---
title: Gene therapy slows Parkinson progression
nct_id: NCT07216066
phase: PHASE1
status: RECRUITING
sponsor: Regeneron Pharmaceuticals
study_type: INTERVENTIONAL
canonical_url: "https://parkinsonspathways.com/trial/NCT07216066"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT07216066"
last_fetched: "2026-05-10T14:05:02.916Z"
source: "Parkinson's Pathways (curated)"
---
# Gene therapy slows Parkinson progression

**Goal (in five words):** Gene therapy slows Parkinson progression

**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

**Trial ID:** [NCT07216066](https://clinicaltrials.gov/study/NCT07216066)

## Key Facts

- **Phase:** PHASE1
- **Status:** RECRUITING
- **Study Type:** INTERVENTIONAL
- **Sponsor:** Regeneron Pharmaceuticals
- **Target Enrollment:** 46 participants
- **Start Date:** 2025-12-05
- **Completion Date:** 2029-06-07
- **Conditions:** Parkinsons Disease
- **Interventions:** ALN-SNCA, Placebo
- **Intervention Types:** DRUG

## Summary For Families

The goal is to test whether lowering production of alpha-synuclein in the central nervous system is safe and biologically active in people with early Parkinson's, since alpha-synuclein buildup is linked to disease progression. The approach gives single ascending intrathecal doses of ALN-SNCA into the cerebrospinal fluid; ALN-SNCA is an RNA-based drug designed to reduce SNCA (alpha-synuclein) production in brain cells, and it is studied for safety, drug levels, and biological effects rather than immediate symptom relief, so it does not directly replace or change how levodopa works. The trial is enrolling people aged 50 to 80 with Parkinson's diagnosed within the last 4 years, who are either not yet on dopaminergic meds or have been on a stable oral dopaminergic regimen for at least 3 months, and who can safely undergo lumbar puncture and brain MRI; people with major medical issues, prior gene/cell or surgical PD treatments, or bleeding risks are excluded.

## Eligibility

- **Minimum age:** 50 Years
- **Maximum age:** 80 Years
- **Sex:** ALL

### Full 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
```

## Locations (2)

- Montreal Neurological Institute and Hospital, Montreal, Quebec, Canada _(45.5088, -73.5878)_
- Center for Human Drug Research, Leiden, South Holland, Netherlands _(52.1583, 4.4931)_

## Central Contacts

- Clinical Trials Administrator — (CONTACT) — 844-734-6643 — clinicaltrials@regeneron.com

---

*Canonical: https://parkinsonspathways.com/trial/NCT07216066*  
*HTML version: https://parkinsonspathways.com/trial/NCT07216066*  
*Source data: https://clinicaltrials.gov/study/NCT07216066*