Experimental protein therapy targets rare brain disease

NCT ID NCT07476352

Not yet recruiting Disease control Sponsor: yilong Wang Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tests a drug called ALT001, a nerve repair protein, in 60 people with multiple system atrophy (MSA), a rare and progressive brain disorder. The study is open-label, meaning everyone knows they are getting the drug, and it focuses on safety and whether the drug shows any signs of helping. Participants receive ALT001 through a spinal injection or IV.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: ALT001 (a nerve repair protein derived from stem cells)
What this could lead to: If it works, this could point toward a treatment that slows or repairs nerve damage in multiple system atrophy.
What could go wrong: This is a very early (Phase 1), small, open-label trial with no placebo group. Results may not confirm effectiveness, and safety is still being evaluated.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

multiple system atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MULTIPLE SYSTEM ATROPHY

Contacts and locations

Locations

Beijing Tiantan Hospital

Beijing, Beijing Municipality, 100070, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.