A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH (NCT04544410) | Clinical Trial Compass
TerminatedPhase 2
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
Stopped: Study did not meet its primary endpoint.
United States100 participantsStarted 2021-02-22
Plain-language summary
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic Congenital adrenal hyperplasia (CAH) subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
ā. Male and female subjects ā„18 years old at screening
ā. Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-hydroxyprogesterone (17-OHP) and currently treated with hydrocortisone (HC), HC acetate, prednisone, prednisolone, methylprednisolone, dexamethasone (or a combination of the aforementioned glucocorticoid \[GCs\])
ā. Has lower limit of detection ā¤ androstenedione (A4) ā¤ 2.5x upper limit of normal (ULN) at screening measured before a morning GC dose
ā. Has been on a stable, supraphysiologic dose of GC replacement (defined as ā„30 mg/day and ā¤60 mg/day in HCe) for ā„1 month before screening
ā. For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ā„1 month before screening
ā. Agrees to follow contraception guidelines. Male subjects must also agree to refrain from donating sperm throughout the Treatment Period and for 90 days after the last dose of study drug
ā. Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol
Exclusion criteria
ā. Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21-hydroxylase deficiency)
ā. Has a history that includes bilateral adrenalectomy or hypopituitarism
ā
What they're measuring
1
Change in Total Daily GC Dose in Subjects With Classic CAH Over the 24-week, Double Blind, Placebo-Controlled Treatment Period
. Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
ā. Shows clinical signs or symptoms of adrenal insufficiency
ā. Has had a clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening, including but not limited to:
ā. An ongoing malignancy or \<3 years of remission history from any malignancy, other than successfully treated localized skin cancer
ā. eGFR of \<45 mL/min/1.73 m2
ā. Current or history of liver disease (with the exception of Gilbert's syndrome)