Health TrialsbyHealthPanel

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

ConditionParkinsons Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age50-80
SponsorRegeneron Pharmaceuticals

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

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

Disqualifiers

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)

Trial design

Design model

Sequential

Treatments tested in this trial

  • ALN-SNCA

    Drug

    Administered per the protocol

  • Placebo

    Drug

    Administered per the protocol

Treatment groups

46 Participants
are divided into 2 treatment groups
Group A: ALN-SNCA Dose EscalationExperimental treatment 1 intervention
Group B: Matching PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Occurrence of Treatment-Emergent Adverse Events (TEAEs)

Time frame
Through Week 80
2

Severity of TEAEs

Time frame
Through Week 80

Secondary outcomes

1

Concentration of alpha (a)-synuclein protein in CSF

Time frame
Through Week 48
2

Change from baseline in concentration of a-synuclein protein in CSF

Time frame
Through Week 48
3

Concentration of ALN-SNCA in plasma

Time frame
Through Week 48
4

Concentration of major metabolites in plasma

Time frame
Through Week 48

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Regeneron Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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