Classic Congenital Adrenal Hyperplasia MedDRA version: 20.0 Level: LLT Classification code 10010323 Term: Congenital adrenal hyperplasia System Organ Class: 100000004850
Conditions
Sponsors
Spruce Biosciences, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Male and female subjects = 18 years old at screening 2.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 combinaton of the aforementionned GCs) 3.Has LLD = A4 = 2.5x ULN at screening measured before an am GC dose 4. Has been on a stable, supraphysiologic dose of GC replacement (defined as =30 mg/day and =60 mg/day in HCe) for =1 month before screening 5.For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for =1 month before screening 6. 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. 7.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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 85 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: 1.Has a known or suspected diagnosis of any other known form of classic CAH (not due to 21 hydroxylase deficiency) 2.Has a history that includes bilateral adrenalectomy or hypopituitarism 3.Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist 4.Shows clinical signs or symptoms of adrenal insufficiency 5.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: a.An ongoing malignancy or 12 for either depression or anxiety at screening or baseline 7.Has clinically significant abnormal ECG or clinical laboratory results. 8. Routinely works overnight shifts 9.Subjects with travel plans/work schedules that result in significant and frequent changes in time zones (>2 hours) will require Medical Monitor approval for enrollment. 10.Females who are pregnant or nursing 11.Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study 12.Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before the start of the Treatment Period to the end of the study a. Rosiglitazone, aromatase inhibitors, testosterone, or growth hormones, or any other medication or supplement that could impact subject safety or confound interpretation of study results b. The drugs listed in Section 13.1 13.Donation or receipt of blood from 90 days before Screening to the end of the study; donation or receipt of platelets, white blood cells, or plasma from 30 days before Screening to the end of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the proportion of subjects who can reduce GC use in subjects with CAH;Secondary Objective: -To evaluate the percentage change in GC use in subjects with CAH -To evaluate the effect of tildacerfont in reducing the median cumulative HCe dose -To evaluate the effect of tildacerfont in reducing cardiovascular risk in subjects with CAH -To evaluate the effect of tildacerfont in improving homeostatic model assessment of insulin resistance (HOMA-IR) in subjects with CAH -To evaluate the effect of tildacerfont on body weight after 24 weeks in subjects with CAH -To evaluate the effect of tildacerfont on body weight after 52 weeks of tildacerfont treatment in subjects with CAH;Primary end point(s): Proportion of subjects with at least a 5mg/day HCe reduction from baseline in GC dose and A4 = ULN at Week 24;Timepoint(s) of evaluation of this end point: Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Percent change from baseline in GC dose at Week 24 -Median total cumulative GC dose in HCe at Week 24 -Proportion of subjects with improvement in at least one cardiovascular risk factor at Week 24 -Change from baseline in the HOMA-IR at Week 24 -Percent change from baseline in body weight at Week 24 -Percent change from baseline in body weight after 52 weeks of tildacerfont treatment;Timepoint(s) of evaluation of this end point: Week 24 and Week 52 | — |
Countries
Australia, Canada, Denmark, Estonia, Germany, Italy, Latvia, Lithuania, Netherlands, Poland, Spain, Sweden, Switzerland, Turkey, United Kingdom, United States
Contacts
Public ContactPamela Wedel
Spruce Biosciences, Inc.
Outcome results
None listed