Clinical trial NCT04544410: A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Congenital Adrenal Hyperplasia
Keywords: CAH
Adrenal Disorder
Congenital Adrenal Hyperplasia
Lead sponsor: Spruce Biosciences
Sponsor class: INDUSTRY
First posted: 2020-09-10
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Subjects will be randomized in a 1:1 manner to either Tildacerfont or Placebo for 24 weeks followed by 52 weeks open label Tildacerfont
Primary purpose: TREATMENT

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.

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.

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

Primary outcomes:
measure: Proportion of subjects who can reduce GC dose at Week 24
timeFrame: 24 Weeks
Secondary outcomes:
measure: Percentage change in GC use in subjects with CAH
timeFrame: 24 weeks
measure: Change in the median cumulative HCe dose in subjects with CAH
timeFrame: 24 Weeks
measure: Effectiveness in reducing cardiovascular risk in subjects with CAH
timeFrame: 24 Weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Spruce Study Site
state: Alabama
city: Los Angeles
country: United States
facility: Spruce Study Site
state: California
city: San Diego
country: United States
facility: Spruce Study Site
state: California
city: Indianapolis
country: United States
facility: Spruce Study Site
state: Indiana
city: Baltimore
country: United States
facility: Spruce Study Site
state: Maryland
city: Ann Arbor
country: United States
facility: Spruce Biosciences Clinical Site
state: Michigan
city: Minneapolis
country: United States
facility: Spruce Study Site
state: Minnesota
city: New Brunswick
country: United States
facility: Spruce Study Site
state: New Jersey
city: Canton
country: United States
facility: Spruce Study Site
state: Ohio
city: Cincinnati
country: United States
facility: Spruce Study Site
state: Ohio
city: Cleveland
country: United States
facility: Spruce Study Site
state: Ohio
city: Columbus
country: United States
facility: Spruce Study Site
state: Ohio
city: Philadelphia
country: United States
facility: Spruce Study Site
state: Pennsylvania
city: Philadelphia
country: United States
facility: Spruce Study Site
state: Pennsylvania
city: Philadelphia
country: United States
facility: Spruce Study Site
state: Pennsylvania
city: Providence
country: United States
facility: Spruce Study Site
state: Rhode Island
city: Columbia
country: United States
facility: Spruce Study Site
state: South Carolina
city: Fort Worth
country: United States
facility: Spruce Study Site
state: Texas
city: Blacktown
country: Australia
facility: Spruce Study Site
city: Brisbane
country: Australia
facility: Spruce Study Site
city: Elizabeth Vale
country: Australia
facility: Spruce Study Site
city: Parkville
country: Australia
facility: Spruce Study Site
city: Curitiba
country: Brazil
facility: Spruce Study Site
city: São Paulo
country: Brazil
facility: Spruce Study Site
city: Ottawa
country: Canada
facility: Spruce Study Site
state: Ontario
city: Sherbrooke
country: Canada
facility: Spruce Study Site
state: Quebec
city: Tallinn
country: Estonia
facility: Spruce Study Site
city: Tartu
country: Estonia
facility: Spruce Study Site
city: Munich
country: Germany
facility: Spruce Study Site
city: Roma
country: Italy
facility: Spruce Study Site
city: Seoul
country: Korea, Republic of
facility: Spruce Study Site
city: Riga
country: Latvia
facility: Spruce Study Site
city: Kaunas
country: Lithuania
facility: Spruce Study Site
city: Kraków
country: Poland
facility: Spruce Study Site
city: Warsaw
country: Poland
facility: Spruce Study Site
city: Bucharest
country: Romania
facility: Spruce Study Site
city: Bucuresti
country: Romania
facility: Spruce Study Site
city: Barcelona
country: Spain
facility: Spruce Study Site
city: Madrid
country: Spain
facility: Spruce Study Site
city: Sevilla
country: Spain
facility: Spruce Study Site
city: Tarragona
country: Spain
facility: Spruce Study Site
city: Stockholm
country: Sweden
facility: Spruce Study Site
city: Istanbul
country: Turkey
facility: Spruce Study Site
city: Birmingham
country: United Kingdom
facility: Spruce Study Site
Study officials:
affiliation: Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
name: Ron Newfield, M.D
role: PRINCIPAL_INVESTIGATOR
References: