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ALN-SNCA for Early Parkinson's Disease

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Study Aim

The study aims to evaluate the safety and tolerability of ALN-SNCA as a treatment for individuals with early-stage Parkinson's disease.

What is being tested

ALN-SNCA

+ Placebo

Drug
Who is being recruted

Synucleinopathies+25

+ Autonomic Nervous System Diseases

+ Basal Ganglia Diseases

From 50 to 80 Years
+12 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Placebo-ControlledPhase 1
Interventional
Study Start: December 2025
See protocol details

Summary

Principal SponsorRegeneron Pharmaceuticals
Study ContactClinical Trials Administrator
Last updated: January 30, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: December 5, 2025

Actual date on which the first participant was enrolled.

This clinical trial is evaluating a new experimental drug called ALN-SNCA for people with early-stage Parkinson's disease, which affects movement due to nerve cell loss in the brain. The focus is on understanding whether this drug can be safely administered to patients by checking if it is tolerable and what potential side effects may occur. Parkinson's disease involves the harmful accumulation of a protein called α-synuclein, and this study is important because reducing this protein could potentially slow down the disease's progression, offering hope for more effective treatments in the future. Participants in the study receive the ALN-SNCA drug through an injection into the spinal fluid. Researchers monitor how the drug affects the levels of the α-synuclein protein in the cerebrospinal fluid, which surrounds the brain and spinal cord. They also measure how much of the drug is present in the blood, urine, and cerebrospinal fluid at different times to understand its distribution in the body. By doing this, the study aims to gather comprehensive data on the drug's safety and its potential effects on disease symptoms, paving the way for further research.

Official TitleFirst-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
NCT07216066
Principal SponsorRegeneron Pharmaceuticals
Study ContactClinical Trials Administrator
Last updated: January 30, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

46 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.



Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 50 to 80 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

SynucleinopathiesAutonomic Nervous System DiseasesBasal Ganglia DiseasesMental DisordersBrain DiseasesCentral Nervous System DiseasesCognition DisordersMovement DisordersMuscle HypertoniaMuscle RigidityMuscular DiseasesMusculoskeletal DiseasesNervous System DiseasesNeurologic ManifestationsParkinson DiseaseSigns and SymptomsSleep Wake DisordersPathological Conditions, Signs and SymptomsTremorNeurodegenerative DiseasesMood DisordersNeurocognitive DisordersParasomniasParkinsonian DisordersDyskinesiasNeuromuscular ManifestationsPrimary DysautonomiasCognitive Dysfunction

Criteria

4 inclusion criteria required to participate
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

A diagnosis of Parkinson's disease for 4 years or less at the screening visit

BMI ≤35 kg/m^2 at time of screening visit

Participant must meet one of the following criteria: 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 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

8 exclusion criteria prevent from participating
Medical history indicating a Parkinsonian syndrome other than Parkinson's disease, as defined in the protocol

Current history of bleeding diatheses that would increase risk of bleeding upon LP

Has undergone gene therapy, cell therapy or surgical treatment, including deep brain stimulation, for Parkinson's disease

Any contraindications to undergo a brain Magnetic Resonance Imaging (MRI)

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

2 intervention groups are designated in this study

50% chance of being blinded to the placebo group

Treatment Groups

Group I

Experimental

Group II

Placebo

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Recruiting

Center for Human Drug Research

Leiden, NetherlandsOpen Center for Human Drug Research in Google Maps
Recruiting
One Study Center