A Trial of MitoQ for the Treatment of People With Parkinson's Disease

Study Overview
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Brief Summary
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In Parkinson's Disease, the mitochondrial membranes in cells that produce dopamine become damaged by oxidants, leading to the death of these cells and progressive tremor, slowness of movement and the loss of neurons in the substantia nigra (a part of the brain that is involved in movement). Mitoquinone is targeted to reach the membrane of mitochondria and provide protection from damaging oxidants. There are no treatments currently available to slow the progression of PD and this trial will help advance the development of this unique disease modifying drug. This trial will enroll 120 participants with untreated early onset of PD. Participants will be randomized to receive 1 of 3 treatments: 40 mg of MitoQ tablets, 80 mg of MitoQ tablets or placebo. The researchers, participants and sponsor will all be blinded to the treatment allocation. Participants will be assessed after 1, 2, 3, 6, 9, 12 months of treatment and again 28 days after their last dose. The effectiveness of the trial drug will be measured via the Unified Parkinson's Disease Rating Scale (UPDRS). The safety of the trial drug will be monitored via regular participant examinations, blood tests, ECG and collecting information on adverse events.

Official Title
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A Double-Blind, Prospective, Randomized Comparison of 2 Doses of MitoQ and Placebo for the Treatment of Patients With Parkinson's Disease

Conditions
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Parkinson's Disease

Intervention / Treatment
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* Drug: MitoQ


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Informed consent 30 yrs or older Diagnosis of PD (2 or more of bradykinesia; rest tremor, rigidity) Adequate contraceptive measures (females) Exclusion Criteria: Malignancy within last 2 years Pregnancy & breast-feeding Treatment with any anti-PD drugs within 30 days of enrolment Prior treatment with anti-PD medication exceeding 42 days in total Medication-induced PD/PD not of idiopathic origin CoQ10/idebenone doses of 300mg/day or higher within 120 days, >25mg/day within 7 days of enrolment Methylphenidate HCl, neuroleptics, reserpine, amphetamines, selegeline or MAOIs within 6 months of enrolment CNS medications at unstable doses within 60 days of enrolment Dietary supplements > 5 x RDI Hypersensitivity to CoQ10, idebenone or any components of the study drug Unable to swallow Diseases with features of PD Seizure(s) within 12 months prior to enrolment UPDRS tremor score of 4 Hamilton Depression Rating Scale score > 10 History of stroke Requirement for dopaminergic drugs Modified Hoehn & Yahr score > 2.5 History of brain surgery for Parkinson's disease History of structural brain disease / congenital brain abnormality History of ECT Any other clinically significant medical or psychiatric condition or lab abnormality Enrolment in any other pharmacological study within 30 days of enrolment

Ages Eligible for Study
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Minimum Age: 30 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>40 mg MitoQ OD | Drug: MitoQ<br>* Two doses will be compared to placebo: 40 or 80 mg of MitoQ once daily for a period of 12 months.<br>|
| Experimental: 2<br>80 mg MitoQ OD | Drug: MitoQ<br>* Two doses will be compared to placebo: 40 or 80 mg of MitoQ once daily for a period of 12 months.<br>|
| Placebo Comparator: 3<br>Placebo | Drug: MitoQ<br>* Two doses will be compared to placebo: 40 or 80 mg of MitoQ once daily for a period of 12 months.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Unified Parkinson's Disease Rating Scale (UPDRS) score at the final study visit compared to baseline |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The following assessments performed at the final study visit compared to baseline |  | 12 months | 
| UPDRS sub scores |  | 12 months | 
| Mini Mental State Examination |  | 12 months | 
| Schwab and England Scale |  | 12 months | 
| Modified Hoehn and Yahr Scale |  | 12 months | 
| Timed tapping score |  | 12 months | 
| The following safety outcomes will be measured over the course of the trial |  | 12 months | 
| Adverse events |  | 12 months | 
| ECG changes |  | 12 months | 
| Laboratory sample results |  | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Parkinson's disease, MitoQ, UPDRS