Experimental protein therapy targets rare brain disease
NCT ID NCT07476352
First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 2 times
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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 60 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2026
- Expected to finish
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Oct 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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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30 to 75 years
- 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: * 1\. Age between 30 and 75 years inclusive, either sex; * 2\. Clinically established or clinically probable MSA (including both MSA-C and MSA-P subtypes); * 3\. Ability to walk independently or with the aid of a walking device for at least 10 meters; * 4\. Provision of written informed consent. Exclusion Criteria: * 1\. Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA; * 2\. Other significant pathological findings on brain MRI at screening, including but not limited to: cerebral hemorrhage, acute cerebral infarction, aneurysm, vascular malformation, infectious lesions, brain tumors, or other space-occupying lesions (meningiomas or arachnoid cysts with a maximum diameter \<1 cm do not require exclusion); * 3\. Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents; * 4\. Known history of allergies to biologic agents, such as proteins or cell-based products; * 5\. Receipt of any vaccination within the past 1 month; * 6\. Patients with a prior definitive diagnosis of malignancy or those currently receiving anti-tumor drug therapy; * 7\. Patients with a prior definitive diagnosis of epilepsy or those currently taking anti-epileptic drugs; * 8\. Concurrent severe hepatic insufficiency, renal insufficiency, or severe cardiac insufficiency (severe hepatic insufficiency defined as ALT ≥1.5 times the upper limit of normal or AST ≥1.5 times the upper limit of normal; severe renal insufficiency defined as serum creatinine \[CRE\] ≥1.5 times the upper limit of normal or estimated glomerular filtration rate \[eGFR\] \<40 mL/min/1.73 m²; severe cardiac insufficiency defined as NYHA class 3-4), or any significant abnormalities on physical examination, vital signs, laboratory tests, or electrocardiogram that, in the investigator's opinion, require further examination or treatment, or may interfere with the study procedures or safety; * 9\. Patients with a history of alcohol or substance abuse, or alcohol or substance dependence within the past 2 years; * 10\. Patients diagnosed with psychiatric disorders according to DSM-V criteria, or those with obvious suicidal intent; * 11\. Patients who are pregnant, lactating, or have the potential to become pregnant, or those planning a pregnancy; * 12\. Inability to comply with follow-up assessments due to other reasons; * 13\. Patients deemed by the investigator to be unsuitable for participation in this study.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Beijing Tiantan Hospital
RECRUITINGBeijing, Beijing Municipality, 100070, China
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