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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04457336




Registration number
NCT04457336
Ethics application status
Date submitted
25/06/2020
Date registered
7/07/2020

Titles & IDs
Public title
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
Scientific 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
Secondary ID [1] 0 0
CAHmelia 203
Secondary ID [2] 0 0
SPR001-203
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Adrenal Hyperplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tildacerfont/Placebo

Experimental: Tildacerfont Group 1 - Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1

Experimental: Tildacerfont Group 2 - Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2

Experimental: Tildacerfont Group 3 - Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3

Placebo comparator: Placebo - Placebo administered daily via oral tablet for 12 weeks.


Treatment: Drugs: Tildacerfont/Placebo
Tablet, administered daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in androstenedione
Timepoint [1] 0 0
12 weeks
Secondary outcome [1] 0 0
Proportion of subjects who achieve reduction A4 levels
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Proportion of subjects who achieve reduction in 17-OHP
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Effectiveness in reducing TART(s) in Male CAH subjects
Timepoint [3] 0 0
12 weeks

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/08/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/05/2024
Sample size
Target
Accrual to date
Final
96
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Spruce Study Site - Nedlands
Recruitment hospital [2] 0 0
Spruce study site - Brisbane
Recruitment hospital [3] 0 0
Spruce Study Site - Elizabeth Vale
Recruitment hospital [4] 0 0
Spruce Study Site - Melbourne
Recruitment postcode(s) [1] 0 0
6009 - Nedlands
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Elizabeth Vale
Recruitment postcode(s) [4] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
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United States of America
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Florida
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United States of America
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Illinois
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United States of America
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Indiana
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United States of America
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Maryland
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United States of America
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Minnesota
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United States of America
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Nevada
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United States of America
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North Carolina
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United States of America
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Ohio
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United States of America
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Oregon
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United States of America
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Pennsylvania
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Rhode Island
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United States of America
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South Carolina
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United States of America
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Tennessee
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United States of America
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Texas
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Brazil
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Brasília
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Brazil
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São Paulo
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Canada
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Newfoundland and Labrador
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Canada
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Ontario
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Canada
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Ottawa
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Canada
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Sherbrooke
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Denmark
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Aarhus
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Denmark
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Copenhagen
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Estonia
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Tallinn
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Estonia
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Tartu
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Germany
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Munich
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Ireland
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Dublin
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Italy
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Milan
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Italy
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Napoli
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Italy
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Rome
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Italy
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Torino
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Korea, Republic of
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Seoul
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Latvia
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Riga
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Lithuania
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Kaunas
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Netherlands
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Nijmegen
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Poland
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Kraków
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Poland
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Warsaw
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Romania
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Bucharest
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Sevilla
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Spain
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Tarragona
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Sweden
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Falun
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Sweden
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Stockholm
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Switzerland
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Saint Gallen
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Switzerland
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Zürich
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Turkey
State/province [49] 0 0
Istanbul
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United Kingdom
State/province [50] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Spruce Biosciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Kyriakie Sarafoglou, M.D
Address 0 0
Dept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04457336