Study in PRE-manifest Huntington's Disease of Coenzyme Q10 (UbiquinonE) Leading to Preventive Trials (PREQUEL) - Full Text View

Purpose

To establish the tolerability of treatment with 600, 1200 or 2400 mg per day of coenzyme Q10 in pre-manifest participants carrying the CAGn expansion for Huntington's Disease (HD).

Condition	Intervention	Phase
Huntington's Disease	Drug: CoQ10	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment
Official Title:	A Multi-Center, Double-Blind, Randomized, Parallel Group Tolerability Study of Coenzyme Q10 (UbiquinonE)in PRE-manifest Huntington's Disease

Resource links provided by NLM:

Genetics Home Reference related topics: chorea-acanthocytosis Huntington disease McLeod neuroacanthocytosis syndrome

MedlinePlus related topics: Huntington's Disease

Drug Information available for: Ubidecarenone

U.S. FDA Resources

Further study details as provided by Huntington Study Group:

Primary Outcome Measures:

Tolerability: Ability to complete the study on the originally randomized treatment assignment. [ Time Frame: 20 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Additional Tolerability [ Time Frame: 20 weeks ] [ Designated as safety issue: Yes ]
Safety (labs and clinical) [ Time Frame: 20 weeks ] [ Designated as safety issue: Yes ]
Biomarkers (8OHdG/8OHrG and OGG1) [ Time Frame: 20 weeks ] [ Designated as safety issue: Yes ]
CoQ10 Levels [ Time Frame: 20 weeks ] [ Designated as safety issue: Yes ]
Clinical (UHDRS '99;FASRBE;IADL;BDI-II;C-SSRS) [ Time Frame: 20 weeks ] [ Designated as safety issue: Yes ]
Feasibility (Enrollment rate;completing the study;visit and study med compliance) [ Time Frame: 20 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	90
Study Start Date:	February 2010
Estimated Study Completion Date:	June 2012
Primary Completion Date:	March 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 600 mg per day of CoQ10 All participants will start on a dosage of 600 mg/day of CoQ10 in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20.	Drug: CoQ10 Capsules containing 300 mg of CoQ10 or matching placebo taken orally twice a day. All participant will start on a dosage of 600 mg/day in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20. Other Names: -Ubiquinone -coenzyme Q10
Experimental: 1200 mg per day of CoQ10 All participants will start on a dosage of 600 mg/day in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20.	Drug: CoQ10 Capsules containing 300 mg of CoQ10 or matching placebo taken orally twice a day. All participant will start on a dosage of 600 mg/day in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20. Other Names: -Ubiquinone -coenzyme Q10
Experimental: 2400 mg per day of CoQ10 All participants will start on a dosage of 600 mg/day in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20.	Drug: CoQ10 Capsules containing 300 mg of CoQ10 or matching placebo taken orally twice a day. All participant will start on a dosage of 600 mg/day in divided doses, increasing weekly by 600 mg/day to a maximum dosage of 2400 mg/day at week 4. Dosage should be stable from week 4 until week 20. Other Names: -Ubiquinone -coenzyme Q10

Detailed Description:

secondary objectives:

To assess the change from baseline to 20 weeks on biomarkers of oxidative stress (8OHdG and 8OHrG) and DNA repair mechanisms (OGG1) in pre-manifest participants treated with 600, 1200 or 2400 mg per day of CoQ10.
To assess the dose-response relationship between CoQ10 at dosages of 600, 1200 or 2400 mg per day and 8OHdG/8OHrG and OGG1.
To assess the serum levels of CoQ10 at 600, 1200 or 2400 mg in pre-manifest participants and their relationship to 8OHdG/8OHrG and OGG1.
To assess the feasibility of implementing a preventive therapeutic trial in a pre-manifest population.
To assess the utility and stability of clinical measures of HD, social relations, behavior and employment in a pre-manifest sample enrolled in a treatment trial.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Participants will be positive for the CAGn expansion in the Huntingtin gene (>36 repeats) and be pre-manifest by virtue of scoring 3 or less on diagnostic confidence level (Question 17 of the UHDRS)
Participants will have received genetic testing prior to enrollment through a standard pre-manifest testing protocol.
18 years of age or older.
Concomitant medications are permitted with the exception of CoQ10, creatine > 5g/day and warfarin.

Exclusion Criteria:

History of intolerability to CoQ10.
CoQ10 use within 60 days prior to randomization.
Unstable medical or psychiatric illness;
Substance abuse within one year of the baseline visit.
Pregnancy, breastfeeding or lack of reliable contraception in women of childbearing age.
Subjects with known allergy to FD&C #6 yellow food coloring.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00920699

Locations

United States, California
University of California Davis
Sacramento, California, United States, 95817
United States, Colorado
Colorado Neurological Institute
Englewood, Colorado, United States, 80120
United States, Florida
University of Miami School of Medicine
Miami, Florida, United States, 33136
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30329
United States, Indiana
Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
United States, Kansas
Hereditary Neurological Disease Centre (HNDC)
Wichita, Kansas, United States, 67206
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21287
United States, Minnesota
Hennepin County Medical Center
Minneapolis, Minnesota, United States, 55415
United States, Missouri
Washington University
St. Louis, Missouri, United States, 63110
United States, New York
Albany Medical College
Albany, New York, United States, 12208
University of Rochester
Rochester, New York, United States, 14618
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030

Sponsors and Collaborators

Huntington Study Group

National Institute of Neurological Disorders and Stroke (NINDS)

Investigators

Principal Investigator:	Christopher A Ross, MD, PhD	Johns Hopkins University
Principal Investigator:	Kevin M Biglan, MD, MPH	University of Rochester

More Information

Additional Information:

Huntington Study Group This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Christopher A. Ross, MD, PhD-Principal Investigator, Johns Hopkins University School of Medicine
ClinicalTrials.gov Identifier:	NCT00920699 History of Changes
Other Study ID Numbers:	PREQUEL-01.00, NIH grant: 1 R01 NS060118-01A1
Study First Received:	June 9, 2009
Last Updated:	March 28, 2012
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases

Genetic Diseases, Inborn
Cognition Disorders
Delirium, Dementia, Amnestic, Cognitive Disorders
Mental Disorders
Coenzyme Q10
Ubiquinone
Micronutrients
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions
Vitamins

ClinicalTrials.gov processed this record on May 23, 2013