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

Regeneron Pharmaceuticals

Status and phase

Enrolling

Phase 1

Conditions

Parkinsons Disease

Treatments

Drug: ALN-SNCA

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT07216066

ALN-SNCA-PD-2482

2025-521431-37-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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

Enrollment

46 estimated patients

Sex

All

Ages

50 to 80 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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

Key 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