NCT05190744已完成2 期
A Multi-center, Open-Label, Exploratory Study to Assess the Efficacy of PB in Decreasing the Urine Output and Increasing the Urine Osmolality in Patients With Hereditary Nephrogenic Diabetes Insipidus, Patients With Autosomal Dominant Polycystic Kidney Disease Treated With Tolvaptan, And Severely Polyuric Patients With Previous Lithium Administration (Serendipity-PB1)
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 36
- 试验地点
- 1
- 主要终点
- Change in urine osmolality
研究概览
简要总结
The purpose of this research is to study the effectiveness and safety of the medication PB in slowing the frequent urination related to tolvaptan as long-term treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), or frequent urination related to inherited nephrogenic diabetes insipidus as an inherited condition or as an acquired condition from prior treatment with lithium.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, ≥ 18 years of age (inclusive) at time of screening
- •Diagnosis of one of the following:
- •ADPKD(as delineated in cohort 1)
- •Congenital NDI (as delineated in cohort 1)
- •Lithium-induced NDI (as delineated in cohort 1)
- •Glomerular filtration rate (GFR) ≥ 25 ml/min/1.73 m2 at time of screening visit calculated as in cohort
- •24 hours urine volume in baseline 1 visit ≥ 5000 ml/ day
- •If hypertensive, blood pressure controlled on antihypertensives (<130/80 mm Hg) at least 30 days before day
- •Antihypertensives may be adjusted at time of baseline 2 per PI discretion.
- •Female participants (see details in cohort 1 inclusion criteria)
- •Have read, understood, and provided written informed consent after the nature of the study has been fully explained and must be willing to comply with protocol requirements and study-related procedures.
- •Negative urinary pregnancy test (if applicable) at baseline 2
- •Capable of providing urine samples as dictated by the protocol
排除标准
- •Advanced diabetes (e.g., glycosylated hemoglobin [HgbA1c] >7.5%, and/or glycosuria by dipstick, significant proteinuria [>300 mcg albumin/mg creatinine]), other significant kidney disease, kidney cancer, transplanted kidney, single kidney, kidney surgery within the past 6 months (including cyst drainage or fenestration) or acute kidney injury within 6 months prior to screening.
- •Clinically significant incontinence, overactive bladder, or urinary retention (e.g., benign prostatic hyperplasia).
- •Other significant chronic medical disease (heart failure, diabetes mellitus, liver disease, transient or persistent elevated transaminases)
- •History of acute gout attack in the past 30 days
- •History of clinically significant drug or alcohol abuse in the 2 years prior to screening visit.
- •Uncontrolled hyperuricemia or active gout
- •History of hepatotoxicity related to tolvaptan; or clinically significant liver disease or impairment; or alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin values >1.2 x Upper Limit of Normal (ULN) during screening.
- •Medical history or findings that preclude safe participation in the trial or participants who are likely to be non-compliant with trial procedures in the opinion of the investigator or medical monitor.
- •Requirement for ongoing diuretic use.
- •Participants who are currently taking, or are expected to be taking, strong or moderate CYP3A4 or CYP2C8 inhibitors or inducers including regular use of grapefruit juice, Seville oranges, or St. John's wort. If applicable, there should be a 14-day washout of these treatments prior to Day
- •Prior use of a sodium-glucose cotransporter 2 inhibitor (SGLT2i) (e.g., canagliflozin, dapagliflozin, empagliflozin, etc.) within the 2 months prior to screening visit or expected need for initiation of treatment with a SGLT2i inhibitor during the study. Current use of SGLT2i will be reviewed by PI and allow enrollment if patient has been on stable dose for at least 2 months.
- •Prior use of a hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor within the 2 months prior to screening visit or expected need for initiation of treatment with a HIF-PH inhibitor during the study;
- •Participants who have taken any investigational drug or used an investigational device within 30 days, or 5 half-lives, whichever is longer, prior to screening visit 1a or plan to participate in an interventional trial during the study.
- •Allergy to probenecid
- •History of persistent hyponatremia
- •Positive test results for hepatitis B surface antigen (HBsAg).
- •Positive test results for hepatitis C (HCV) antibody (Anti-HCV), with the exception of participants for whom the reflex HCV RNA titer test is negative.
研究组 & 干预措施
Polyuric subjects with Hereditary Nephrogenic Diabetes Insipidus
Experimental
Polyuric subjects with hereditary nephrogenic diabetes insipidus with loss of function of arginine vasopressin receptor 2 (AVPR2) or aquaporin 2 (AQP2) will be treated with PB
干预措施: PB (Drug)
Polyuric subjects with Autosomal Dominant Polycystic Kidney Disease treated with Tolvaptan
Experimental
Polyuric subjects with autosomal dominant polycystic kidney disease on chronic tolvaptan treatment will be treated with PB
干预措施: PB (Drug)
Polyuric subject secondary to lithium administration
Experimental
Polyuric subject post lithium administration will receive PB
干预措施: PB (Drug)
结局指标
主要结局
Change in urine osmolality
时间窗: Baseline, 30 days
Measured in milliosmoles per kilogram of water (mOsm/kg) from a urine specimen and is a measure of the concentration of osmotically active particles, principally sodium, chloride, potassium, and urea
次要结局
- Change in urine output(From Baseline 2 to Post Treatment Follow Up at the end of 5 weeks)
研究者
Fouad T. Chebib
Principal Investigator
Mayo Clinic
研究点 (1)
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