An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- 99mTc-DMSA
研究概览
简要总结
The primary objective of this trial is to:
1. Evaluate the efficacy of piclidenoson to increase renal uptake of 99mTc-labeled DMSA, in comparison to baseline, after 6 months (26 weeks) of treatment as a measure the reabsorption capacity of LMWPs by renal proximal tubules.
The secondary objectives of this trial are to:
- Evaluate changes in urinary excretion of LMWPs and other clinical parameters of renal Fanconi syndrome
- Evaluate safety of piclidenoson in patients with Lowe syndrome
详细描述
Objectives
- Primary Objective:
- The primary objective of the study is to test the efficacy of piclidenoson to increase renal uptake of 99mTc-labeled DMSA after 6 months (26 weeks) of treatment as a measure the reabsorption capacity of LMWPs by renal proximal tubules. 2. Secondary Objectives of the study are:
- to evaluate changes in urinary excretion of LMWPs and other clinical parameters of renal Fanconi syndrome,
- to evaluate the safety of piclidenoson in patients with Lowe syndrome.
Primary Endpoint
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males 18 years and above;
- •Documentation of genetically-proven Lowe Syndrome;
- •Estimated glomerular filtration rate (eGFR) ≥ 40 mL/min/1.73m2, as calculated by the CKD-EPI equation;
- •Male subjects must refrain from sperm donation during treatment and until at least 1 month after the last dose of study medication. Male subjects must agree to use condoms throughout the course of the trial and for 1 month after the last dose of study medication;
- •Ability to complete the study in compliance with the protocol; and
- •Ability to understand and provide written informed consent (subject or legal guardian).
排除标准
- •Subjects receiving chronic therapies not related to Lowe syndrome; Estimated glomerular filtration rate (eGFR) <40 mL/min/1.73m2 by the CKD-EPI equation;
- •Liver aminotransferase levels greater than 1.5 times the laboratory's upper limit of normal;
- •QTcF interval > 450 milliseconds (msec) on ECG (average of triplicate ECGs) (except when QT prolongation is associated with right or left bundle branch block or cardiac pacemaker, in which case enrollment is allowed);
- •A condition which increases proarrhythmic risk, including hypokalemia, hypomagnesemia, or congenital Long QT Syndrome;
- •Ongoing or planned use of a concomitant medication that is on the CredibleMedsTM list of drugs known to cause Torsades des Pointes; https://crediblemeds.org/;
- •Active gastrointestinal disease which could interfere with the absorption of oral medication;
- •Active drug or alcohol dependence;
- •Concomitant use of strong cytochrome P450 inducers, e.g., rifampin, phenobarbital, phenytoin, carbamazepine;
- •Significant acute or chronic medical or psychiatric illness, including chronic systemic infection or malignancy, that, in the judgment of the Investigator, could compromise subject safety, limit the subject's ability to complete the study, and/or compromise the objectives of the study; and
- •Participation in another investigational drug or vaccine trial concurrently or within 30 days prior to the Screening visit.
研究组 & 干预措施
Piclidenoson (CF101) open-label treated patients
Piclidenoson will be administered orally at a dose of 3 mg twice per day for 6 months
干预措施: Piclidenoson (Drug)
结局指标
主要结局
99mTc-DMSA
时间窗: 6 months
The primary end-point is to assess whether treatment for 6 months with piclidenoson improves the functioning of renal proximal tubular cells (PTCs), as assessed by the increase in 99mTc-DMSA uptake as compared to Baseline
次要结局
- beta-2 microglobulin(3 and 6 months of treatment)
- urinary excretion of sodium(3 and 6 months)
- urinary excretion glucose(3 and 6 months)
- urinary excretion of phosphate(3 and 6 months)
- urinary excretion of amino acids(3 and 6 months)
- serum bicarbonate(3 and 6 months)