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

NCT07040137 | Recruiting Phase 3

• 2 other identifiers: KPS1306jRCT
• Study Type: interventional
• Target: 142
• Locations: 1 country
• Sites: 1

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.

Conditions

Spinocerebellar Degeneration

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Change from baseline in SARA total score

  Up to 24 weeks

• Symptom Maintenance Ratio (Ratio of Improvement and Stability Cases) based on SARA total score

  Up to 24 weeks

• Change from baseline in SARA individual scores

  Up to 24 weeks

• Change from baseline in SF-8

  Up to 24 weeks

Study Arms (2)

KPS-0373 group

EXPERIMENTAL

* 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

Placebo group

PLACEBO COMPARATOR

* 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

Interventions

KPS-0373 DRUG

Oral administration

KPS-0373 group

Placebo DRUG

Oral administration

Placebo group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Japanese SCD patients with mild to moderate ataxia

You may not qualify if:

• Patients with secondary ataxia
• Patients with clinically significant hepatic, renal, or cardiovascular dysfunction

Sponsors & Collaborators

• Kissei Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Research Site

Multiple Locations, Japan

RECRUITING

MeSH Terms

Conditions

Spinocerebellar Degenerations

Condition Hierarchy (Ancestors)

Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Kissei Pharmaceutical Co., Ltd Kissei Pharmaceutical Co., Ltd

CONTACT

Email only; rinsyousiken@pharm.kissei.co.jp

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2025
• First Posted: June 27, 2025
• Study Start: July 1, 2025
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): March 31, 2027
• Last Updated: June 27, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP

Locations

JP (1)