakie Sarafoglou, M.D
Research Sponsored by Spruce Biosciences
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
Has been on a stable supraphysiologic dose of GC replacement ≥15 mg/day and ≤60 mg/day in HC equivalents
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights

Study Summary

This trial is testing a possible new treatment for classic CAH, which is a condition that affects the adrenal glands. The trial will last up to 70 weeks, and there is an optional extension period of up to 240 weeks.

Who is the study for?
Adults over 18 with classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency, on a stable dose of certain steroids. Not for those who've had both adrenal glands removed, have other forms of CAH, are on dexamethasone, or allergic to Tildacerfont.Check my eligibility
What is being tested?
The trial tests the effectiveness and safety of Tildacerfont compared to a placebo in adults with CAH over up to 70 weeks. Participants must have high biomarkers while on their current steroid regimen. An optional extension allows treatment for up to 240 weeks.See study design
What are the potential side effects?
While specific side effects aren't listed here, participants may experience reactions related to Tildacerfont or its components. Side effects could include typical drug reactions such as allergies, gastrointestinal issues, fatigue or hormonal imbalances.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I was diagnosed with classic CAH in childhood and am currently treated with specific steroids.
Select...
I have been on a stable high dose of steroid treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in androstenedione
Secondary outcome measures
Effectiveness in reducing TART(s) in Male CAH subjects
Proportion of subjects who achieve reduction A4 levels
Proportion of subjects who achieve reduction in 17-OHP
Other outcome measures
Change in acne in patients with acne at baseline
Change in adrenal hormones [time frame: up to 12 months]
Change in hirsutism in female patients with hirsutism at baseline
+1 more

Trial Design

4Treatment groups
Experimental Treatment
Placebo Group
Group I: Tildacerfont Group 3Experimental Treatment1 Intervention
Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3
Group II: Tildacerfont Group 2Experimental Treatment1 Intervention
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2
Group III: Tildacerfont Group 1Experimental Treatment1 Intervention
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1
Group IV: PlaceboPlacebo Group1 Intervention
Placebo administered daily via oral tablet for 12 weeks.

Find a Location

Who is running the clinical trial?

Spruce BiosciencesLead Sponsor
5 Previous Clinical Trials
209 Total Patients Enrolled
Kyriakie Sarafoglou, M.DPrincipal InvestigatorDept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota
Site NameJohns Hopkins
JH IRB Protocol NumberIRB00240449
Principal InvestigatorDr. Hamrahian

Media Library

Tildacerfont (Corticosteroid) Clinical Trial Eligibility Overview. Trial Name: NCT04457336 — Phase 2
Congenital Adrenal Hyperplasia Research Study Groups: Tildacerfont Group 3, Tildacerfont Group 2, Placebo, Tildacerfont Group 1
Congenital Adrenal Hyperplasia Clinical Trial 2023: Tildacerfont Highlights & Side Effects. Trial Name: NCT04457336 — Phase 2
Tildacerfont (Corticosteroid) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04457336 — Phase 2
~20 spots leftby Jun 2025
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