New drug targets Parkinson's protein in first human test
NCT ID NCT07216066
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an experimental drug called ALN-SNCA in 46 people with early Parkinson's disease. The drug is designed to lower levels of a harmful protein, alpha-synuclein, in the brain. The main goal is to check if the drug is safe and tolerable, and to see how it moves through the body.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ALN-SNCA
- What this could lead to
- If it works, this could point toward a treatment that slows or stops Parkinson's disease progression by reducing harmful protein buildup in the brain.
- What could go wrong
- This is a very early, small Phase 1 study focused on safety, not effectiveness. The drug may not lower the protein enough or could cause side effects, and many early-stage drugs fail to advance.
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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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 46 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2025
- Expected to finish
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Jun 2029
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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50 to 80 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.
Key Inclusion Criteria: 1. Diagnosis of Parkinson's disease according to the Movement Disorder Society (MDS) criteria, as assessed by the investigator, with bradykinesia plus at least one of the other cardinal signs of Parkinson's disease (resting tremor, rigidity), without any other known or suspected cause of Parkinsonism 2. A diagnosis of Parkinson's disease for 4 years or less at the screening visit 3. Participant must meet one of the following criteria: 1. Currently not receiving any standard-of-care (SoC) therapy for Parkinson's disease, has not been on oral dopaminergic therapy (ie, levodopa, dopamine agonists, or Monoamine Oxidase B \[MAO-B\] inhibitors) prior to dosing, and is not anticipated to require SoC therapy for Parkinson's disease within approximately 6 months following dosing, or 2. Has been on a stable regimen of oral dopaminergic therapy for at least 3 months prior to dosing and is not anticipated to require dose adjustments within approximately 6 months following dosing 4. BMI ≤35 kg/m\^2 at time of screening visit Key Exclusion Criteria: 1. Medical history indicating a Parkinsonian syndrome other than Parkinson's disease, as defined in the protocol 2. Clinically significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, psychiatric or neurological disease (excluding Parkinson's disease) that, as assessed by the investigator, may confound the results of the study or pose an additional risk to the participant during study participation 3. Medical history of brain or spinal disease/injury that would interfere with the Lumbar Puncture (LP) procedure or CSF circulation, as defined in the protocol 4. Any contraindications to undergo a brain Magnetic Resonance Imaging (MRI) 5. An established allergy or intolerance to lidocaine anesthetic, as defined in the protocol 6. History of intolerance to Intrathecal (IT) injection(s) 7. Current history of bleeding diatheses that would increase risk of bleeding upon LP 8. Has undergone gene therapy, cell therapy or surgical treatment, including deep brain stimulation, for Parkinson's disease NOTE: Other Protocol-defined Inclusion/Exclusion Criteria Apply
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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
2 sites in 2 countries. 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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Center for Human Drug Research
RECRUITINGLeiden, South Holland, 2333 CL, Netherlands
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Montreal Neurological Institute and Hospital
RECRUITINGMontreal, Quebec, H3A 2B4, Canada
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