A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
NCT ID: NCT04544410
Last Updated: 2025-10-01
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
100 participants
INTERVENTIONAL
2021-02-22
2025-01-31
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
Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks
Tildacerfont/Placebo
Tablet, administered daily
Placebo
Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 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-hydroxyprogesterone (17-OHP) and currently treated with hydrocortisone (HC), HC acetate, prednisone, prednisolone, methylprednisolone, dexamethasone (or a combination of the aforementioned glucocorticoid \[GCs\])
3. Has lower limit of detection ≤ androstenedione (A4) ≤ 2.5x upper limit of normal (ULN) at screening measured before a morning GC dose
4. Has been on a stable, supraphysiologic dose of GC replacement (defined as ≥30 mg/day and ≤60 mg/day in HCe) for ≥1 month before screening
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. Shows clinical signs or symptoms of adrenal insufficiency
5. 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
6. 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:
Increased risk of suicide based on the Investigator's judgment or the results of the C-SSRS conducted at screening and baseline (eg, C-SSRS Type 3, 4, or 5 ideation within the past 6 months or any suicidal behavior within the past 12 months) b. Hospital Anxiety and Depression Scale (HADS) score \>12 for either depression or anxiety at screening or baseline
7. 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
8. Routinely works overnight shifts
9. 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
10. Females who are pregnant or nursing
11. Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study
12. Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before the start of the Treatment Period 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 drugs which are:
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)
13. 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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Ron Newfield, M.D
Role: PRINCIPAL_INVESTIGATOR
Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.
Locations
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Spruce Study Site
Birmingham, Alabama, United States
Spruce Study Site
Los Angeles, California, United States
Spruce Study Site
San Diego, California, United States
Spruce Study Site
Indianapolis, Indiana, United States
Spruce Study Site
Baltimore, Maryland, United States
Spruce Biosciences Clinical Site
Ann Arbor, Michigan, United States
Spruce Study Site
Minneapolis, Minnesota, United States
Spruce Study Site
New Brunswick, New Jersey, 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 Study Site
Philadelphia, Pennsylvania, 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 Study Site
Fort Worth, Texas, United States
Spruce Study Site
Blacktown, , Australia
Spruce Study Site
Brisbane, , Australia
Spruce Study Site
Elizabeth Vale, , Australia
Spruce Study Site
Parkville, , Australia
Spruce Study Site
Curitiba, , Brazil
Spruce Study Site
São Paulo, , Brazil
Spruce Study Site
Ottawa, Ontario, Canada
Spruce Study Site
Sherbrooke, Quebec, Canada
Spruce Study Site
Tallinn, , Estonia
Spruce Study Site
Tartu, , Estonia
Spruce Study Site
Munich, , Germany
Spruce Study Site
Roma, , Italy
Spruce Study Site
Riga, , Latvia
Spruce Study Site
Kaunas, , Lithuania
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
Stockholm, , Sweden
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
Document Type: Informed Consent Form
Other Identifiers
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CAHmelia 204
Identifier Type: OTHER
Identifier Source: secondary_id
SPR001-204
Identifier Type: -
Identifier Source: org_study_id
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