A Placebo-Controlled, Double-Blind, Randomized, Group-Controlled Trial Evaluating the Efficacy and Safety of AJA030 in Spinocerebellar Degeneration

Authors
Category Primary study
Registry of TrialsJapan Primary Registry Network
Year 2020
INTERVENTION: Randomized, Double‐Blind, Placebo‐Controlled Study CONDITION: spinocerebellar degeneration (SCD) PRIMARY OUTCOME: Change from baseline in SARA "Total" scores at Week 48 SECONDARY OUTCOME: 1) SARA total scores after 4, 8, 16, 24, 32 ,40 and 48 weeks; 2) SARA Walking, Standing Scores; 3) SARA scores (other than walking and standing scores); 4) Inspection of center of gravity oscillation (total locus length for 30 seconds, rectangular area); 5) Timed Up and Go Test (TUG) (shortest time); 6) Beck Depression Inventory‐II (BDI‐II); 7) Clinical Global Impression (CGI); 8) Patient Global Impression of Improvement (PGI‐I); 9) Short‐Form 8 (SF‐8) INCLUSION CRITERIA: 1) Patients with genetically diagnosed autosomal dominant hereditary spinocerebellar degeneration (SCA6). Note 1) 2) Patients aged 20 years or older at the time of informed consent. 3) Patients who scored 1 or more In SARA(Scale for the Assessment and Rating of Ataxia) gait scores in the pre‐enrollment test and had a SARA "sum" score of 10 or more. 4) Patients who are able to walk 10m or more with or without assistive devices in the pre‐enrollment test. 5) Patients for whom written informed consent is obtained from the individual for participation in the clinical trial. Note 1) Genetic diagnosis of the patient is not indispensable, when the disease type is confirmed by the genetic diagnosis in the family.
Epistemonikos ID: 73663591892096dc34ac87c95b2e977488329ee5
First added on: Dec 20, 2022