2023-503771-13-00已完成2 期
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
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 44
- 试验地点
- 21
- 主要终点
- Absolute change from baseline in glucocorticoid dose in hydrocortisone equivalent(s) at Week 24
研究概览
简要总结
To evaluate the mean absolute glucocorticoid change in subjects with congenital adrenal hyperplasia over the 24-week, Double blind, Placebo-Controlled Treatment period
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •1.Male and female subjects ≥18 years old at screening.
- •2.Has a documented historical diagnosis of classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or elevated 17-hydroxyprogesterone and currently treated with hydrocortisone, hydrocortisone acetate, prednisone, prednisolone, methylprednisolone, dexamethasone.
- •3.Has lower limit of detection ≤ androstenedione ≤ 2.5x upper limit of normal at screening measured before an AM glucocorticoid dose.
- •4.Has been on a stable, supraphysiologic dose of glucocorticoid replacement for ≥1 month before screening.
- •For subjects with the salt-wasting form of congenital adrenal hyperplasia, subject has been on a stable dose of mineralocorticoid replacement for ≥1 months 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.
排除标准
- •1.Has a known or suspected diagnosis of any other known form of classic congenital adrenal hyperplasia (not due to 21-hydroxylase deficiency).
- •12.a Rosiglitazone, aromatase inhibitors, testosterone, growth hormones, or any other medication or supplement that could impact subject safety or confound interpretation of study results.
- •12.b. The drugs listed in protocol.
- •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.
- •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.
- •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.
- •7.Has clinically significant abnormal ECG or clinical laboratory results.
- •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.
- •2.Has a history that includes bilateral adrenalectomy or hypopituitarism.
- •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 Glucocorticoid Conversion Period to the end of the study.
结局指标
主要结局
Absolute change from baseline in glucocorticoid dose in hydrocortisone equivalent(s) at Week 24
Absolute change from baseline in glucocorticoid dose in hydrocortisone equivalent(s) at Week 24
次要结局
- 1. 1. Proportion of subjects with baseline GC dose ≤ 35mg HCe who achieve GC dose ≤11 mg/m2/day in HCe and A4 ≤ 1.2x baseline or ≤ ULN at Week 24
- 2. Proportion of subjects with GC dose ≤11 mg/m2/day in HCe and A4 ≤ 1.2x baseline or A4 ≤ ULN at Week 24
- 3. Proportion of subjects with improvement in at least one cardiovascular risk factor at Week 24.
研究者
Clinical Trials Helpline
Scientific
Spruce Biosciences Inc.
研究点 (21)
Loading locations...
相似试验
招募中
4 期
Congenital Adrenal Hyperplasia Once Daily Hydrocortisone TreatmentCongenital Adrenal HyperplasiaNCT03760835Federico II University150
Unknown
2 期
Continuous Subcutaneous Hydrocortisone Infusion in Congenital Adrenal HyperplasiaAdrenal Hyperplasia, CongenitalNCT01771328Haukeland University Hospital20
已完成
2 期
Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal HyperplasiaAdrenal InsufficiencyExcess AndrogenCongenital Adrenal Hyperplasia (CAH)NCT01859312National Institutes of Health Clinical Center (CC)8
招募中
1 期
A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)NCT07264595Changchun GeneScience Pharmaceutical Co., Ltd.88
招募中
2 期
A Phase 2 Randomized Trial Evaluating Efficacy of AGA2118 in Postmenopausal Women with Low Bone Mass (ARTEMIS)Postmenopausal Osteoporosis2024-515610-41-00Angitia Inc. Ltd.260