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Coenzyme Q10 in Adult-Onset Ataxia (CoQATAX)

This study has been completed.
Sponsor:
Collaborator:
University of Florida
Information provided by:
The University of Texas, Galveston
ClinicalTrials.gov Identifier:
NCT00957216
First received: June 22, 2009
Last updated: April 15, 2010
Last verified: April 2010
  Purpose

This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the cerebellum, with or without additional central nervous system (CNS) manifestations, in the absence of family history of degenerative ataxias.


Condition Intervention Phase
Sporadic Ataxia
Drug: Placebo (sugar pill)
Drug: Coenzyme Q10
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Supportive Care
Official Title: Safety and Tolerability of Coenzyme Q10 in Adult-Onset Sporadic Spinocerebellar Ataxia

Resource links provided by NLM:


Further study details as provided by The University of Texas, Galveston:

Primary Outcome Measures:
  • Measure cerebellar functions by a validated scale (SARA) [ Time Frame: 6 weeks ] [ Designated as safety issue: No ]

Enrollment: 1
Study Start Date: April 2008
Study Completion Date: August 2009
Primary Completion Date: August 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: sugar pill Drug: Placebo (sugar pill)
placebo: 2 wafers 4 times a day
Active Comparator: Coenzyme Q10
The CoQ10 arm will be compared with the placebo arm to determine if high-dose CoQ10 is safe and well tolerated in subjects with sporadic adult-onset spinocerebellar ataxias
Drug: Coenzyme Q10
300mg wafers; 2 wafers 4 times a day
Other Name: CoQ10

Detailed Description:

This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the cerebellum, with or without additional central nervous system (CNS) manifestations, in the absence of family history of degenerative ataxias.

CoQ10 is an essential cofactor of the electron transport chain and is a potent free radical scavenger in lipid and mitochondrial membranes. CoQ10 has shown efficacy in treatment of Parkinson's disease patients, and a Huntington's disease trial gave a trend of slowing down disease progression (CARE-HD). A small trial of CoQ10 in patients with Friedreich's ataxia suggested potential beneficial effects on ventricular thickness. CoQ10 is being tested on ALS patients and is considered to be potentially useful for treatment of Alzheimer's disease. Thus, CoQ10 is considered to be a promising therapeutic agent that might slow down the disease progression in a wide variety of neurodegenerative disorders. To date, very high doses of CoQ10 have not been used in patients with ataxia and the safety and tolerability in this group of patients should be established before efficacy trials are launched.

Twenty patients with SAOA will be recruited for a double-blind, randomized, placebo-controlled, multicenter study. Fifteen patients will receive a total of 2400 mg of oral CoQ10 daily, and five patients will receive placebo, for a period or 4 weeks. Cerebellar functions will be measured using a validated rating scale (SARA), an oculomotor examination, and functional measurement of motor function using a 9 hole peg test and timed walk. Safety labs will be collected and a digital movie will be recorded at the beginning (prior to treatment with CoQ10) and at the end of the study.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosis of SAOA
  2. Age 18 or older
  3. Adult onset of ataxia
  4. Ambulatory capability (with or without an assisting device)
  5. Women with 2 years post menopause or surgical sterility or practicing adequate birth control
  6. Stable doses of psychotropic drugs
  7. Stable doses of drugs for movement disorders
  8. Ability to give informed consent
  9. Ability to comply with trial procedures
  10. Able to take oral medication
  11. No active and significant systemic disease (cardiac, pulmonary, hepatic, renal disease or cancer) that is not under adequate medical control
  12. Women with child-bearing potential who have a negative urine pregnancy test and practice adequate contraception during the study

Exclusion Criteria:

  1. A history or known sensitivity of intolerability to Coenzyme Q10
  2. Diagnosis of secondary (non-degenerative) ataxia
  3. Family history of degenerative ataxia
  4. Diagnosis of childhood-onset ataxia
  5. DNA diagnosis of inherited ataxia in the absence of family history
  6. Other investigational agent within 30 days of screening
  7. Ingestion of Coenzyme Q10 within 120 days of the baseline visit
  8. Diagnosis of ongoing malignancy
  9. Women who are pregnant or lactating or who have child bearing potential and not using effective birth control
  10. Uncontrolled hypertension
  11. Symptomatic orthostatic hypotension
  12. Uncontrolled diabetes mellitus
  13. Untreated thyroid disease
  14. Major psychiatric disease within 12 months of screening
  15. History of non-compliance with other therapies
  16. Drug or alcohol abuse within 12 months of screening
  17. Other condition or therapy that may prevent participation in the opinion of the investigator
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00957216

Locations
United States, Texas
The Universtity of Texas, Galveston
Galveston, Texas, United States, 77555-0539
Sponsors and Collaborators
The University of Texas, Galveston
University of Florida
Investigators
Principal Investigator: Sankarasubramoney H Subramony, MD University of Texas Medical Branch, Galveston
  More Information

No publications provided

Responsible Party: Tetsuo Ashizawa, M.D., The University of Texas Medical Branch, Galveston
ClinicalTrials.gov Identifier: NCT00957216     History of Changes
Other Study ID Numbers: 08-129, 08-SAOA-001
Study First Received: June 22, 2009
Last Updated: April 15, 2010
Health Authority: United States: Food and Drug Administration

Keywords provided by The University of Texas, Galveston:
adult onset ataxia

Additional relevant MeSH terms:
Ataxia
Dyskinesias
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Coenzyme Q10
Ubiquinone
Growth Substances
Micronutrients
Pharmacologic Actions
Physiological Effects of Drugs
Vitamins

ClinicalTrials.gov processed this record on November 23, 2014