ALN-SNCA in adults with early Parkinson's disease
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
This trial will test ALN-SNCA in adults with early Parkinson's disease to see if it is safe, well tolerated, and lowers α-synuclein in cerebrospinal fluid.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 46 (estimated) |
| Ages | 50 Years to 80 Years |
| Sex | All |
| Sponsor | Regeneron Pharmaceuticals Industry-sponsored |
| Locations | 2 sites (Montreal, Quebec and 1 other locations) |
| Trial ID | NCT07216066 on ClinicalTrials.gov |
What this trial studies
This is a first-in-human, Phase 1 interventional trial of the investigational agent ALN-SNCA with placebo control to characterize safety and tolerability in adults with early Parkinson's disease. Participants will receive ALN-SNCA or placebo and undergo serial monitoring of adverse events, pharmacokinetics in blood and urine, and cerebrospinal fluid measures including α-synuclein. The study enrolls people diagnosed with Parkinson's disease for 4 years or less who are either treatment‑naive or on a stable oral dopaminergic regimen. The trial is sponsored by Regeneron and conducted at specialized clinical pharmacology/neuroscience centers in Montreal and Leiden.
Who should consider this trial
Good fit: Adults diagnosed with Parkinson's disease within the past 4 years who have bradykinesia plus another cardinal sign and who are either not yet on oral dopaminergic therapy or are on a stable oral dopaminergic regimen are ideal candidates.
Not a fit: People with Parkinsonism due to other causes, disease duration longer than 4 years, or those unable or unwilling to undergo lumbar puncture and repeated site visits are unlikely to benefit from this trial.
Why it matters
Potential benefit: If successful, ALN-SNCA could lower pathogenic α-synuclein and potentially slow progression of Parkinson's disease.
How similar studies have performed: Preclinical work supports α-synuclein lowering as a therapeutic strategy, but first-in-human clinical data for ALN-SNCA are novel and clinical benefit has not yet been shown.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Montreal, Quebec and 1 other locations
- Montreal Neurological Institute and Hospital — Montreal, Quebec, Canada (Recruiting)
- Center for Human Drug Research — Leiden, South Holland, Netherlands (Recruiting)
Study contacts
- Study coordinator: Clinical Trials Administrator
- Email: clinicaltrials@regeneron.com
- Phone: 844-734-6643
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.