Parkinson DiseasePhase 4NCT05355064
Efficacy of Oral Administration of Trehalose in Patients With Parkinson Disease
A Phase 4 study testing Trehalose in people with Parkinson Disease, run by Neuromed IRCCS.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- Trehalose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- May 2022to May 2023
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
Who cannot join
- 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.
About this study
Official title: 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.
- Conditions
- Parkinson Disease
- Being tested
- Trehalose
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrehaloseDrug
Also called CITOPLAS
Enrolled subjects will daily take 4 g of trehalose in oral administration for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Single arm | Experimental | Single treatment, no placebo. Receives: Drug: Trehalose |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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
Measured: 1-36 weeks
Secondary outcomes (4)
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
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.
Measured: 1-36 weeks
Assessment of General Cognitive Functioning on the Mini Mental State Examination (MMSE)
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).
Measured: 1- 36 weeks
The differences between the MoCa test score from baseline to follow up period
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.
Measured: 1-36 weeks
Changes from baseline in the Quality of Life in Neurological Disorders ( NeuroQol) and EQ-5D-5L questionnaires
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.
Measured: 1- 36 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2022
- Study startMay 2022 (estimated)
- Primary completion31 Aug 2022 (estimated)
- Study completionMay 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neuromed IRCCS
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nicola Modugno, Neuromed IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05355064
- Sponsor's protocol ID
- Trehalose treatment in PD
- Status last verified
- Apr 2022
- First submitted
- 18 Dec 2021
- Last updated
- 2 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Oral Administration of Trehalose in Patients With Parkinson Disease in Both Idiopathic and Induced by LRRK2 Mutation
- ClinicalTrials.gov ID
- NCT05355064
- Acronym / sponsor ID
- Trehalose treatment in PD
- Conditions
- Parkinson Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Neuromed IRCCSUniversity, hospital or other
- Study start
- May 2022 (estimated)
- Primary completion
- 31 Aug 2022 (estimated)
- Study completion
- May 2023 (estimated)
- First posted
- 2 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm | Experimental | Drug: TrehaloseSingle treatment, no placebo. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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;
Key exclusion criteria (7 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TrehaloseAlso: CITOPLASEnrolled subjects will daily take 4 g of trehalose in oral administration for 24 weeks. | Drug | Single arm |
Primary outcome
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. 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
4 secondary outcomes
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Time frame: 1-36 weeks
Assessment of General Cognitive Functioning on the Mini Mental State Examination (MMSE)
Time frame: 1- 36 weeks
The differences between the MoCa test score from baseline to follow up period
Time frame: 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Trehalose in people with Parkinson Disease, run by Neuromed IRCCS.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 May 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical laboratory test results at each visit from baseline to follow-up period (time frame: 1-36 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 May 2022.