- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05355064
Efficacy of Oral Administration of Trehalose in Patients With Parkinson Disease
Efficacy of Oral Administration of Trehalose in Patients With Parkinson Disease in Both Idiopathic and Induced by LRRK2 Mutation
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.
Study Overview
Study Type
Enrollment (Anticipated)
Phase
- Phase 4
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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;
Exclusion Criteria:
- 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Single arm
Single treatment, no placebo.
|
Enrolled subjects will daily take 4 g of trehalose in oral administration for 24 weeks.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Clinical laboratory test results at each visit from baseline to follow-up period
Time Frame: 1-36 weeks
|
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.
Time Frame: 1-36 weeks
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Time Frame: 1-36 weeks
|
To compare the efficacy of trealose therapy as measured by the sum of the MDS-UPDRS Part III score changes from Baseline to Weeks 18, 36.
The score range is 0-132, where a higher score means more severe motor impairment.
|
1-36 weeks
|
Assessment of General Cognitive Functioning on the Mini Mental State Examination (MMSE)
Time Frame: 1- 36 weeks
|
The MMSE is a 30-point questionnaire that examines functions including registration (repeating named prompts), attention and calculation, recall, language, ability to follow simple commands and orientation.
The score ranges from 0 to 30 (cut-off = 24).
|
1- 36 weeks
|
The differences between the MoCa test score from baseline to follow up period
Time Frame: 1-36 weeks
|
The MoCa test is a one page 30 points test that can be done in 10 minutes in a routine visit.
The MoCa assesses several cognitive domains.
The maximum score is 30.
|
1-36 weeks
|
Changes from baseline in the Quality of Life in Neurological Disorders ( NeuroQol) and EQ-5D-5L questionnaires
Time Frame: 1- 36 weeks
|
NeuroQol will provide a means of assessing quality of life in patient populations with chronic diseases, such as PD.
Europeans Quality of life questionnaire 5 dimensions ,5 levels (EQ-5D-5L) is a standardized measure of health status which provides a simple, generic measure of health for clinical and economic appraisal.
|
1- 36 weeks
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Anticipated)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- Trehalose treatment in PD
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Parkinson Disease
-
National Heart, Lung, and Blood Institute (NHLBI)CompletedParkinson Disease 6, Early-Onset | Parkinson Disease (Autosomal Recessive, Early Onset) 7, Human | Parkinson Disease Autosomal Recessive, Early Onset | Parkinson Disease, Autosomal Recessive Early-Onset, Digenic, Pink1/Dj1United States
-
ProgenaBiomeRecruitingParkinson Disease | Parkinsons Disease With Dementia | Parkinson-Dementia Syndrome | Parkinson Disease 2 | Parkinson Disease 3 | Parkinson Disease 4United States
-
King's College LondonGlaxoSmithKlineCompletedParkinson Disease | Idiopathic Parkinson Disease | Parkinson Disease, PARK8United Kingdom
-
Ohio State UniversityCompletedParkinson's Disease | Parkinson Disease | Idiopathic Parkinson Disease | Idiopathic Parkinson's Disease | Parkinson Disease, Idiopathic | Parkinson's Disease, IdiopathicUnited States
-
National Yang Ming UniversityUnknownEarly Onset Parkinson Disease | Early Stage Parkinson Disease
-
Michele Tagliati, MDRecruitingREM Sleep Behavior Disorder | Symptomatic Parkinson Disease | Pre-motor Parkinson DiseaseUnited States
-
Cedars-Sinai Medical CenterEnrolling by invitationREM Sleep Behavior Disorder | Symptomatic Parkinson Disease | Pre-motor Parkinson DiseaseUnited States
-
Mahatma Gandhi Institute of Medical SciencesCompletedStroke, Parkinson' s Disease, Neurological Impairments, Tele-rehabilitationIndia
-
Merck Sharp & Dohme LLCCompletedParkinson Disease | Idiopathic Parkinson Disease | Idiopathic Parkinson's Disease
-
University of DeustoCompletedPARKINSON DISEASE (Disorder)Spain
Clinical Trials on Trehalose
-
National University of MalaysiaCompletedSpinocerebellar Ataxia 3Malaysia
-
Medical College of WisconsinRecruitingType 2 Diabetes | Healthy AgingUnited States
-
Consorcio Centro de Investigación Biomédica en...Stanley Medical Research InstituteCompleted
-
University of Colorado, BoulderCompletedVascular AgingUnited States
-
National University of MalaysiaRadboud University Medical CenterRecruiting
-
Mashhad University of Medical SciencesUnknownAlzheimer DiseaseIran, Islamic Republic of
-
Seelos Therapeutics, Inc.Massachusetts General Hospital; National Institute of Neurological Disorders... and other collaboratorsNo longer available
-
Bioblast Pharma Ltd.WithdrawnOculopharyngeal Muscular DystrophyCanada
-
Samsung Medical CenterCompletedNon-alcoholic Fatty Liver
-
Instituto Universitario de Oftalmobiología Aplicada...Completed