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Phase 2 of CellCept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

CellCept 治疗幼年神经元蜡质脂褐质沉着症的第 2 期研究

基本信息

批准号：
8653838
负责人：
Erika Augustine
金额：
$ 17.6万
依托单位：
UNIVERSITY OF ROCHESTER
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
起止时间：
2011-06-22 至 2016-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8653838
关键词：
Phase CellCept Treatment Juvenile Neuronal

项目摘要

DESCRIPTION (provided by applicant): JNCL is a devastating progressive neurologic disorder. Initial symptoms for children with JNCL typically occur between ages 5 and 9 years and include progressive impairment of vision, seizures, difficulty with movement, and dementia. Scientists are still learning about how and why this disorder occurs. Although the seizures associated with JNCL can be treated, unfortunately there are no specific treatments for the underlying cause of JNCL at this time. Children with JNCL have antibodies to parts of brain cells. In animal models of JNCL, studies have shown that minimizing the ability to make antibodies may lead to slowing of the progression of the disease. Mycophenolate (CellCept) is a medication approved by the FDA for prevention of kidney transplant rejection in children; it acts by minimizing the formation of antibodies and therefore, is potentially an effective treatment for JNCL. A 22 week study of mycophenolate in children with JNCL is proposed. Each child will receive eight weeks of mycophenolate, and eight weeks of placebo, separated by four weeks off study medication. Investigators will be blinded to the order of treatments for each child (mycophenolate followed by placebo, or placebo followed by mycophenolate). Signs and symptoms of JNCL will be evaluated at the beginning and end of each eight week study period. Between evaluations, children will be monitored closely for potential medication side effects. The main objective is to assess whether patients with JNCL are able to complete a course of this medication without difficulty and also examine the impact of a short course of mycophenolate on signs of JNCL, including seizures, behavior, thinking, and motor function.

描述（由申请人提供）： JNCL是一种破坏性的进行性神经系统疾病。JNCL儿童的初始症状通常发生在5至9岁之间，包括进行性视力损害、癫痫发作、运动困难和痴呆。科学家们仍在研究这种疾病是如何发生的，以及为什么会发生。虽然与JNCL相关的癫痫发作可以治疗，但不幸的是，目前尚无针对JNCL潜在原因的特异性治疗方法。患有JNCL的儿童对部分脑细胞有抗体。在JNCL的动物模型中，研究表明，最大限度地减少产生抗体的能力可能会减缓疾病的进展。霉酚酸酯（CellCept）是FDA批准用于预防儿童肾移植排斥反应的药物;它通过最大限度地减少抗体的形成来发挥作用，因此可能是JNCL的有效治疗方法。建议在JNCL儿童中进行一项为期22周的霉酚酸酯研究。每名儿童将接受8周的麦考酚酯治疗和8周的安慰剂治疗，间隔四周的研究药物治疗。研究者将对每例儿童的治疗顺序（先麦考酚酯后安慰剂，或先安慰剂后麦考酚酯）设盲。将在每8周研究期开始和结束时评价JNCL的体征和症状。在评估之间，将密切监测儿童的潜在药物副作用。主要目的是评估JNCL患者是否能够毫无困难地完成一个疗程，并检查短期麦考酚酯对JNCL体征的影响，包括癫痫发作、行为、思维和运动功能。

项目成果

期刊论文数量（6）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

Fiber-Optic Distributed Sensing Network for Thermal Mapping of Gold Nanoparticles-Mediated Radiofrequency Ablation.

DOI：
10.3390/bios12050352
发表时间：
2022-05-18
期刊：
Biosensors
影响因子：
0
作者：
通讯作者：

Cost-Effective Fiber Optic Solutions for Biosensing.

DOI：
10.3390/bios12080575
发表时间：
2022-07-28
期刊：
Biosensors
影响因子：
0
作者：
通讯作者：

Bite Force Mapping Based on Distributed Fiber Sensing Network Approach.

DOI：
10.3390/s24020537
发表时间：
2024-01-15
期刊：
Sensors (Basel, Switzerland)
影响因子：
0
作者：
Katrenova Z;Alisherov S;Yergibay M;Kappasov Z;Blanc W;Tosi D;Molardi C
通讯作者：
Molardi C

A novel, hybrid, single- and multi-site clinical trial design for CLN3 disease, an ultra-rare lysosomal storage disorder.

针对 CLN3 疾病（一种极其罕见的溶酶体贮积症）的新型、混合、单中心和多中心临床试验设计。

DOI：
10.1177/1740774519855715
发表时间：
2019
期刊：
Clinical trials (London, England)
影响因子：
0
作者：
Adams,HeatherR;Defendorf,Sara;Vierhile,Amy;Mink,JonathanW;Marshall,FrederickJ;Augustine,ErikaF
通讯作者：
Augustine,ErikaF

Short-Term Administration of Mycophenolate Is Well-Tolerated in CLN3 Disease (Juvenile Neuronal Ceroid Lipofuscinosis).