Efficacy and Safety of Tildacerfont in Adult Subjects with Classic Con
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
IRAS ID
278765
Contact name
Alessandro Prete
Contact email
Sponsor organisation
Spruce Biosciences, Inc.
Eudract number
2019-004764-22
Duration of Study in the UK
6 years, 5 months, days
Research summary
Research Summary
Congenital adrenal hyperplasia (CAH) is an inherited genetic disorder that affects the adrenal glands, a pair of walnut sized organs above the kidneys.
The adrenal glands are responsible for producing three different types of hormones: glucocorticoids (to counteract stress), mineralocorticoids (to control the amount of salt and water in the body) and male sex hormones (also known as androgens). CAH is characterised by deficiency of certain types of enzymes that will impair the production of glucocorticoids. This ultimately results in overproduction of male sex hormones (androgens) in order to compensate for the loss.
CAH is a serious, chronically debilitating and life threatening condition. The current standard of care for CAH is lifelong use of glucocorticoids (GCs) to replace the deficient hormone and supress androgen overproduction. However, these can have significant side effects and do not always work well in treating CAH. A non-steroidal treatment option that helps control adrenal hormone levels is much warranted and would be beneficial to CAH patients.
Tildacerfont (SPR001) is a small molecule drug being developed by Spruce Biosciences Inc. for the treatment of CAH. The study drug blocks the feedback system in the body which is responsible for increased adrenal androgens in CAH patients.
This is a Phase II study. Approximately 72 participants between 18 and 55 years old are expecting to be enrolled across 40 research sites in the United States of America, Europe and other regions, with an overall participation of around 66 weeks.
The purpose of this study is to evaluate the safety and efficacy of the study drug in CAH patients.
Summary of Results
The main goal of the study was to see if tildacerfont reduced the amount of the hormone A4 in participants’ blood. To assess it, the amount of A4 in participants’ blood was tested during the 12 weeks of double-blind treatment in Part A. The results showed after the 12 weeks of treatment there were no significant changes from baseline A4 levels of participants taking tildacerfont, when compared to A4 levels seen in participants taking placebo. This happened in the 3 different doses of tildacerfont tested (50, 100, or 200 mg once a day). In addition, during the 12 weeks of treatment in Part B and the 46 weeks of treatment in Part C, no significant changes were seen in any of the other effectiveness measurements tested.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
20/WM/0277
Date of REC Opinion
23 Dec 2020
REC opinion
Further Information Favourable Opinion