Can a daily pill slow the wobble of spinocerebellar degeneration?
NCT ID NCT07040137
First seen Sep 04, 2026 · Last updated Sep 04, 2026
Summary
This Phase 3 trial tests whether an experimental oral drug, KPS-0373, can improve movement and coordination in people with spinocerebellar degeneration, a condition that causes progressive clumsiness and balance problems. About 142 Japanese adults with mild to moderate ataxia will take either the drug or a placebo once daily for 24 weeks. Researchers will measure changes in a standard ataxia score that rates walking, standing, speech, and hand control.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- an experimental oral drug called KPS-0373
- What this could lead to
- If it works, KPS-0373 could become one of the first treatments to slow or ease the movement and coordination decline seen in spinocerebellar degeneration.
- What could go wrong
- This is a confirmatory Phase 3 trial, but success is not guaranteed. The drug may fail to outperform placebo, and side effects are possible. Results will need to be confirmed in this specific group of Japanese patients.
Why investors are watching
Kissei Pharmaceutical is running a late-stage, Phase III trial of its drug KPS-0373 in 142 patients with spinocerebellar degeneration, a rare disease that affects coordination and movement. For a small company like Kissei, this single readout carries outsized weight because the drug's approval and future revenue depend on it. The trial is double-blind and placebo-controlled, meaning researchers will compare the drug against a dummy treatment to see if it truly slows or improves the disease.
If it works: If the trial shows KPS-0373 works and is safe, Kissei could gain a new approved treatment for a disease with few options, which would give the company a new product to sell. A positive result could also strengthen the company's pipeline and its position in the rare-disease market.
If it fails: Phase III trials often fail, and a negative or unclear result would likely end KPS-0373's development, leaving Kissei without this potential product. A delay in the readout would also keep the company waiting longer for an answer, with no revenue from the drug in the meantime.
AI-written from the trial record. Speculative, and not investment advice.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 142 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2025
- Expected to finish
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Mar 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Research Site
Tokyo and Other Japanese Cities, Japan