Efficacy and Safety of tildacerfont in Reducing Supraphysiologic GC do
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia
IRAS ID
278766
Contact name
Heidi Petersen
Contact email
Sponsor organisation
Spruce Biosciences, Inc.
Eudract number
2019-004765-40
Duration of Study in the UK
6 years, 3 months, days
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 suppress 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 60 participants between 18 and 55 years old are expecting to be enrolled across 55 research sites in the United States of America, Europe and other regions, with an overall participation of around 72 weeks.
The purpose of this study is to evaluate the potential of tildacerfont to reduce the amount of Glucocorticoids (e.g., hydrocortisone) that participants need to take and also to reduce the level of certain hormones in participants bodies.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
20/EE/0271
Date of REC Opinion
27 Jan 2021
REC opinion
Further Information Favourable Opinion