Experimental protein therapy targets rare brain disease
NCT ID NCT07476352
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Beijing Tiantan Hospital
Beijing, Beijing Municipality, 100070, China