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NCT04457336 Clinical Trial

A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

Congenital Adrenal Hyperplasia Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data
NCT number NCT04457336
Other study ID # SPR001-203
Secondary ID CAHmelia 203
Status Terminated
Phase Phase 2
First received
Last updated
Start date August 26, 2020
Est. completion date May 23, 2024

Study information
Verified date June 2024
Source Spruce Biosciences
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Terminated
Enrollment 96
Est. completion date May 23, 2024
Est. primary completion date February 9, 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 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

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• Tildacerfont/Placebo
Tablet, administered daily

Locations
Country Name City State
Australia Spruce study site Brisbane
Australia Spruce Study Site Elizabeth Vale
Australia Spruce Study Site Melbourne
Australia Spruce Study Site Nedlands Western Australia
Brazil Spruce Study Site Brasília
Brazil Spruce Study Site São Paulo
Canada Spruce Study Site London Ontario
Canada Spruce Study Site Ottawa
Canada Spruce Study Site Sherbrooke
Canada Spruce Study Site St. John's Newfoundland and Labrador
Denmark Spruce Study Site Aarhus
Denmark Spruce Study Site Copenhagen
Estonia Spruce Study Site Tallinn
Estonia Spruce Study Site Tartu
Germany Spruce Study Site Munich
Ireland Spruce Study Site Dublin
Italy Spruce Study Site Milan
Italy Spruce Study Site Napoli
Italy Spruce Study Site Rome
Italy Spruce Study Site Torino
Korea, Republic of Spruce Study Site Seoul
Latvia Spruce Study Site Riga
Lithuania Spruce Study Site Kaunas
Netherlands Spruce Study Site Nijmegen
Poland Spruce Study Site Kraków
Poland Spruce Study Site Warsaw
Romania Spruce Study Site Bucharest
Spain Spruce Study Site Barcelona
Spain Spruce Study Site Madrid
Spain Spruce Study Site Sevilla
Spain Spruce Study Site Tarragona
Sweden Spruce Study Site Falun
Sweden Spruce Study Site Stockholm
Switzerland Spruce Study Site Saint Gallen
Switzerland Spruce Study Site Zürich
Turkey Spruce Study Site Istanbul
United Kingdom Spruce Study Site Birmingham
United States Spruce Study Site Austin Texas
United States Spruce Study Site Baltimore Maryland
United States Spruce Clinical Site Bend Oregon
United States Spruce Study Site Birmingham Alabama
United States Spruce Study Site Canton Ohio
United States Spruce Study Site Chicago Illinois
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 Clinical Site Edinburg Texas
United States Spruce Study Site Englewood Colorado
United States Spruce Clinical Site Fort Worth Texas
United States Spruce Study Site Hickory North Carolina
United States Spruce Clinical Site Indianapolis Indiana
United States Spruce Clinical Site Las Vegas Nevada
United States Spruce Study Site Los Angeles California
United States Spruce Clinical Site Memphis Tennessee
United States Spruce Clinical Site Minneapolis Minnesota
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 Providence Rhode Island
United States Spruce Study Site Rochester Minnesota
United States Spruce Study Site Sacramento California
United States Spruce Clinical Site Tampa Florida
United States Spruce Study Site West Palm Beach Florida

Sponsors (1)
Lead Sponsor Collaborator
Spruce Biosciences

Countries where clinical trial is conducted

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

Outcome
Type Measure Description Time frame Safety issue
Primary Change in androstenedione Percent change of androstenedione 12 weeks
Secondary Proportion of subjects who achieve reduction A4 levels Proportion of subjects who achieve A4 = ULN 12 weeks
Secondary Proportion of subjects who achieve reduction in 17-OHP Proportion of subjects who achieve 17-OHP= 1200ng/dL 12 weeks
Secondary Effectiveness in reducing TART(s) in Male CAH subjects Change in lesion volume of TART(s) from baseline 12 weeks
See also
  Status Clinical Trial Phase
Completed NCT03687242 - Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia Phase 2
Active, not recruiting NCT04544410 - A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH Phase 2
Not yet recruiting NCT04087148 - Linear Growth of Children With Congenital Adrenal Hyperplasia
Completed NCT03162159 - Adult Height Prediction in Congenital Adrenal Hyperplasia
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Enrolling by invitation NCT05162950 - Effects and Importance of Epinephrine/Adrenalin Deficiency in CAH
Withdrawn NCT03532022 - Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy Phase 3
Recruiting NCT02795871 - Prenatal Dex Study N/A
Completed NCT02934399 - Dynamic Hormone Diagnostics in Endocrine Disease
Recruiting NCT04903587 - Gonadal Changes In Congenital Adrenal Hyperplasia Patients
Not yet recruiting NCT04536662 - Comparisons of Different Forms of Glucocorticoid on the Recovery of Reproductive Function in Patients With 21α-hydroxylase Deficiency Phase 4
Recruiting NCT04463316 - GROWing Up With Rare GENEtic Syndromes
Active, not recruiting NCT04490915 - Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia Phase 3
Completed NCT01807364 - Cardiovascular Risk Profile in Patients With Congenital Adrenal Hyperplasia N/A
Completed NCT02804178 - A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia Phase 2
Completed NCT03019614 - An Open Label Study in Healthy Volunteers to Compare Chronocort® to Hydrocortisone Phase 1
Not yet recruiting NCT04293133 - Final Height in Patients With CAH
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Recruiting NCT05663320 - A Study of a Virtual Education-Based Transition Intervention to Improve Transition Readiness in Adolescent and Young Adults With Congenital Adrenal Hyperplasia N/A
Active, not recruiting NCT04806451 - Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study) Phase 3