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Recruiting NCT07216066

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

Phase I Interventional Parkinsons Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ALN-SNCA, Placebo.
Who it may be relevant to
Registry conditions: Parkinsons Disease. Basic parameters: 50 years — 80 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Canada, Netherlands
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Interventions

  • Drug ALN-SNCA
    Administered per the protocol
  • Drug Placebo
    Administered per the protocol

Primary outcome measures

  • Occurrence of Treatment-Emergent Adverse Events (TEAEs) [Time frame: Through Week 80]
  • Severity of TEAEs [Time frame: Through Week 80]
Secondary outcome measures (8)
  • Concentration of alpha (a)-synuclein protein in CSF [Time frame: Through Week 48]
  • Change from baseline in concentration of a-synuclein protein in CSF [Time frame: Through Week 48]
  • Concentration of ALN-SNCA in plasma [Time frame: Through Week 48]
  • Concentration of major metabolites in plasma [Time frame: Through Week 48]
  • Concentration of ALN-SNCA in CSF [Time frame: Through Week 48]
  • Concentration of major metabolites in CSF [Time frame: Through Week 48]
  • Fraction [fc] of ALN-SNCA excreted in urine [Time frame: Over 24 hours]
  • Fc of major metabolites excreted in urine [Time frame: Over 24 hours]

Eligibility criteria

Inclusion criteria

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

Exclusion criteria

  • Medical history indicating a Parkinsonian syndrome other than Parkinson's disease, as defined in the protocol
  • 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
  • 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
  • Any contraindications to undergo a brain Magnetic Resonance Imaging (MRI)
  • An established allergy or intolerance to lidocaine anesthetic, as defined in the protocol
  • History of intolerance to Intrathecal (IT) injection(s)
  • 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

NOTE: Other Protocol-defined Inclusion/Exclusion Criteria Apply

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Sequential
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Canada · 1 center
  • Montreal Neurological Institute and Hospital — Montreal
Netherlands · 1 center
  • Center for Human Drug Research — Leiden

Identifiers

NCT: NCT07216066 · ALN-SNCA-PD-2482 · 2025-521431-37-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗