Repurposing Lithium for Parkinson's Disease: a RCT

NCT06339034 | Active Not Recruiting Phase 1

• 1 other identifier: STUDY
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This study will examine the effects of lithium 20mg/day compared to placebo on MRI and blood-based biomarkers among 20 early-stage Parkinson's disease patients.

Conditions

Parkinson Disease

Keywords

Parkinson's; Lithium; Free water; Disease-modifying therapy

Outcome Measures

Primary Outcomes (3)

• MRI-derived free water (FW) levels.

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

  Change from baseline (BL) to 24 week

• Peripheral blood mononuclear cell (PBMC) nuclear receptor-related 1 protein (Nurr1) mRNA expression

  PBMC Nurr1 mRNA expression using Taqman PCR.

  Change from baseline (BL) to 24 week

• Serum neurofilament light chain (NfL)

  Serum NfL assessed using SIMOA platform by Quanterix (Lexington, MA)

  Change from baseline (BL) to 24 week

Secondary Outcomes (13)

• PBMC superoxide dismutase type-1 (SOD-1) mRNA expression

  Change from baseline (BL) to 24 week

• PBMC pS9/total glycogen synthase kinase-3B (GSK-3B) ratio

  Change from baseline (BL) to 24 week

• PBMC pThr308 and pS473/total protein kinase B (Akt) ratios

  Change from baseline (BL) to 24 week

• Serum interleukin-6

  Change from baseline (BL) to 24 week

• Serum glial fibrillary acidic protein (GFAP)

  Change from baseline (BL) to 24 week

Other Outcomes (1)

• Adverse events

  Throughout 24 week study

Study Arms (2)

Lithium

ACTIVE COMPARATOR

Lithium: 10mg, 2x/day

Dietary Supplement: Lithium

Placebo

PLACEBO COMPARATOR

Identical capsules filled with cellulose: 10mg, 2x/day

Other: Placebo

Interventions

Lithium DIETARY_SUPPLEMENT

5mg of elemental lithium/capsule

Lithium

Placebo OTHER

Cellulose-filled capsules

Placebo

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• State University of New York at Buffalo: lead
• Cure Parkinson's: collaborator

Study Sites (1)

UBMD Neurology

Williamsville, New York, 14221, United States

Location

Related Publications (1)

• Guttuso T Jr, Shepherd R, Frick L, Feltri ML, Frerichs V, Ramanathan M, Zivadinov R, Bergsland N. Lithium's effects on therapeutic targets and MRI biomarkers in Parkinson's disease: A pilot clinical trial. IBRO Neurosci Rep. 2023 May 7;14:429-434. doi: 10.1016/j.ibneur.2023.05.001. eCollection 2023 Jun.

  PMID: 37215748 BACKGROUND

MeSH Terms

Conditions

Parkinson Disease

Interventions

Lithium

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Metals, Alkali; Elements; Inorganic Chemicals; Metals, Light; Metals

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Model Details: Double-blind, placebo-controlled

Study Record Dates

• First Submitted: March 24, 2024
• First Posted: April 1, 2024
• Study Start: July 3, 2024
• Primary Completion: December 1, 2025
• Study Completion: February 1, 2026
• Last Updated: July 8, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)