Spinocerebellar Degeneration
Conditions
Brief summary
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase III study to evaluate the efficacy and safety of KPS-0373 given once daily after breakfast in 142 patients with spinocerebellar degeneration . This study consists of the 4-week screening period, 24-week treatment period, and 4-week follow-up period.
Interventions
DRUGKPS-0373
Oral administration
DRUGPlacebo
Oral administration
Sponsors
Kissei Pharmaceutical Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Japanese SCD patients with mild to moderate ataxia
Exclusion criteria
* Patients with secondary ataxia * Patients with clinically significant hepatic, renal, or cardiovascular dysfunction
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in SARA total score at 24 weeks of treatment period | Up to 24 weeks | The SARA is a scale that assesses cerebellar ataxia. An evaluator observes the subject's ability to perform motor activities across in 8 individual items: ("gait: 0-8 points," "stance: 0-6 points," "sitting: 0-4 points," "speech disturbance: 0-6 points," "finger chase: 0-4 points," "nose-finger test: 0-4 points," "fast alternating hand movements: 0-4 points," and "heel-shin slide: 0-4 points." The total SARA score ranges from 0 (no ataxia) to 40 (severe ataxia). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in SARA total score | Up to 24 weeks | The SARA is a scale that assesses cerebellar ataxia. An evaluator observes the subject's ability to perform motor activities across in 8 individual items: ("gait: 0-8 points," "stance: 0-6 points," "sitting: 0-4 points," "speech disturbance: 0-6 points," "finger chase: 0-4 points," "nose-finger test: 0-4 points," "fast alternating hand movements: 0-4 points," and "heel-shin slide: 0-4 points." The total SARA score ranges from 0 (no ataxia) to 40 (severe ataxia). |
| Symptom Maintenance Ratio (Ratio of Improvement and Stability Cases) based on SARA total score | Up to 24 weeks | Subjects with the change in total SARA score from baseline of ≤ -1 point are defined as improved subjects, subjects with the change in total SARA score of \> -1 point and \< 1 point as unchanged subjects, and subjects with improvement and subjects with no change are defined as subjects maintaining symptoms. |
| Change from baseline in SARA individual scores | Up to 24 weeks | The SARA is a scale that assesses cerebellar ataxia. An evaluator observes the subject's ability to perform motor activities across in 8 individual items: ("gait: 0-8 points," "stance: 0-6 points," "sitting: 0-4 points," "speech disturbance: 0-6 points," "finger chase: 0-4 points," "nose-finger test: 0-4 points," "fast alternating hand movements: 0-4 points," and "heel-shin slide: 0-4 points." The total SARA score ranges from 0 (no ataxia) to 40 (severe ataxia). |
| Change from baseline in SF-8 | Up to 24 weeks | SF-8 is a questionnaire that comprehensively measures health-related QOL. Subjects answer questions in the following 8 items on a scale of 5 or 6: physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health. A higher score indicates better health in that item. |
Countries
Japan
Outcome results
None listed