Clinical trial NCT04457336: A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
Official title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) 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: 2020-07-07
Enrollment: 96
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 1:1:1:1 manner to one of three doses of Tildacerfont or Placebo.
Primary purpose: TREATMENT

Brief 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.

Detailed description: This is a study that will test the efficacy and safety of Tildacerfont. The first 12-weeks will be a double-blind, placebo controlled, dose ranging study. The following 58-weeks will assess the long term safety of Tildacerfont. Optional open label treatment extension period up to 240 weeks with 200mg Tildacerfont QD.

Eligibility criteria: Inclusion Criteria:

* Male and female subjects over 18 years old, inclusive
* Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented 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 ≥15 mg/day and ≤60 mg/day in HC equivalents
* 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
* Current treatment with dexamethasone as GC therapy for CAH. Prior treatment with dexamethasone is allowed as long as the transition to an alternative GC regimen (eg, HC, prednisone, or prednisolone) has resulted in a stable dose of GC replacement for ≥1 month before screening.
* Shows clinical signs or symptoms of adrenal insufficiency

Primary outcomes:
measure: Change in androstenedione
timeFrame: 12 weeks
Secondary outcomes:
measure: Proportion of subjects who achieve reduction A4 levels
timeFrame: 12 weeks
measure: Proportion of subjects who achieve reduction in 17-OHP
timeFrame: 12 weeks
measure: Effectiveness in reducing TART(s) in Male CAH subjects
timeFrame: 12 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: Orange
country: United States
facility: Spruce Clinical Site
state: California
city: Sacramento
country: United States
facility: Spruce Study Site
state: California
city: Englewood
country: United States
facility: Spruce Study Site
state: Colorado
city: Tampa
country: United States
facility: Spruce Clinical Site
state: Florida
city: West Palm Beach
country: United States
facility: Spruce Study Site
state: Florida
city: Chicago
country: United States
facility: Spruce Study Site
state: Illinois
city: Indianapolis
country: United States
facility: Spruce Clinical Site
state: Indiana
city: Baltimore
country: United States
facility: Spruce Study Site
state: Maryland
city: Minneapolis
country: United States
facility: Spruce Clinical Site
state: Minnesota
city: Rochester
country: United States
facility: Spruce Study Site
state: Minnesota
city: Las Vegas
country: United States
facility: Spruce Clinical Site
state: Nevada
city: Hickory
country: United States
facility: Spruce Study Site
state: North Carolina
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: Bend
country: United States
facility: Spruce Clinical Site
state: Oregon
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: Memphis
country: United States
facility: Spruce Clinical Site
state: Tennessee
city: Austin
country: United States
facility: Spruce Study Site
state: Texas
city: Dallas
country: United States
facility: Spruce Study Site
state: Texas
city: Edinburg
country: United States
facility: Spruce Clinical Site
state: Texas
city: Fort Worth
country: United States
facility: Spruce Clinical Site
state: Texas
city: Nedlands
country: Australia
facility: Spruce Study Site
state: Western Australia
city: Brisbane
country: Australia
facility: Spruce study site
city: Elizabeth Vale
country: Australia
facility: Spruce Study Site
city: Melbourne
country: Australia
facility: Spruce Study Site
city: Brasília
country: Brazil
facility: Spruce Study Site
city: São Paulo
country: Brazil
facility: Spruce Study Site
city: St. John's
country: Canada
facility: Spruce Study Site
state: Newfoundland and Labrador
city: London
country: Canada
facility: Spruce Study Site
state: Ontario
city: Ottawa
country: Canada
facility: Spruce Study Site
city: Sherbrooke
country: Canada
facility: Spruce Study Site
city: Aarhus
country: Denmark
facility: Spruce Study Site
city: Copenhagen
country: Denmark
facility: Spruce Study Site
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: Dublin
country: Ireland
facility: Spruce Study Site
city: Milan
country: Italy
facility: Spruce Study Site
city: Napoli
country: Italy
facility: Spruce Study Site
city: Rome
country: Italy
facility: Spruce Study Site
city: Torino
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: Nijmegen
country: Netherlands
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: 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: Falun
country: Sweden
facility: Spruce Study Site
city: Stockholm
country: Sweden
facility: Spruce Study Site
city: Saint Gallen
country: Switzerland
facility: Spruce Study Site
city: Zürich
country: Switzerland
facility: Spruce Study Site
city: Istanbul
country: Turkey
facility: Spruce Study Site
city: Birmingham
country: United Kingdom
facility: Spruce Study Site
Study officials:
affiliation: Dept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota
name: Kyriakie Sarafoglou, M.D
role: PRINCIPAL_INVESTIGATOR
References: