C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration

NCT04107740 | Completed Phase 4

• 1 other identifier: CTT-004
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

Spinocerebellar Degeneration

Keywords

Spinocerebellar degeneration; Ataxia

Outcome Measures

Primary Outcomes (1)

• 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.

  24 weeks of administration compared to the baseline

Secondary Outcomes (7)

• 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

Study Arms (2)

C-Trelin OD Tab(5mg Taltirelin Hydrate)

EXPERIMENTAL

BID, 10mg per day, for 24 weeks

Drug: C-Trelin OD Tab(5mg Taltirelin Hydrate)

Placebo (0mg Taltirelin Hydrate)

PLACEBO COMPARATOR

BID, 10mg per day, for 24 weeks

Drug: Placebo

Interventions

C-Trelin OD Tab(5mg Taltirelin Hydrate) DRUG

BID, 10mg per day, for 24 weeks

C-Trelin OD Tab(5mg Taltirelin Hydrate)

Placebo DRUG

BID, 10mg per day, for 24 weeks

Placebo (0mg Taltirelin Hydrate)

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• HLB Pharmaceutical Co., Ltd.: lead

Study Sites (8)

Chonnam National University Hospital

Gwangju, South Korea

Location

Gangnam Severance Hospital

Seoul, South Korea

Location

Korea University Anam Hospital

Seoul, South Korea

Location

Korea University Guro Hospital

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

Seoul Metropolitan Government Seoul National University Boramae Medical Center

Seoul, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

The Catholic University of Korea, Seoul ST. Mary's Hospital

Seoul, South Korea

Location

MeSH Terms

Conditions

Spinocerebellar Degenerations; Ataxia

Interventions

TA 0910

Condition Hierarchy (Ancestors)

Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Seong-beom Koh

  Korea University Guro Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2019
• First Posted: September 27, 2019
• Study Start: March 4, 2019
• Primary Completion: February 8, 2021
• Study Completion: February 8, 2021
• Last Updated: July 24, 2023

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will not share

Locations

KR (8)