Parkinson's disease affects about 1 million people in the US, causing tremor, slowness, stiffness, and balance problems as dopamine-producing brain cells die. Current treatment relies mainly on levodopa and related drugs that replace dopamine, plus deep brain stimulation for some people. Symptoms usually progress over years despite treatment.
What's actually going on in research
Trials are testing drugs that may slow or stop the disease itself, not just manage symptoms. These include GLP-1 receptor agonists like exenatide and lixisenatide, gene therapies delivering growth factors to protect brain cells, and drugs targeting alpha-synuclein, the protein that clumps in Parkinson's. Other studies focus on improving motor fluctuations, freezing, and non-motor problems like constipation and cognitive decline.
Alpha-synuclein targeting
Antibodies and small molecules aim to clear or prevent the spread of misfolded alpha-synuclein protein. Several drugs have reached mid-stage trials testing whether reducing this protein slows disease progression.
GLP-1 receptor agonists
Diabetes drugs like exenatide and lixisenatide may protect dopamine neurons through anti-inflammatory effects. Phase 2 trials showed signs of slowing motor decline, and larger trials are now underway.
Gene therapy
One-time delivery of protective genes like GDNF or enzymes to restore dopamine production is being tested. Early results show safety, with ongoing trials measuring whether symptoms improve long-term.
What to know before you search
Eligibility typically depends on time since diagnosis, medication regimen, motor symptom severity, and whether you have certain genetic variants like GBA or LRRK2 mutations.
What types of trials are currently open
- Disease-modifying trials — Testing drugs that aim to slow or stop Parkinson's progression by protecting brain cells or clearing toxic proteins, not just treating symptoms.
- Symptomatic trials — Testing new drugs or devices to better control tremor, stiffness, motor fluctuations, or non-motor symptoms like constipation and low blood pressure.
- Gene therapy trials — One-time brain delivery of genes that may protect dopamine neurons or restore dopamine production.
- Device trials — Testing new deep brain stimulation targets, non-invasive brain stimulation, or devices to improve gait and freezing.
- Natural history studies — Following people with early Parkinson's to understand how the disease progresses and identify biomarkers that predict outcomes.
Recently added Parkinson's Disease trials
Prospective Multicenter Registry Study of Multiple System Atrophy in China
Multiple system atrophy is a rare, rapidly progressive neurodegenerative disease characterized by variable combinations of parkinsonism, cerebellar ataxia, and autonomic dysfunction. Existing natural history studies from North America, Europe, and Japan suggest that clinical phenotypes and disease progression may differ across populations. However, comprehensive multicenter prospective data from Chinese patients with multiple system atrophy remain limited. This prospective multicenter registry study aims to describe the clinical characteristics, longitudinal progression, and outcomes of Chinese patients with multiple system atrophy, to identify factors associated with disease progression and prognosis, and to establish a longitudinal cohort for future biomarker validation and clinical trial design.
Striatal and Extra-Striatal Cholinergic Terminal Density in LRRK2-PD Mutation
This study explores how a specific genetic mutation of leucine-rich repeat kinase 2 (LRRK2) affects individuals with Parkinson's disease (PD), comparing those with the mutation to others with Parkinson's disease and without the mutation (iPD). Participants will complete positron emission tomography (PET) and magnetic resonance imaging (MRI) brain imaging, cognitive tests, motor tests, sensory tests, and questionnaires. The aims of this study are to compare brain chemicals in LRRK2 PD patients with iPD patients and to correlate brain chemicals with motor and cognitive tests in LRRK2 PD and iPD patients.
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