MedPath

Repurposing Lithium for Parkinson's Disease

Phase 1
Active, not recruiting
Conditions
Parkinson's Disease
Interventions
Dietary Supplement: Lithium aspartate
Registration Number
NCT06099886
Lead Sponsor
State University of New York at Buffalo
Brief Summary

This study will examine the effects of lithium aspartate 30-45mg/day on MRI biomarkers and blood-based therapeutic targets among 15 early-stage Parkinson's disease patients.

Detailed Description

In observational studies, small daily doses of lithium have been associated with a 77% reduced risk of developing Parkinson's disease (PD). In addition, lithium therapy has been effective in preventing neuronal death and behavioral symptoms in several PD animal models. Recently, our group has shown 24-weeks of low-dose lithium aspartate therapy 45mg/day in PD to engage blood-based and the MRI disease progression biomarker, free water, to a greater extent than 15mg/day or 150mg/day of lithium carbonate. However, these blood-based and MRI biomarker findings stem from only four and two PD patients, respectively, who received lithium aspartate 45mg/day. In addition, two other PD patients receiving this dosage withdrew from the study due to side effects of sedation and dizziness. Subsequently, one of these patients who withdrew resumed lithium aspartate at 30mg/day and reported no side effects. Although these findings suggest that this dosage of lithium aspartate has positive effects on PD biomarkers, data from a larger number of PD patients will be required to justify conducting a larger, randomized controlled trial (RCT). The proposed study will enroll 15 additional PD patients over five months who will receive lithium aspartate 30-45mg/day for 24 weeks ensuring that the study will be completed within 12 months. The dosage will be slowly titrated in each patient up to the maximum tolerated dosage in this range. Blood-based biomarkers and MRIs will be assessed at baseline and 24 weeks. It is anticipated that a similar magnitude of biomarker engagement will be observed among these additional 15 patients as was seen in the handful from the pilot study. Such findings would provide strong preliminary evidence to support conducting a larger RCT including both clinical and biomarker outcomes. Positive results from such a RCT would support lithium aspartate as a disease-modifying therapy for PD.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
17
Inclusion Criteria

Have PD for <4 years diagnosed by a movement disorder specialist. Have normal thyroid and renal function at the screening visit. Have no previous exposure to lithium therapy. Have no history of brain surgery. Have no hx of brain imaging findings suggesting another neurological condition besides PD.

Have no use of tobacco or THC products for >1 year. Have stable PD medications for >30 days without current need for adjustments in the investigator's opinion.

Have stable psychiatric and diuretic medications for >60 days with no anticipated need for changes for at least 24 weeks.

Have no active medical or psychiatric condition that may interfere with study procedures in the investigator's opinion.

Exclusion Criteria

Have PD for >4 years or does not have PD. Have abnormal normal thyroid and renal function at the screening visit. Have previous exposure to lithium therapy. Have history of brain surgery. Have hx of brain imaging findings suggesting another neurological condition besides PD.

Have use of tobacco or THC products within the past year. Have PD medication adjustments within 30 days or needs PD medication adjustments in the investigator's opinion.

Have psychiatric or diuretic medication adjustments within the last 60 days or is anticipated to need changes over next 24 weeks.

Have active medical or psychiatric condition that may interfere with study procedures in the investigator's opinion.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Lithium aspartateLithium aspartateLithium aspartate capsules will be titrated in each patient to the maximum tolerated dosage between 30-45mg/day.
Primary Outcome Measures
NameTimeMethod
MRI-derived free water (FW) levelsChange from baseline (BL) to 24 weeks.

FW in the posterior substantia nigra (pSN), dorsomedial nucleus of the thalamus (DMN-T) and the nucleus basalts of Meynert (nbM).

Peripheral blood mononuclear cell (PBMC) nuclear receptor-related 1 protein (Nurr1) mRNA expression.Change from BL to 24 weeks.

PBMC Nurr1 mRNA expression using Taqman PCR.

Secondary Outcome Measures
NameTimeMethod
Serum neurofilament light (NfL)Change from BL to 24 weeks.

Assessed using SIMOA platform

Serum glial fibrillary acidic protein (GFAP)Change from BL to 24 weeks.

Assessed using SIMOA platform

PBMC superoxide dismutase type-1 (SOD-1) mRNA expressionChange from BL to 24 weeks.

PBMC SOD-1 mRNA expression using Taqman PCR.

PBMC pS9/total glycogen synthase kinase-3B (GSK-3B) ratioChange from BL to 24 weeks.

Assessed using ELISA

PBMC pThr308 and pS473/total protein kinase B (Akt) ratiosChange from BL to 24 weeks.

Assessed using ELISA

Serum interleukin-6Change from BL to 24 weeks.

Assessed using ELISA

Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III (Motor Examination)Change from BL to 24 weeks.

Assessed in the "on" state. Score range 0-132 with higher scores indicating worse outcomes.

Montreal Cognitive Assessment (MoCA)Change from BL to 24 weeks.

Score range 0-30 with higher scores indicating better outcomes.

Parkinson's Anxiety ScaleChange from BL to 24 weeks.

Score range 0-48 with higher scores indicating worse outcomes.

Geriatric Depression Scale-15Change from BL to 24 weeks.

Score range 0-15 with higher scores indicating worse outcomes.

Fatigue Severity ScaleChange from BL to 24 weeks.

Score range 9-63 with higher scores indicating worse outcomes.

Insomnia Severity IndexChange from BL to 24 weeks.

Score range 0-28 with higher scores indicating worse outcomes.

Parkinson's Disease Questionnaire-8Change from BL to 24 weeks.

Score range 0-32 with higher scores indicating worse outcomes.

Levodopa equilavent doseChange from BL to 24 weeks.

Higher scores indicate higher dose of dopaminergic therapy.

Trial Locations

Locations (1)

University at Buffalo

🇺🇸

Williamsville, New York, United States

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