NCT07216066 First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)
| NCT ID | NCT07216066 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Regeneron Pharmaceuticals |
| Condition | Parkinsons Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 46 participants |
| Start Date | 2025-12-05 |
| Primary Completion | 2029-06-07 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 46 participants in total. It began in 2025-12-05 with a primary completion date of 2029-06-07.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
Eligibility 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
Contact & Investigator
Clinical Trial Management
STUDY DIRECTOR
Regeneron Pharmaceuticals
Frequently Asked Questions
Who can join the NCT07216066 clinical trial?
This trial is open to participants of all sexes, aged 50 Years or older, up to 80 Years, studying Parkinsons Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07216066 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07216066 currently recruiting?
Yes, NCT07216066 is actively recruiting participants. Contact the research team at clinicaltrials@regeneron.com for enrollment information.
Where is the NCT07216066 trial being conducted?
This trial is being conducted at Montreal, Canada, Leiden, Netherlands.
Who is sponsoring the NCT07216066 clinical trial?
NCT07216066 is sponsored by Regeneron Pharmaceuticals. The principal investigator is Clinical Trial Management at Regeneron Pharmaceuticals. The trial plans to enroll 46 participants.
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