Tildacerfont Group 3 for Congenital Adrenal Hyperplasia

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Spruce Study Site, Napoli, ItalyCongenital Adrenal Hyperplasia
Eligibility
18 - 55
All Sexes
What conditions do you have?
Select

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.

Eligible Conditions
  • Congenital Adrenal Hyperplasia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

2 Primary · 7 Secondary · Reporting Duration: 52 weeks

12 weeks
Change in androstenedione
Change in androstenedione [time frame: up to 3 months]
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
18 weeks
Absolute change in key hormones
Change in acne in patients with acne at baseline
Change in hirsutism in female patients with hirsutism at baseline
Percentage change from Baseline in key hormones
Proportion of subjects who achieve ≤ ULN (upper limit normal) or ≤ 4x ULN in key hormones
52 weeks
Change in adrenal hormones [time frame: up to 12 months]
Incidence of Adverse Events and Serious Adverse Events (safety analysis) [Time Frame: up to 12 months]
Normalization of androstenedione [time frame: up to 12 months]

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

4 Treatment Groups

Tildacerfont Group 3
1 of 4
Tildacerfont Group 2
1 of 4
Tildacerfont Group 1
1 of 4
Placebo
1 of 4

Experimental Treatment

Non-Treatment Group

72 Total Participants · 4 Treatment Groups

Primary Treatment: Tildacerfont Group 3 · Has Placebo Group · Phase 2

Tildacerfont Group 3
Drug
Experimental Group · 1 Intervention: Tildacerfont/Placebo · Intervention Types: Drug
Tildacerfont Group 2
Drug
Experimental Group · 1 Intervention: Tildacerfont/Placebo · Intervention Types: Drug
Tildacerfont Group 1
Drug
Experimental Group · 1 Intervention: Tildacerfont/Placebo · Intervention Types: Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Tildacerfont/Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 52 weeks

Who is running the clinical trial?

Spruce BiosciencesLead Sponsor
5 Previous Clinical Trials
186 Total Patients Enrolled
Kyriakie Sarafoglou, M.DPrincipal InvestigatorDept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota

Eligibility Criteria

Age 18 - 55 · All Participants · 4 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

How many healthcare centers in this state are administering this trial?

"Forty-two clinical sites across the United States are actively recruiting patients for this trial. These locations span from Boston and Baltimore to Orange, as well as numerous other areas in between - you should select a site closer to your residence so that travel demands remain low if accepted into the study." - Anonymous Online Contributor

Unverified Answer

Is there still availability for patients to join this experiment?

"According to clinicaltrials.gov, this trial is currently enrolling patients. This study was initially published on August 26th 2020 and has been recently revised November 4th 2022." - Anonymous Online Contributor

Unverified Answer

What end goal is this investigation attempting to achieve?

"The primary efficacy outcome of this 12-week trial is the alteration in androstenedione. Secondary objectives include a safety analysis, measurement of A4 levels below the upper limit of normal values, as well as assessment of any changes to TART(s) lesion volume for male CAH subjects over up to 12 months." - Anonymous Online Contributor

Unverified Answer

For whom is enrollment in this research project open?

"This clinical trial is searching for 72 individuals, aged 18 to 55, diagnosed with congenital adrenal hyperplasia. In addition to this criteria, the participants must be of any gender over 18 years old and have a genetic mutation in CYP21A2 or elevated 17-OHP levels which are treated with hormonal corticosteroids at doses between 15 - 60 milligrams per day. Furthermore, those with salt wasting forms require stable mineralocorticoid replacement treatment periods that exceed 1 month before screening begins." - Anonymous Online Contributor

Unverified Answer

Are individuals aged 25 and over being included in the experimental program?

"The requisite criteria for enrolment in this trial necessitate that participants are aged 18 to 55. Meanwhile, 28 and 105 trials respectively cater to those below 18 or above 65 years of age." - Anonymous Online Contributor

Unverified Answer

How many participants have registered for this experiment?

"Affirmative. According to clinicaltrials.gov, this research is actively seeking participants; the trial was first published on August 26th 2020 and has been recently updated on November 4th 2022. The study seeks a total of 72 individuals from 42 distinct sites." - Anonymous Online Contributor

Unverified Answer

How efficacious is Tildacerfont Group 3 for individuals?

"Our team at Power has assigned the safety of Tildacerfont Group 3 a rating of 2, as there is existing evidence that it is harmless yet no data to affirm its efficacy." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.