Parkinsons DiseasePhase 1NCT07216066
First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)
A Phase 1 study testing ALN-SNCA in people with Parkinsons Disease, run by Regeneron Pharmaceuticals at 3 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 50 to 80 years
- Conditions the study is for
- Parkinsons Disease
- What is being tested?
- ALN-SNCA
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 5 Dec 2025to 7 Jun 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Show all 9Show fewer
- 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
Trial locations and contacts
3 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 2 countries
Canada2 sites, 2 recruiting
- Recruiting
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
- Recruiting
The Ottawa Hospital - Civic Campus
Ottawa, Ontario, K1Y 4E9
Netherlands1 site, 1 recruiting
- Recruiting
Center for Human Drug Research
Leiden, South Holland, 2333 CL
About this study
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
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.
Read more
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
- Conditions
- Parkinsons Disease
- Being tested
- ALN-SNCA, Placebo
- Keywords
- Early Parkinsons disease, Motor and non-motor brain systems, Bradykinesia plus, Resting tremor, Rigidity, Postural instability, Cognitive impairment, Mood disorders, Sleep disturbances, Autonomic dysfunction
- Medical subject headings
- Parkinson Disease, Tremor, Muscle Rigidity, Cognitive Dysfunction, Mood Disorders, Parasomnias, Primary Dysautonomias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-SNCADrug
Administered per the protocol
PlaceboDrug
Administered per the protocol
| Study group | Role | What they receive |
|---|---|---|
| ALN-SNCA Dose Escalation | Experimental | Not described Receives: Drug: ALN-SNCA |
| Matching Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of Treatment-Emergent Adverse Events (TEAEs)
Measured: Through Week 80
Severity of TEAEs
Measured: Through Week 80
Secondary outcomes (8)
Concentration of alpha (a)-synuclein protein in CSF
Measured: Through Week 48
Change from baseline in concentration of a-synuclein protein in CSF
Measured: Through Week 48
Concentration of ALN-SNCA in plasma
Measured: Through Week 48
Concentration of major metabolites in plasma
Measured: Through Week 48
Concentration of ALN-SNCA in CSF
Measured: Through Week 48
Concentration of major metabolites in CSF
Measured: Through Week 48
Fraction [fc] of ALN-SNCA excreted in urine
Measured: Over 24 hours
Fc of major metabolites excreted in urine
Measured: Over 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2025
- Study start5 Dec 2025
- Primary completion7 Jun 2029 (estimated)
- Study completion7 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinsons DiseaseStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 46 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07216066
- Sponsor's protocol ID
- ALN-SNCA-PD-2482
- Other IDs
- 2025-521431-37-00, 2025
- Status last verified
- Sep 2026
- First submitted
- 9 Oct 2025
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07216066
- Acronym / sponsor ID
- ALN-SNCA-PD-2482
- Conditions
- Parkinsons Disease
- Keywords
- Early Parkinsons disease, Motor and non-motor brain systems, Bradykinesia plus, Resting tremor, Rigidity, Postural instability, Cognitive impairment, Mood disorders
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 46 participants (estimated)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 5 Dec 2025 (actual)
- Primary completion
- 7 Jun 2029 (estimated)
- Study completion
- 7 Jun 2029 (estimated)
- First posted
- 14 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALN-SNCA Dose Escalation | Experimental | Drug: ALN-SNCA |
| Matching Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 50 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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:
- BMI ≤35 kg/m^2 at time of screening visit
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALN-SNCAAdministered per the protocol | Drug | ALN-SNCA Dose Escalation |
| PlaceboAdministered per the protocol | Drug | Matching Placebo |
Primary outcome
Occurrence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Through Week 80
Severity of TEAEs
Time frame: Through Week 80
8 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2026.
- Individual participant dataSharing planned: Yes. All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
Related trials for Parkinsons Disease
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Not yet recruiting
Compulsive Eating and Brain's Reward System in Different Pathologies
PARKINSON DISEASE (Disorder), Bipolar 1 Disorder
Not yet recruiting
Evaluating the Effect of the Zeen Mobility Device on Clinical Practicality, Safety, and Impact (Via Assessments of Walking, Standing Tolerance, Balance, Endurance, Effort, and Participation in Daily Tasks) in an Outpatient Rehabilitation Setting for Individuals With Mobility Impairments.
Stroke, Parkinson's Disease (PD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Clinical Trial Management study director
Regeneron Pharmaceuticals
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALN-SNCA in people with Parkinsons Disease, run by Regeneron Pharmaceuticals at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Canada, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials Administrator (844-734-6643, clinicaltrials@regeneron.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of Treatment-Emergent Adverse Events (TEAEs) (time frame: Through Week 80). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Sep 2026.