Plain-English translation of NCT06478238 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This trial is testing as a potential treatment for spastic paraplegia 56 (SPG56), a rare inherited condition that affects the spinal cord and causes muscle weakness and stiffness. Right now, there is no standard treatment for this disease, so researchers want to see if this medication might help patients and whether it is safe to use.
SPG56 is caused by a genetic mutation and currently has no proven treatment options. This trial exists to explore whether the medication could slow down or improve symptoms in people living with this rare condition.
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You would first be admitted for a 5-day hospital stay to receive by IV infusion. After that, you would take the medication by mouth twice daily at home while attending regular clinical evaluations. This phase of treatment and close monitoring continues during your hospital stay, and then you would take the medication long-term at home. The entire study lasts 6 years, with ongoing appointments to check how you're doing and assess whether the treatment is helping.
AI-generated summary from trial data · Jun 13, 2026 · Not medical advice
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