Spinocerebellar Ataxia
Conditions
Interventions
Experimental group:Oral Taltirelin tablets
Control group:Oral placebo
Sponsors
Third Xiangya Hospital of Central South University
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Age between 18 and 75 years old (inclusive); gender unrestricted; 2. Genotypically diagnosed with spinocerebellar ataxia (SCA) (test results issued by laboratories certified by CLIA, ISO, NCCL or equivalent, confirmed by the investigator); 3. After the screening and 4-week run-in period, SARA score >= 3, gait score between 2 and 5, and able to independently walk 8 meters; 4. Medication compliance during the 4-week run-in period >= 80%; 5. Willing to comply with visit schedules, dosing plans, and other trial procedures; willing to keep a subject diary, undergo scale assessments, laboratory tests, ECG, and other trial-related examinations; voluntarily participate and sign informed consent.
Exclusion criteria
Exclusion criteria: 1. Patients with secondary cerebellar ataxia caused by cerebrovascular disease, alcohol, or medication; 2. Patients with motor disorders caused by motor paralysis or musculoskeletal diseases; 3. Patients with a history or concomitant diagnosis of neurodegenerative diseases such as dementia or Parkinson’s disease; 4. Patients with thyroid dysfunction at screening (hyperthyroidism or hypothyroidism); 5 . Patients diagnosed with hypothalamic dysfunction or pituitary tumors; 6. Patients diagnosed with malignant tumors; 7. Patients with a history of renal or hepatic failure; 8. Patients diagnosed with schizophrenia or major depressive disorder; 9. Patients with cognitive impairment: Mini-Mental State Examination (MMSE) score 160 mmHg or diastolic BP > 100 mmHg), pulmonary, hepatic diseases, diabetes (random blood glucose >= 11.1 mmol/L or fasting blood glucose >= 7 mmol/L), or immune system disorders; 14. History of drug or alcohol abuse within the past 12 months; 15. Abnormal laboratory test results at screening, including: (1) AST, ALT, or ?-GGT > 3× ULN; (2) Total bilirubin > 2× ULN; (3) Creatinine > 1.5× ULN; (4) Hemoglobin < 120 g/L in males or < 110 g/L in females; (5) Positive for HIV antibodies or specific antibodies to syphilis; 16. Patients allergic to Taltirelin or any excipients (mannitol, corn starch, povidone K30, or magnesium stearate); 17. Pregnant or breastfeeding female; 18. Patients planning to become pregnant during the trial period or not using medically reliable contraceptive methods (e.g., intrauterine device, condoms, or diaphragm with spermicide), except those who are postmenopausal for over 1 year or have undergone sterilization procedure; 19. Participation in another clinical trial (excluding non-interventional studies) within 12 weeks prior to screening; 20. Any other condition that, in the opinion of the investigator, makes the patient unsuitable for participation in the clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The mean change from baseline in the total SARA score of subjects after 28 weeks of medication; | — |
Secondary
| Measure | Time frame |
|---|---|
| The mean change from baseline in the total Scale for the Assessment and Rating of Ataxia (SARA) score at Weeks 4, 12, and 24 after medication;The mean change from baseline in the total Spinocerebellar Ataxia Functional Index (SCAFI) score at Weeks 12, 24, and 28 after medication;The mean change from baseline in the total Inventory of Non-Ataxia Symptoms (INAS) score at Weeks 12, 24, and 28 after medication;The mean change from baseline in the total Clinical Global Impression-Severity (CGI-S) score at Weeks 12, 24, and 28 after medication;The mean change from baseline in the total Patient Global Impression-Severity (PGI-S) score at Weeks 12, 24, and 28 after medication;The mean change from baseline in the total Activities of Daily Living (ADL) score at Weeks 12, 24, and 28 after medication;The mean change from baseline in the total Short Form Health Survey (SF-36) score at Weeks 12, 24, and 28 after medication;The mean change from baseline in the total Eating Assessment Tool-10 (EAT-10) score at Weeks 12, 24, and 28 after medication; | — |
Countries
China
Contacts
Public ContactHong Jiang
Third Xiangya Hospital of Central South University
Outcome results
None listed