Brief Summary
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic CAH subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.
Brief Title
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
Detailed Description
This is a study that will evaluate the ability of Tildacerfont to reduce the glucocorticoid steroid dose used by adult CAH subjects. The first 24-weeks will be a double-blind, placebo controlled, comparison of Tildacerfont vs Placebo. The following 52-weeks will allow all subjects to move to open label Tildacerfont to continue to reduce steroid dose where appropriate, and observe long term safety. Subjects will be offered a long term open label extension up to 240 weeks.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Congenital Adrenal Hyperplasia
Eligibility Criteria
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
* 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
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Keywords
CAH
Adrenal Disorder
Congenital Adrenal Hyperplasia
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT04544410
Org Class
Industry
Org Full Name
Spruce Biosciences
Org Study Id
SPR001-204
Overall Status
Terminated
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official 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
Primary Outcomes
Outcome Description
Absolute change from baseline in GC dose in HCe (mg/day and mg/m2/day) at Week 24
Outcome Measure
Proportion of subjects who can reduce GC dose at Week 24
Outcome Time Frame
24 Weeks
Secondary Ids
Secondary Id
CAHmelia 204
Secondary Outcomes
Outcome Description
Proportion of subjects with GC dose ≤11mg/m2/day in HCe and A4 ≤1.2x baseline or A4 ≤ ULN at Week 24
Outcome Time Frame
24 weeks
Outcome Measure
Percentage change in GC use in subjects with CAH
Outcome Description
Proportion of subjects with baseline GC dose ≤ 35mg HCe who achieve GC dose ≤11 mg/m2/day in HCe and A4 ≤ 1.2x baseline or ≤ ULN at Week 24
Outcome Time Frame
24 Weeks
Outcome Measure
Change in the median cumulative HCe dose in subjects with CAH
Outcome Description
Proportion of subjects with improvement in at least one cardiovascular risk factor at week 24
Outcome Time Frame
24 Weeks
Outcome Measure
Effectiveness in reducing cardiovascular risk in subjects with CAH
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Smita Abraham
Investigator Email
smabraham@montefiore.org
Investigator Phone
240-460-2096