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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04544410




Registration number
NCT04544410
Ethics application status
Date submitted
30/08/2020
Date registered
10/09/2020
Date last updated
24/01/2024

Titles & IDs
Public title
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
Scientific 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
Secondary ID [1] 0 0
CAHmelia 204
Secondary ID [2] 0 0
SPR001-204
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 - Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks

Placebo Comparator: Placebo - Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 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
Proportion of subjects who can reduce GC dose at Week 24
Timepoint [1] 0 0
24 Weeks
Secondary outcome [1] 0 0
Percentage change in GC use in subjects with CAH
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Change in the median cumulative HCe dose in subjects with CAH
Timepoint [2] 0 0
24 Weeks
Secondary outcome [3] 0 0
Effectiveness in reducing cardiovascular risk in subjects with CAH
Timepoint [3] 0 0
24 Weeks
Secondary outcome [4] 0 0
Effectiveness in improving HOMA-IR in subjects with CAH
Timepoint [4] 0 0
24 Weeks
Secondary outcome [5] 0 0
Effect on body weight in subjects with CAH
Timepoint [5] 0 0
24 weeks
Secondary outcome [6] 0 0
Effect on body weight in subjects with CAH
Timepoint [6] 0 0
52 weeks

Eligibility
Key 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
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

- 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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/09/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2029
Actual
Sample size
Target
90
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Spruce Study Site - Brisbane
Recruitment hospital [2] 0 0
Spruce Study Site - Camperdown
Recruitment hospital [3] 0 0
Spruce Study Site - Melbourne
Recruitment hospital [4] 0 0
Spruce Study Site - Sydney
Recruitment postcode(s) [1] 0 0
4029 - Brisbane
Recruitment postcode(s) [2] 0 0
- Camperdown
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- Sydney
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
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
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United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
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United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
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Oregon
Country [10] 0 0
United States of America
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Pennsylvania
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United States of America
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Rhode Island
Country [12] 0 0
United States of America
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South Carolina
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United States of America
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Texas
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Brazil
State/province [14] 0 0
Curitiba
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Brazil
State/province [15] 0 0
São Paulo
Country [16] 0 0
Canada
State/province [16] 0 0
Ontario
Country [17] 0 0
Canada
State/province [17] 0 0
Quebec
Country [18] 0 0
Estonia
State/province [18] 0 0
Tallinn
Country [19] 0 0
Estonia
State/province [19] 0 0
Tartu
Country [20] 0 0
Germany
State/province [20] 0 0
Munich
Country [21] 0 0
Italy
State/province [21] 0 0
Roma
Country [22] 0 0
Korea, Republic of
State/province [22] 0 0
Seoul
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Latvia
State/province [23] 0 0
Riga
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Lithuania
State/province [24] 0 0
Kaunas
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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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Romania
State/province [28] 0 0
Bucuresti
Country [29] 0 0
Spain
State/province [29] 0 0
Barcelona
Country [30] 0 0
Spain
State/province [30] 0 0
Madrid
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Spain
State/province [31] 0 0
Sevilla
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Spain
State/province [32] 0 0
Tarragona
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Sweden
State/province [33] 0 0
Stockholm
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Turkey
State/province [34] 0 0
Istanbul
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Birmingham

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

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04544410
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ron Newfield, M.D
Address 0 0
Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04544410