A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH

Congenital Adrenal Hyperplasia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04544410
• Other study ID #: SPR001-204
• Secondary ID: CAHmelia 204
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: September 29, 2020
• Est. completion date: September 2029

Study information

• Verified date: January 2024
• Source: Spruce Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 90
• Est. completion date: September 2029
• Est. primary completion date: September 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Drug:
• Tildacerfont/Placebo
Tablet, administered daily

Locations

Country	Name	City	State
Australia	Spruce Study Site	Brisbane
Australia	Spruce Study Site	Camperdown
Australia	Spruce Study Site	Melbourne
Australia	Spruce Study Site	Sydney
Brazil	Spruce Study Site	Curitiba
Brazil	Spruce Study Site	São Paulo
Canada	Spruce Study Site	Ottawa	Ontario
Canada	Spruce Study Site	Sherbrooke	Quebec
Estonia	Spruce Study Site	Tallinn
Estonia	Spruce Study Site	Tartu
Germany	Spruce Study Site	Munich
Italy	Spruce Study Site	Roma
Korea, Republic of	Spruce Study Site	Seoul
Latvia	Spruce Study Site	Riga
Lithuania	Spruce Study Site	Kaunas
Poland	Spruce Study Site	Kraków
Poland	Spruce Study Site	Warsaw
Romania	Spruce Study Site	Bucharest
Romania	Spruce Study Site	Bucuresti
Spain	Spruce Study Site	Barcelona
Spain	Spruce Study Site	Madrid
Spain	Spruce Study Site	Sevilla
Spain	Spruce Study Site	Tarragona
Sweden	Spruce Study Site	Stockholm
Turkey	Spruce Study Site	Istanbul
United Kingdom	Spruce Study Site	Birmingham
United States	Spruce Biosciences Clinical Site	Ann Arbor	Michigan
United States	Spruce Study Site	Baltimore	Maryland
United States	Spruce Study Site	Bend	Oregon
United States	Spruce Study Site	Birmingham	Alabama
United States	Spruce Study Site	Canton	Ohio
United States	Spruce Study Site	Cincinnati	Ohio
United States	Spruce Study Site	Cleveland	Ohio
United States	Spruce Study Site	Columbia	South Carolina
United States	Spruce Study Site	Columbus	Ohio
United States	Spruce Study Site	Dallas	Texas
United States	Spruce Study Site	Fort Worth	Texas
United States	Spruce Study Site	Indianapolis	Indiana
United States	Spruce Study Site	Los Angeles	California
United States	Spruce Study Site	Minneapolis	Minnesota
United States	Spruce Study Site	New Brunswick	New Jersey
United States	Spruce Clinical Site	Orange	California
United States	Spruce Study Site	Philadelphia	Pennsylvania
United States	Spruce Study Site	Philadelphia	Pennsylvania
United States	Spruce Study Site	Philadelphia	Pennsylvania
United States	Spruce Study Site	Providence	Rhode Island
United States	Spruce Study Site	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Spruce Biosciences

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  Canada,  Estonia,  Germany,  Italy,  Korea, Republic of,  Latvia,  Lithuania,  Poland,  Romania,  Spain,  Sweden,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of subjects who can reduce GC dose at Week 24	Proportion of subjects with at least a 5 mg/day HCe reduction from baseline in GC dose and A4 =ULN at Week 24	24 Weeks
Secondary	Percentage change in GC use in subjects with CAH	Percent change from baseline in GC dose at week 24	24 weeks
Secondary	Change in the median cumulative HCe dose in subjects with CAH	Median total cumulative GC dose in HCe at Week 24	24 Weeks
Secondary	Effectiveness in reducing cardiovascular risk in subjects with CAH	Proportion of subjects with improvement in at least one cardiovascular risk factor at week 24	24 Weeks
Secondary	Effectiveness in improving HOMA-IR in subjects with CAH	Change from baseline in the HOMA-IR at Week 24	24 Weeks
Secondary	Effect on body weight in subjects with CAH	Percent change from baseline in body weight after 24 weeks of tildacerfont treatment	24 weeks
Secondary	Effect on body weight in subjects with CAH	Percent change from baseline in body weight after 52 weeks of tildacerfont treatment	52 weeks