RecruitingPhase 1NCT07216066

First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)

First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Ascending Doses of Intrathecally Administered ALN-SNCA in Participants With Early Parkinson's Disease

Sponsor

Regeneron Pharmaceuticals

Enrollment

46 participants

Start Date

Dec 5, 2025

Study Type

INTERVENTIONAL

Conditions

Parkinsons Disease

Summary

This study is researching an experimental drug called ALN-SNCA (called "study drug"). The study is focused on people with early Parkinson's disease, a disorder of the nervous system that affects movement. Parkinson's disease is caused by a gradual loss of nerve cells in the brain, especially those due to the harmful build-up of a protein called α-synuclein. The aim of the study is to see if the study drug is safe and tolerated well enough to continue testing it in future studies and what side effects may happen from taking the study drug. The study is looking at several other research questions, including: * Whether the study drug can lower the level of α-synuclein protein in the Cerebrospinal Fluid (also referred to as "CSF", the fluid that surrounds the brain and spinal cord) * How much study drug is in the blood, urine, and CSF at different times * Compatible research to better understand the study drug (ALN-SNCA) and Parkinson's disease, including (but not limited to), whether the study drug can slow down the progression of Parkinson's disease symptoms

Eligibility

Min Age: 50 YearsMax Age: 80 Years

Plain Language Summary

Simplified for easier understanding

This study is the first-in-human trial of a new drug called ALN-SNCA, which targets a protein called alpha-synuclein (linked to Parkinson's disease). The drug is given by injection into the spinal fluid and aims to reduce the build-up of this protein in the brain in people with early Parkinson's disease. **You may be eligible if...** - You have been diagnosed with Parkinson's disease according to established criteria (with specific movement symptoms like slowness, tremor, or stiffness) - Your diagnosis was made 4 years ago or less - You are either not currently on Parkinson's medication or have been on a stable dose for at least 3 months - Your BMI is 35 or below **You may NOT be eligible if...** - Your movement disorder is caused by something other than Parkinson's disease - You have significant heart, lung, liver, kidney, blood, psychiatric, or neurological problems (other than Parkinson's) - You have had brain or spine surgery, deep brain stimulation, or gene/cell therapy for Parkinson's - You have a bleeding disorder or allergy to local anesthetics used in the injection procedure - MRI scans are contraindicated for you Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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