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

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

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.

Study Overview

• Status: Completed
• Conditions: Parkinson's Disease
• Intervention / Treatment: Drug: MitoQ

• Study Type: Interventional
• Enrollment (Actual): 128
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia
      • Westmead Hospital
  • Queensland
    • Brisbane, Queensland, Australia
      • The Royal Brisbane and Women's Hospital
  • Victoria
    • Melbourne, Victoria, Australia
      • Austin Hospital
• New Zealand
  • Auckland, New Zealand
    • Auckland City Hospital
  • Christchurch, New Zealand
    • Van der Veer Institute for Parkinson's and Brain Research
  • Hamilton, New Zealand
    • Waikato Hospital
  • Hastings, New Zealand
    • Hawke's Bay Hospital
  • Nelson, New Zealand
    • Nelson Hospital
  • Palmerston North, New Zealand
    • Palmerston North Hospital
  • Tauranga, New Zealand
    • Tauranga Hospital
  • Wellington, New Zealand
    • Wellington Hospital
  • Whangarei, New Zealand
    • Whangarei Hospital
  • Otago
    • Dunedin, Otago, New Zealand
      • Dunedin Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 30 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Informed consent
2. 30 yrs or older
3. Diagnosis of PD (2 or more of bradykinesia; rest tremor, rigidity)
4. Adequate contraceptive measures (females)

Exclusion Criteria:

1. Malignancy within last 2 years
2. Pregnancy & breast-feeding
3. Treatment with any anti-PD drugs within 30 days of enrolment
4. Prior treatment with anti-PD medication exceeding 42 days in total
5. Medication-induced PD/PD not of idiopathic origin
6. CoQ10/idebenone doses of 300mg/day or higher within 120 days, >25mg/day within 7 days of enrolment
7. Methylphenidate HCl, neuroleptics, reserpine, amphetamines, selegeline or MAOIs within 6 months of enrolment
8. CNS medications at unstable doses within 60 days of enrolment
9. Dietary supplements > 5 x RDI
10. Hypersensitivity to CoQ10, idebenone or any components of the study drug
11. Unable to swallow
12. Diseases with features of PD
13. Seizure(s) within 12 months prior to enrolment
14. UPDRS tremor score of 4
15. Hamilton Depression Rating Scale score > 10
16. History of stroke
17. Requirement for dopaminergic drugs
18. Modified Hoehn & Yahr score > 2.5
19. History of brain surgery for Parkinson's disease
20. History of structural brain disease / congenital brain abnormality
21. History of ECT
22. Any other clinically significant medical or psychiatric condition or lab abnormality
23. Enrolment in any other pharmacological study within 30 days of enrolment

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	Time Frame
Adverse events	12 months
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
ECG changes	12 months
Laboratory sample results	12 months

Collaborators and Investigators

• Sponsor: Antipodean Pharmaceuticals, Inc.
• Investigators: Principal Investigator: Barry J Snow, MD, Auckland District Health Board, New Zealand

Study record dates

Study Major Dates

• Study Start: May 1, 2006
• Study Completion (Actual): November 1, 2007

Study Registration Dates

• First Submitted: May 22, 2006
• First Submitted That Met QC Criteria: May 22, 2006
• First Posted (Estimate): May 24, 2006

Study Record Updates

• Last Update Posted (Estimate): July 21, 2010
• Last Update Submitted That Met QC Criteria: July 19, 2010
• Last Verified: July 1, 2010

More Information

Terms related to this study

• Keywords: Parkinson's disease; MitoQ; UPDRS
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: MTQ-PD-001