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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04544410
Ethics application status
Date submitted
30/08/2020
Date registered
10/09/2020

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

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 - Blacktown
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 - Parkville
Recruitment postcode(s) [1] 0 0
- Blacktown
Recruitment postcode(s) [2] 0 0
4029 - Brisbane
Recruitment postcode(s) [3] 0 0
- Elizabeth Vale
Recruitment postcode(s) [4] 0 0
- Parkville
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
Country [5] 0 0
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
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Rhode Island
Country [11] 0 0
United States of America
State/province [11] 0 0
South Carolina
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
Brazil
State/province [13] 0 0
Curitiba
Country [14] 0 0
Brazil
State/province [14] 0 0
São Paulo
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
Country [16] 0 0
Canada
State/province [16] 0 0
Quebec
Country [17] 0 0
Estonia
State/province [17] 0 0
Tallinn
Country [18] 0 0
Estonia
State/province [18] 0 0
Tartu
Country [19] 0 0
Germany
State/province [19] 0 0
Munich
Country [20] 0 0
Italy
State/province [20] 0 0
Roma
Country [21] 0 0
Korea, Republic of
State/province [21] 0 0
Seoul
Country [22] 0 0
Latvia
State/province [22] 0 0
Riga
Country [23] 0 0
Lithuania
State/province [23] 0 0
Kaunas
Country [24] 0 0
Poland
State/province [24] 0 0
Kraków
Country [25] 0 0
Poland
State/province [25] 0 0
Warsaw
Country [26] 0 0
Romania
State/province [26] 0 0
Bucharest
Country [27] 0 0
Romania
State/province [27] 0 0
Bucuresti
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Spain
State/province [30] 0 0
Sevilla
Country [31] 0 0
Spain
State/province [31] 0 0
Tarragona
Country [32] 0 0
Sweden
State/province [32] 0 0
Stockholm
Country [33] 0 0
Turkey
State/province [33] 0 0
Istanbul
Country [34] 0 0
United Kingdom
State/province [34] 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
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

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/NCT04544410