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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
160
BID, 10mg per day, for 24 weeks
BID, 10mg per day, for 24 weeks
Chonnam National University Hospital
Gwangju, South Korea
Gangnam Severance Hospital
Seoul, South Korea
Korea University Anam Hospital
Seoul, South Korea
Korea University Guro Hospital
Seoul, South Korea
Changes in Korean Version of Scale for the Assessment and Rating of Ataxia(K-SARA) scores
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.
Time frame: 24 weeks of administration compared to the baseline
Changes in Korean Version of Scale for the Assessment and Rating of Ataxia(K-SARA) scores
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.
Time frame: 4 weeks and 12 weeks of administration compared to the baseline
Changes in Clinical Global Impression - Severity(CGI-S) scores
Changes in Clinical Global Impression(CGI) is a commonly used measure to assess the severity of symptoms, improvement, the effectiveness of treatment and it consists of Clinical Global Impression-Severity(CGI-S), Clinical Global Impression-Improvement scale(CGI-I), and Clinical Global Impression-Efficacy index(CGI-E). Clinical Global Impression-Severity(CGI-S) means overall clinical impression at the time of drug administration. It ranges from 1 to 7, higher scores indicate higher severity.
Time frame: 4 weeks, 12 weeks, and 24 weeks of administration compared to the baseline
Changes in Clinical Global Impression - Improvement(CGI-I) scores
Changes in Clinical Global Impression(CGI) is a commonly used measure to assess the severity of symptoms, improvement, the effectiveness of treatment and it consists of Clinical Global Impression-Severity(CGI-S), Clinical Global Impression-Improvement scale(CGI-I), and Clinical Global Impression-Efficacy index(CGI-E). Clinical Global Impression-Improvement scale(CGI-I) means current improvement from the patient's baseline status. It ranges from 1 to 7, and higher scores indicate worsening of symptoms.
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Samsung Medical Center
Seoul, South Korea
Seoul Metropolitan Government Seoul National University Boramae Medical Center
Seoul, South Korea
Seoul National University Hospital
Seoul, South Korea
The Catholic University of Korea, Seoul ST. Mary's Hospital
Seoul, South Korea
Time frame: 12 weeks and 24 weeks of administration compared to 4 weeks of administration
Changes in Clinical Global Impression - Efficacy index(CGI-E) scores
Changes in Clinical Global Impression(CGI) is a commonly used measure to assess the severity of symptoms, improvement, the effectiveness of treatment and it consists of Clinical Global Impression-Severity(CGI-S), Clinical Global Impression-Improvement scale(CGI-I), and Clinical Global Impression-Efficacy index(CGI-E). Clinical Global Impression-Efficacy index(CGI-E) indicates the effect of the current treatment and the severity of the side effects. It is calculated by dividing the treatment effect by the side effects. The treatment effect ranges from 1 to 4, and higher scores indicate the better effect. The side effect ranges from 1 to 4, and higher scores indicate the greater side effect.
Time frame: 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
Korean Version of the Scale for Outcomes in Parkinson's Disease-Autonomic(K-SCOPA-AUT) score is a patient report questionnaire for intensive evaluation of autonomic dysfunction in Parkinson's disease to determine the problems with physical functioning, such as difficulty in urinating or sweating over the past month. The total score is 69 points, and each item is evaluated on a scale of 0(never) to 3(often).
Time frame: 24 weeks of administration compared to the baseline
Changes in EQ-5D-5L scores
EQ-5D-5L consists of EQ-5D-5L descriptive system and EQ Visual Analogue Scale (EQ VAS). EQ-5D-5L descriptive system consists of 5 items (mobility, self-care, usual activities, pain / discomfort, anxiety / depression), and 5 levels for each item \[No problems (Level 1), slight problems (Level 2), moderate problems (Level 3), severe problems (Level 4), extreme problems (Level 5)\]. EQ Visual Analogue Scale(EQ VAS) measures and records the subject's rating for their current health-related quality of life state on a 20cm visual analogue scale. Each endpoint is marked with "best imaginable health(100)" and "worst imaginable health(0)".
Time frame: 24 weeks of administration compared to the baseline
Changes in Tinetti balance test & Gait assessment scores
Tinetti balance test \& Gait assessment measures the gait and balance of a subject according to the subject's ability to perform a specific task. Each item is scored by 0 to 2 and higher scores indicate the better performance. The sum of each individual score consists of the total gait scores(12 points), the total balance scores(16 points), and the sum of the gait and balance scores(28 points).
Time frame: 24 weeks of administration compared to the baseline