Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration

Phase III Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration

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.

Study Overview

• Status: Recruiting
• Conditions: Spinocerebellar Degeneration
• Intervention / Treatment: Drug: Placebo; Drug: KPS-0373

• Study Type: Interventional
• Enrollment (Estimated): 142
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Kissei Pharmaceutical Co., Ltd Kissei Pharmaceutical Co., Ltd; Phone Number: Email only; Email: rinsyousiken@pharm.kissei.co.jp

Study Locations

• Japan
  • Multiple Locations, Japan
    • Recruiting
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: KPS-0373 group; Screening period: One placebo tablet is orally administrated once daily after breakfast for 4 weeks.; Treatment period: One KPS-0373 2.4 mg tablet is orally administered once daily after breakfast in 24 weeks.	Drug: KPS-0373; Oral administration
Placebo Comparator: Placebo group; Screening period: One placebo tablet is orally administrated once daily after breakfast for 4 weeks.; Treatment period: One placebo tablet is orally administered once daily after breakfast in 24 weeks.	Drug: Placebo; Oral administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in SARA total score at 24 weeks of treatment period	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).	Up to 24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in SARA total score	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).	Up to 24 weeks
Symptom Maintenance Ratio (Ratio of Improvement and Stability Cases) based on SARA total score	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.	Up to 24 weeks
Change from baseline in SARA individual scores	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).	Up to 24 weeks
Change from baseline in SF-8	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.	Up to 24 weeks

Collaborators and Investigators

• Sponsor: Kissei Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): July 1, 2025
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): March 31, 2027

Study Registration Dates

• First Submitted: June 25, 2025
• First Submitted That Met QC Criteria: June 25, 2025
• First Posted (Actual): June 27, 2025

Study Record Updates

• Last Update Posted (Actual): June 27, 2025
• Last Update Submitted That Met QC Criteria: June 25, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Spinal Cord Diseases; Dyskinesias; Cerebellar Diseases; Cerebellar Ataxia; Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations
• Other Study ID Numbers: KPS1306; jRCT (Registry Identifier: jRCTs051200101)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The Individual Patient Data (IPD) are available upon reasonable request and with permission of Kissei Pharmaceutical Co., Ltd.; For a data sharing request for IPD, please contact Kissei Pharmaceutical at rinsyousiken@pharm.kissei.co.jp.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No