Multicenter, Randomized, Double-blind, Placebo-controlled, Phase IV Clinical Trial to Evaluate and Compare the Efficacy and Safety of C-Trelin OD Tab 5mg(Taltirelin Hydrate) in Patients With Ataxia Induced by Spinocerebellar Degeneration
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 160
- 试验地点
- 8
- 主要终点
- Changes in Korean Version of Scale for the Assessment and Rating of Ataxia(K-SARA) scores
研究概览
简要总结
The aim of this study is to evaluate the efficacy and safety of C-Trelin OD Tablet 5mg(Taltirelin Hydrate) in Multicenter, randomized, double-blind, placebo-controlled clinical trial in patients with ataxia induced by spinocerebellar degeneration.
详细描述
Taltirelin Hydrate, an active substance of C-Trelin OD tablet 5mg, is an analogue of Thyrotropin Releasing Hormone(TRH). TRH is distributed widely in the brain, and exerts variety of central nervous system effects as well as endocrine activity such as releasing of Thyroid Stimulating Hormone(TSH) and Prolactin. Based on these actions, studies have been attempted for the treatment of various neurological diseases such as refractory epilepsy, cerebellar ataxia, amyotrophic lateral sclerosis, cerebellar ataxia and so on. C-Trelin OD tablet 5mg(Taltirelin Hydrate), an investigational product, is a commercially available drug that was approved by Ministry of Food and Drug Safety(MFDS) based on the safety and efficacy. However, the studies with this medicine were conducted before 1997, and the evaluation criteria for the efficacy was quite different from the current evaluation criteria.
In this clinical trial, K-SARA as an objective evaluation criteria validated in Korean patients is used to confirm the efficacy of C-Trelin to improve ataxia, and to prove safety by evaluating changes in clinical laboratory data and adverse events.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients ≥ 20 years of age
- •Patients who voluntarily agreed to enroll in the study and signed an informed consent form
- •Patients diagnosed with ataxia (genetic or non-genetic) due to spinocerebellar degeneration by the investigator's judgment based on the results of essential diagnostic examinations stated below.
- •<Genetic ataxia>
- •Spinocerebellar ataxia(SCA)
- •Friedreich's ataxia(FA)
- •Other genetic ataxia
- •<Non-genetic ataxia>
- •Idiopathic late onset cerebellar ataxia(ILOCA) The following examinations to diagnose ataxia (genetic or non-genetic) induced by spinocerebellar degeneration can be used, and the investigator has to diagnose by combining at least one diagnostic examination result among the following-items.
- •Medical history: alcohol abuse, medication history, family history
- •Genetic test: SCA 1, 2, 3, 6, 7, 8, 17, FA which is available for the patient
- •Brain MRI or CT scan: abnormalities of cerebellum and pons, brain vascular disease, or brain tumors
- •Retinal or optic nerve examination
排除标准
- •Patients with bed-ridden state at the time of screening even though the patient is diagnosed with ataxia induced by spinocerebellar degeneration
- •Patients with ataxia caused by stroke
- •Patients with ataxia caused by cerebrovascular, alcoholic-induced or drug-induced secondary cerebellar abnormalities
- •Patients with complications of other neurodegenerative diseases such as Parkinson's disease and multiple system atrophy(however, patients diagnosed with SCA 2, SCA 3, SCA 17 can be enrolled)
- •Patients with malignant neoplastic disease
- •Patients with kidney failure and liver failure history
- •Patients with abnormalities in clinical laboratory test results as follows( Patients with liver dysfunction: Aspartate Transaminase(AST), Alanine Transaminase(ALT) > 3 times than the upper limit of normal range(ULN), Total bilirubin > 1.5 times than the ULN, Patients with renal dysfunction: Serum creatinine > 1.5mg/dl, Patients with thyroid dysfunction: free T4: above or below the normal range)
- •Patients with thyroid dysfunction at the time of screening(hyperthyroidism, hypothyroidism)
- •Patients accompanied by lesions other than spinocerebellar degeneration from Brain MRI or CT scan
- •Patients with schizophrenia, major depressive disorder
- •Patients with a history of acute myocardial infarction within 2 years of the screening visit
- •Patients with a history of unstable angina pectoris within 2 years of the screening visit
- •Patients taking contraindicated concomitant medication( However, patients with following drugs can be enrolled only if they are administered 4 weeks before the screening visit, and the type, dosage, and volume should be kept unchanged during the clinical trial.: Parkinson's disease medicine, Anxiolytics, Antidepressants, Antiepileptics, Antipsychotics, Medicine for dysuria, Sleep inducer, β blocker)
- •Patients with hypersensitivity to Taltirelin Hydrate
- •Cognitive dysfunction: Korean Version of Mini-Mental State Exam(K-MMSE) ≤ 20
- •Patients who are pregnant or lactating
- •All childbearing females who are planning to pregnant during the clinical trial or who are not using medically reliable contraceptive methods (such as intrauterine contraceptives, condoms or diaphragms combined with spermicides) except menopaused more than 1 year from the last menstruation or had undergone surgical sterilization
- •Patients participating in any other clinical trials or participated 30 days before
- •Patients whom the investigator considers inappropriate for the clinical trial due to any other reasons
研究组 & 干预措施
C-Trelin OD Tab(5mg Taltirelin Hydrate)
BID, 10mg per day, for 24 weeks
干预措施: C-Trelin OD Tab(5mg Taltirelin Hydrate) (Drug)
Placebo (0mg Taltirelin Hydrate)
BID, 10mg per day, for 24 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Changes in Korean Version of Scale for the Assessment and Rating of Ataxia(K-SARA) scores
时间窗: 24 weeks of administration compared to the baseline
K-SARA consists of walking, standing, sitting, speech impairment, finger following, nose-finger test, palm flipping, and heel-shin test. The total score is 40 and higher scores indicate serious ataxia.
次要结局
- Changes in Korean Version of Scale for the Assessment and Rating of Ataxia(K-SARA) scores(4 weeks and 12 weeks of administration compared to the baseline)
- Changes in Clinical Global Impression - Severity(CGI-S) scores(4 weeks, 12 weeks, and 24 weeks of administration compared to the baseline)
- Changes in Clinical Global Impression - Improvement(CGI-I) scores(12 weeks and 24 weeks of administration compared to 4 weeks of administration)
- Changes in Clinical Global Impression - Efficacy index(CGI-E) scores(12 weeks and, 24 weeks of administration compared to 4 weeks of administration)
- Changes in Korean Version of the Scale for Outcomes in Parkinson's Disease-Autonomic(K-SCOPA-AUT) scores(24 weeks of administration compared to the baseline)
- Changes in EQ-5D-5L scores(24 weeks of administration compared to the baseline)
- Changes in Tinetti balance test & Gait assessment scores(24 weeks of administration compared to the baseline)