NL-OMON52374招募中2 期
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 - Efficacy and Safety of Tildacerfont in Adult Subjects with Classical CAH
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Male and female subjects >=18 years old at screening
- •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. For subjects with the salt-wasting form of CAH, subject has been on a stable
- •dose of mineralocorticoid replacement for >= 1 month before screening
- •4. 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.
- •5. 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
- •6. Has androstenedione (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)
- •7. 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.
排除标准
- •1. Has a known or suspected diagnosis of any other known form of classic CAH
- •(not due to 21-hydroxylase deficiency)
- •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:
- •a. An ongoing malignancy or <3 years of remission history from any malignancy,
- •other than successfully treated localized skin cancer.
- •b. Estimated glomerular filtration rate (eGFR) of <45 mL/min/1.73 m2
- •c. Current or history of liver disease (with the exception of Gilbert*s
- •d. 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
- •e. Active hepaptitis B, hepatitis C, or human immunodeficiency virus (HIV) at
- •f. Subjects who plan to undergo bariatric surgery during the study are
- •g. 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:
- •a. Increased risk of suicide based on the Investigator*s judgment or the
- •results of the Columbia-Suicide Severity Rating Scale (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)
- •b. Hospital Anxiety and Depression Scale (HADS) score >12 for either depression
- •or anxiety at screening or Week 6
- •9. Has clinically significant abnormal electrocardiogram (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:
- •a. Any clinically meaningful abnormal ECG results, including
- •Fridericia-corrected QT interval (QTcF) >450 milliseconds (ms) for male
- •participants or >470 ms for female participants
- •b. Alanine aminotransferase (ALT) >2x ULN
- •c. Total bilirubin >1.5x ULN
- •d. Total bile acids >5x ULN
- •10. Routinely works overnight shifts
- •11. Subjects with travel plans/work schedules that result in significant and
- 另有 4 项未显示
研究者
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