A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH (NCT04457336) | Clinical Trial Compass
TerminatedPhase 2
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
Stopped: Study SPR001-203 did not meet its primary and secondary endpoints therefore Spruce Biosciences has decided to terminate the study
United States96 participantsStarted 2020-08-26
Plain-language summary
An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.
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
✓. Male and female subjects ≥18 years old at screening
✓. 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-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
✓. Has been on a stable, supraphysiologic dose of GC replacement (defined as ≥15 mg/day and ≤60 mg/day in HCe) for ≥1 month before screening
✓. Has A4 \>ULN at both screening and Week 4 (measured before any AM GC dose) if daily GC dose \<30 mg OR has A4 \>2.5x ULN at both screening and Week 4 (measured before any AM GC dose)
✓. For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening
✓. 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.
✓. 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
✕. 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
✕
What they're measuring
1
To Evaluate the Effect of Tildacerfont in Reducing A4 in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Over 12 Weeks
Timeframe: Baseline and 12 weeks of treatment (Week 18)
. Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
✕. Current treatment with dexamethasone as GC therapy for CAH
✕. Is not adherent to GC or study drug dosing regimen during the Run-in Period (defined as taking \<80% of expected doses based on drug accountability)
✕. Shows clinical signs or symptoms of adrenal insufficiency
✕. 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:
✕. An ongoing malignancy or \<3 years of remission history from any malignancy, other than successfully treated localized skin cancer