This is a randomized, double-blind, placebo-controlled study evaluating the potential of Tildacerfont to reduce the use of glucocorticoids (GCs) in adult subjects with congenital adrenal hyperplasia (CAH) receiving GC therapy at supraphysiological doses.
Descrizione riassuntiva dello studio
Congenital adrenal hyperplasia (CAH) is a severe, chronically debilitating, and life-threatening genetic disorder characterized by impaired adrenal synthesis of cortisol and a subsequent overproduction of adrenal androgens. Cortisol deficiency leads to hypersecretion or overproduction of certain hormones, ultimately resulting in an excess of androgens. An excess of androgens can lead to irregular menstruation, absence of menstrual periods (amenorrhea), hirsutism, and virilization in women; testicular adrenal rest tumors (TARTs) in men; and increased sebum production, acne, altered blood pressure profiles in the afternoon, and impaired fertility in both sexes. The current standard of care for CAH is long-term use of glucocorticoids (GCs) at supraphysiological doses to replace the missing cortisol and suppress androgen overproduction. This is a problematic therapy with significant side effects, and a non-steroidal treatment option would be preferable. Tildacerfont may allow a CAH patient to have normal androgen levels while taking GC in the normal replacement range. Currently, Tildacerfont has shown an acceptable safety profile at effective doses in non-clinical toxicology studies, phase 1 clinical studies in healthy volunteers, and phase 2 studies in adult patients with classic CAH. The SPR001-204 study will be the first study of Tildacerfont evaluating its ability to reduce the necessary GC dose. Neither the patients nor their investigator physician know which treatment the patients are receiving, and patients will be selected, e.g., by coin toss, to receive either the study drug or the placebo. An optional open-label extension period provides additional treatment with Tildacerfont at 200 mg QD for up to 240 weeks.
(BASEC)
Intervento studiato
The main purpose of this study is to investigate the drug SPR001 (Tildacerfont) for patients with classic congenital adrenal hyperplasia (CAH). SPR001 is an investigational drug, i.e., a drug being tested. The efficacy and safety of SPR001 (Tildacerfont) in reducing the supraphysiological (higher or more effective than naturally occurring dose) use of glucocorticoids in adult patients with classic CAH will be evaluated. The study consists of a two-part treatment phase. During the first 24 weeks of the treatment period, patients will be randomized in a 1:1 ratio, e.g., by coin toss. They will receive either placebo or Tildacerfont at 200 mg once daily. During the remaining 52 weeks of the treatment period, all subjects will receive 200 mg of Tildacerfont once daily.
(BASEC)
Malattie studiate
Congenital adrenal hyperplasia (CAH) is a group of rare disorders. There is a deficiency of one of the enzymes needed to produce certain hormones. CAH affects the adrenal glands, which are located on top of each kidney.
(BASEC)
Men and women ≥ 18 years old at screening (or: at the pre-examination)\n\nHas a known childhood diagnosis of classic CAH based on a genetic CYP21A2 mutation and/or elevated 17-OHP (17α-hydroxyprogesterone) and is currently being treated with GCs (glucocorticoids)\n\nHas received a stable supraphysiological dose of GC for ≥ 1 month prior to screening (BASEC)
Criteri di esclusione
Has a known/suspected diagnosis of another form of CAH\n\nHistory of bilateral adrenalectomy/pituitary insufficiency or allergy/hypersensitivity to the study drug\n\nShows clinical signs/symptoms of adrenal insufficiency (BASEC)
Luogo dello studio
San Gallo, Zurigo
(BASEC)
Sponsor
Medpace Switzerland AG
(BASEC)
Contatto per ulteriori informazioni sullo studio
Persona di contatto in Svizzera
Pamela Wedel
+1 415 655 4169
pwedel@cluttersprucebiosciences.comSpruce Bioscienses, Inc.
(BASEC)
Informazioni generali
Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
(ICTRP)
Informazioni scientifiche
Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
(ICTRP)
Nome del comitato etico approvante (per studi multicentrici solo il comitato principale)
Commissione etica Zurigo
(BASEC)
Data di approvazione del comitato etico
19.10.2022
(BASEC)
ID di studio ICTRP
NCT04544410 (ICTRP)
Titolo ufficiale (approvato dal comitato etico)
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 (BASEC)
Titolo accademico
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 (ICTRP)
Titolo pubblico
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH (ICTRP)
Malattie studiate
Congenital Adrenal Hyperplasia (ICTRP)
Intervento studiato
Drug: Tildacerfont/Placebo (ICTRP)
Tipo di studio
Interventional (ICTRP)
Disegno dello studio
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator). (ICTRP)
Criteri di inclusione/esclusione
Inclusion Criteria:
- Male and female subjects over 18 years old, inclusive
- Has a documented historical diagnosis of classic CAH due to 21-hydroxylase
deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP
and currently treatment with HC, HC acetate, prednisone, prednisolone,
methylprednisolone (or a combination of the aforementioned GCs)
- Has been on a stable, supraphysiologic dose of GC replacement for =1 month before
screening.
- For subjects with the salt-wasting form of CAH, subject has been on a stable dose of
mineralocorticoid replacement for =1 month before screening
Exclusion Criteria:
- Has a known or suspected diagnosis of any other known form of classic CAH (not due
to 21-hydroxylase deficiency)
- Has a history that includes bilateral adrenalectomy or hypopituitarism
- Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients,
or any other CRF1 receptor antagonist
- Shows clinical signs or symptoms of adrenal insufficiency (ICTRP)
non disponibile
Endpoint primari e secondari
Proportion of subjects who can reduce GC dose at Week 24 (ICTRP)
Percentage change in GC use in subjects with CAH;Change in the median cumulative HCe dose in subjects with CAH;Effectiveness in reducing cardiovascular risk in subjects with CAH (ICTRP)
Data di registrazione
non disponibile
Inclusione del primo partecipante
non disponibile
Sponsor secondari
non disponibile
Contatti aggiuntivi
Ron Newfield, M.D, Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine. (ICTRP)
ID secondari
CAHmelia 204, SPR001-204 (ICTRP)
Risultati-Dati individuali dei partecipanti
non disponibile
Ulteriori informazioni sullo studio
https://clinicaltrials.gov/ct2/show/NCT04544410 (ICTRP)
Risultati dello studio
Riepilogo dei risultati
spruce-spr001-204-csr-summary-v1-0-27jun25-de.pdfLink ai risultati nel registro primario
non disponibile