Multicenter, placebo-controlled, randomized, double-blind, parallel-group comparison study evaluating the safety and efficacy of SB-ADSC-01 in patients with spinocerebellar ataxia
A Phase II clinical trial study evaluating the safety and efficacy of SB-ADSC-01 for spinocerebellar ataxia
(1) Patients with SCA3 or SCA6 spinocerebellar ataxia whose diagnosis has been confirmed by genetic testing
(2) Patients whose SARA score is 5-15
(3) Patients whose age at the time of obtaining consent is 20 years or older (gender-independent)
(4) Patients who understand the consent explanation document and can consent to the document from the person
(5) Patients who can participate in clinical trials in compliance with the schedules and procedures described in the clinical trial protocol
(1) Patients whose frequency and intensity of rehabilitation prior to obtaining consent cannot be maintained until the end of the trial
(2) Patients who are unable to keep the dosage and administration of tartyrelin hydrate and protyrelin tartrate hydrate in use before the consent is obtained until the end of the trial
(3) Pregnant women, lactating female patients, female patients who may be pregnant, female patients who wish to become pregnant during the study period, or male patients who wish to become pregnant partners
(4) Males and all fertile female patients who are unable to use appropriate contraceptive methods or obtain appropriate contraceptive consent from obtaining consent to the end of the trial *
*: Excluding those who have undergone permanent contraception and those after menopause (without menstruation for more than 12 months without another medical reason)
(5) History and complications of serious illnesses in critical organs including cardiac dysfunction, liver failure (eg, chronic cirrhosis), respiratory dysfunction and renal failure (hemodialysis or peritoneal dialysis) within 6 months before screening Patients with
(6) Patients with a history or complication of autoimmune disease (eg, systemic lupus erythematosus) affecting evaluation within 6 months before screening
(7) Patients with a history or complication of other neurological diseases (eg, amyotrophic lateral sclerosis) that affect evaluation within 6 months before screening
(8) Patients who received chemotherapy/radiotherapy within 5 years before screening
(9) Patients with a history or combination of malignant tumors
(10) Patients with hypersensitivity/allergy to products containing collagenase, dextran, dimethyl sulfoxide (DMSO), bovine or porcine components
(11) Patients with other mental disorders including dementia or disability depression, bipolar disorder, schizophrenia
(12) Patients with Beck Depression Inventory Second Edition (BDI-II) score of 21 points or more at a screening
(13) Patients who are currently participating in clinical trials of products such as pharmaceuticals, medical devices, and regenerative medicine, or other trial drugs, and 6 weeks from the administration of the trial drug to the start of administration of the trial product in this trial Or patients who have not passed a period of 10 times (whichever is longer) of the half-life (if the half-life of the investigational drug is known)
(14) Other patients who are judged to be inappropriate for study participation / participating physician
20age old over
No limit
Both
Spinocerebellar Ataxia
Intravenous administration of investigational product
Spinocerebellar Ataxia
Changes of SARA score from baseline(Visit 2) to Week 52 (Visit 8)
Efficacy:
(1) SARA score: Change from baseline (Visit 2) to each scheduled visit (Visit 3: Week 4, Visit 4: Week 8, Visit 5: Week 12, Visit 6: Week 24, Visit 7:Week 32, Visit 9: Week 60)
(2) SARA score: Change from the average value of Visit 1 and Visit 2 to Week 52 (Visit 8) of the first dose
(3) SCA Functional Index (SCAFI): Change from baseline (Visit 2) to each scheduled visit (Visit 3: Week 4, Visit 4: Week 8, Visit 5: Week 12, Visit 6: Week 24, Visit 7: Week 32, Visit 8: Week 52, Visit 9: Week 60)
(4) Inventory of Non-Ataxia Signs (INAS): Change from baseline (Visit 2) to scheduled visit (Visit 8: Week 52)
(5) Clinical Global Impression (CGI): Each scheduled visit (Visit 2: Baseline, Visit 3: Week 4, Visit 4: Week 8, Visit 5: Week 12, Visit 6: Week 24, Visit 7: Week 32, Visit 8: Week 52, Visit 9: Week 60)
(6) Patient Global Impression of Improvement (PGI-I): Each scheduled visit (Visit 3: Week 4, Visit 4: Week 8, Visit 5: Week 12, Visit 6: Week 24, Visit 7: Week 32, Visit 8: Week 52, Visit 9: Week 60)
