A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
NCT ID: NCT04457336
Last Updated: 2025-07-08
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
96 participants
INTERVENTIONAL
2020-08-26
2024-05-23
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Tildacerfont Group 1
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 1
Tildacerfont/Placebo
Tablet, administered daily
Tildacerfont Group 2
Tildacerfont administered daily via oral tablet for 12 weeks at dose level 2
Tildacerfont/Placebo
Tablet, administered daily
Tildacerfont Group 3
Tildacerfont administered daily via oral tablet for 70 weeks at dose level 3
Tildacerfont/Placebo
Tablet, administered daily
Placebo
Placebo administered daily via oral tablet for 12 weeks.
Tildacerfont/Placebo
Tablet, administered daily
Interventions
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Tildacerfont/Placebo
Tablet, administered daily
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Has a known childhood diagnosis of classic CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-OHP and currently treated with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned GCs)
3. Has been on a stable, supraphysiologic dose of GC replacement (defined as ≥15 mg/day and ≤60 mg/day in HCe) for ≥1 month before screening
4. Has A4 \>ULN at both screening and Week 4 (measured before any AM GC dose) if daily GC dose \<30 mg OR has A4 \>2.5x ULN at both screening and Week 4 (measured before any AM GC dose)
5. For subjects with the salt-wasting form of CAH, subject has been on a stable dose of mineralocorticoid replacement for ≥1 month before screening
6. Agrees to follow contraception guidelines. Male subjects must also agree to refrain from donating sperm throughout the treatment period and for 90 days after the last dose of study drug.
7. Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol.
Exclusion Criteria
2. Has a history that includes bilateral adrenalectomy or hypopituitarism
3. Has a history of allergy or hypersensitivity to tildacerfont, any of its excipients, or any other CRF1 receptor antagonist
4. Current treatment with dexamethasone as GC therapy for CAH
a. 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.
5. Is not adherent to GC or study drug dosing regimen during the Run-in Period (defined as taking \<80% of expected doses based on drug accountability)
6. Shows clinical signs or symptoms of adrenal insufficiency
7. Has had a clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening, including but not limited to:
1. An ongoing malignancy or \<3 years of remission history from any malignancy, other than successfully treated localized skin cancer
2. eGFR of \<45 mL/min/1.73 m2
3. Current or history of liver disease (with the exception of Gilbert's syndrome).
4. History of alcohol or substance abuse within the last year, or any significant history of alcohol or substance abuse that would likely prevent the subject from reliably participating in the study, based on the opinion of the Investigator
5. Active hepatitis B, hepatitis C, or HIV at screening
6. Subjects who plan to undergo bariatric surgery during the study are excluded.
7. Any other condition that would impact subject safety or confound interpretation of study results
8. Psychiatric conditions, including but not limited to bipolar disorder, schizophrenia, or schizoaffective disorders that are not effectively controlled on medication and may have an adverse impact on study compliance. Symptoms including hallucinations, delusions, and psychosis are exclusionary. Additionally:
1. Increased risk of suicide based on the Investigator's judgment or the results of the C-SSRS conducted at screening and Week 6 (eg, C-SSRS Type 3, 4, or 5 ideation within the past 6 months or any suicidal behavior within the past 12 months)
2. HADS score \>12 for either depression or anxiety at screening or Week 6
9. Has clinically significant abnormal ECG or clinical laboratory results. Abnormal results that must be reviewed and discussed with the Medical Monitor to determine eligibility for this study include but are not limited to:
1. Any clinically meaningful abnormal ECG results, including QTcF \>450 ms for male participants or \>470 ms for female participants
2. ALT \>2x ULN
3. Total bilirubin \>1.5x ULN
4. Total bile acids \>5x ULN
10. Routinely works overnight shifts
11. Subjects with travel plans/work schedules that result in significant and frequent changes in time zones (\>2 hours) will require Medical Monitor approval for enrollment.
12. Females who are pregnant or nursing
13. Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study
14. Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before Day 1 to the end of the study:
1. Rosiglitazone, aromatase inhibitors, testosterone, growth hormones, or any other medication or supplement that could impact subject safety or confound interpretation of study results
2. The following drugs:
i. Moderate to strong inhibitors and/or inducers of CYP3A4 ii. Sensitive substrates or narrow-therapeutic-range substrates of CYP3A4 (except hormonal contraception containing ≤35 μg ethinyl estradiol) iii. Sensitive substrates or narrow-therapeutic-range substrates of BCRP (except those that can be administered QD in the morning, separated by approximately 10 hours from evening administration of study drug)
15. Donation or receipt of blood from 90 days before Screening to the end of the study; donation or receipt of platelets, white blood cells, or plasma from 30 days before Screening to the end of the study
18 Years
ALL
No
Sponsors
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Spruce Biosciences
INDUSTRY
Responsible Party
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Principal Investigators
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Kyriakie Sarafoglou, M.D
Role: PRINCIPAL_INVESTIGATOR
Dept. of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, Univ. of Minnesota
Locations
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Spruce Study Site
Birmingham, Alabama, United States
Spruce Study Site
Los Angeles, California, United States
Spruce Clinical Site
Orange, California, United States
Spruce Study Site
Sacramento, California, United States
Spruce Study Site
Englewood, Colorado, United States
Spruce Clinical Site
Tampa, Florida, United States
Spruce Study Site
West Palm Beach, Florida, United States
Spruce Study Site
Chicago, Illinois, United States
Spruce Clinical Site
Indianapolis, Indiana, United States
Spruce Study Site
Baltimore, Maryland, United States
Spruce Clinical Site
Minneapolis, Minnesota, United States
Spruce Study Site
Rochester, Minnesota, United States
Spruce Clinical Site
Las Vegas, Nevada, United States
Spruce Study Site
Hickory, North Carolina, 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
Columbus, Ohio, United States
Spruce Clinical Site
Bend, Oregon, 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
Columbia, South Carolina, United States
Spruce Clinical Site
Memphis, Tennessee, United States
Spruce Study Site
Austin, Texas, United States
Spruce Study Site
Dallas, Texas, United States
Spruce Clinical Site
Edinburg, Texas, United States
Spruce Clinical Site
Fort Worth, Texas, United States
Spruce Study Site
Nedlands, Western Australia, Australia
Spruce study site
Brisbane, , Australia
Spruce Study Site
Elizabeth Vale, , Australia
Spruce Study Site
Melbourne, , Australia
Spruce Study Site
Brasília, , Brazil
Spruce Study Site
São Paulo, , Brazil
Spruce Study Site
St. John's, Newfoundland and Labrador, Canada
Spruce Study Site
London, Ontario, Canada
Spruce Study Site
Ottawa, , Canada
Spruce Study Site
Sherbrooke, , Canada
Spruce Study Site
Aarhus, , Denmark
Spruce Study Site
Copenhagen, , Denmark
Spruce Study Site
Tallinn, , Estonia
Spruce Study Site
Tartu, , Estonia
Spruce Study Site
Munich, , Germany
Spruce Study Site
Dublin, , Ireland
Spruce Study Site
Milan, , Italy
Spruce Study Site
Napoli, , Italy
Spruce Study Site
Rome, , Italy
Spruce Study Site
Torino, , Italy
Spruce Study Site
Riga, , Latvia
Spruce Study Site
Kaunas, , Lithuania
Spruce Study Site
Nijmegen, , Netherlands
Spruce Study Site
Krakow, , Poland
Spruce Study Site
Warsaw, , Poland
Spruce Study Site
Bucharest, , Romania
Spruce Study Site
Seoul, , South Korea
Spruce Study Site
Barcelona, , Spain
Spruce Study Site
Madrid, , Spain
Spruce Study Site
Seville, , Spain
Spruce Study Site
Tarragona, , Spain
Spruce Study Site
Falun, , Sweden
Spruce Study Site
Stockholm, , Sweden
Spruce Study Site
Sankt Gallen, , Switzerland
Spruce Study Site
Zurich, , Switzerland
Spruce Study Site
Istanbul, , Turkey (Türkiye)
Spruce Study Site
Birmingham, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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CAHmelia 203
Identifier Type: OTHER
Identifier Source: secondary_id
SPR001-203
Identifier Type: -
Identifier Source: org_study_id
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