Efficacy of Oral Administration of Trehalose in Patients With Parkinson Disease

NCT05355064 | Unknown Phase 4

• 1 other identifier: Trehalose treatment in PD
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief Summary

Parkinson's disease (PD) is a neurodegenerative disease characterized by the neurodegeneration of substance nigra pars compacta (SNpc) and the formation of alpha-synuclein protein aggregates in neurons. Although most PD patients are sporadic, it is now clear that genetic factors contribute to the pathogenesis of PD. Indeed, LRRK2 G2019S mutation is one of the most common causes of familial PD. The phenotype corresponding to this mutation is a late-onset form of PD characterized by the accumulation of the N-ethylmaleimide sensitive factor (NSF) in neurons. It is due to a dysfunction of the physiological autophagy processes occurring at cellular level, mainly affecting autophagy mediated by chaperone proteins (Chaperon Mediated Autophagy, CMA), responsible for the clearance of alpha synuclein at the lysosomal level. This condition, although sensitive to treatment with L-DOPA and dopamine agonists, does not currently have any specific therapy. Recently, in a mouse model carrying the LRRK2 mutation, it has been demonstrated that treatment with trehalose is able to reduce the accumulation of NSF deposits in neurons of various brain areas such as the substantia nigra, striatum, cortex and hippocampus. The reduction of protein aggregates correlates with intracellular molecules related to the activation of apoptotic processes in damaged neurons. Moreover, it has been found a significant improvement in motor and cognitive performance. The aim of the present study is to evaluate the safety and tolerability of trehalose in two groups of patients affected by idiopathic PD and PD carrying the LRRK2 mutation, respectively. Moreover, the investigators will collect preliminary data on the effect that this molecule potentially has on disease course in both groups. The treatment duration will be 24 weeks and the overall study duration approximately 12 months. The populations observed will be composed of subjects affected by idiopathic PD and familial PD carrying the genetically confirmed LRRK2 mutation. Enrolled subjects will daily take trehalose in oral administration. Safety will be assessed by detecting any adverse events and analyzing blood chemistry parameters. The effect of trehalose will be evaluated through periodic clinical examinations, including the administration of specific scales and questionnaires.

Conditions

Parkinson Disease

Outcome Measures

Primary Outcomes (2)

• Clinical laboratory test results at each visit from baseline to follow-up period

  Summary statistics for laboratory tests will be presented at baseline and at each visit. The severity of select laboratory results will be graded according the Common Terminology Criteria for Adverse Events (CTCAE).

  1-36 weeks

• 1. Physical and neurological examination findings, at each visit.

  Body system (General appearance, Musculo-skeletal, Cardiovascular, Lungs, Abdomen, Neurological) findings that is judged by the investigator as a clinically significant change (worsening) compared to a baseline value will be considered an adverse event coded using MedDRA

  1-36 weeks

Secondary Outcomes (4)

• Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS)

  1-36 weeks

• Assessment of General Cognitive Functioning on the Mini Mental State Examination (MMSE)

  1- 36 weeks

• The differences between the MoCa test score from baseline to follow up period

  1-36 weeks

• Changes from baseline in the Quality of Life in Neurological Disorders ( NeuroQol) and EQ-5D-5L questionnaires

  1- 36 weeks

Study Arms (1)

Single arm

EXPERIMENTAL

Single treatment, no placebo.

Drug: Trehalose

Interventions

Trehalose DRUG

Enrolled subjects will daily take 4 g of trehalose in oral administration for 24 weeks.

Also known as: CITOPLAS

Single arm

Eligibility Criteria

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

You may qualify if:

• Ability to provide written informed consent;
• PD diagnosis according to UK Parkinson's Disease Society Brain Bank Clinical Diagnostic Criteria (UKPDS);
• Age between 18 and 80 years (inclusive);
• Hoehn \& Yahr staging \> 1;

You may not qualify if:

• Inability to provide written informed consent;
• Diagnosis of other concomitant neurodegenerative disease;
• Concomitant treatment with drugs similar to trehalose;
• Hypersensitivity or intolerance to the active substance administered;
• Severe swallowing problems;
• Participation in other interventional studies within 30 days from the screening;
• Other medical conditions that can interfere with results or endanger the participant.

Sponsors & Collaborators

• Neuromed IRCCS: lead

MeSH Terms

Conditions

Parkinson Disease

Interventions

Trehalose

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)

Glucans; Polysaccharides; Carbohydrates; Disaccharides; Oligosaccharides; Sugars

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: December 18, 2021
• First Posted: May 2, 2022
• Study Start: May 1, 2022
• Primary Completion: August 31, 2022
• Study Completion: May 1, 2023
• Last Updated: May 2, 2022

Record last verified: 2022-04

Data Sharing

• IPD Sharing: Will not share