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

Phase 2CompletedNCT00920699

Johns Hopkins University90 enrolled

Overview

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).

secondary objectives: 1. 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. 2. To assess the dose-response relationship between CoQ10 at dosages of 600, 1200 or 2400 mg per day and 8OHdG/8OHrG and OGG1. 3. 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. 4. To assess the feasibility of implementing a preventive therapeutic trial in a pre-manifest population. 5. 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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Huntington's Disease

Interventions

CoQ10DRUG

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.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (13)

University of California Davis

Sacramento, California, United States

Colorado Neurological Institute

Englewood, Colorado, United States

University of Miami School of Medicine

Miami, Florida, United States

Outcomes

Primary Outcomes

Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment.

No dosage modifications, reported as a %

Time frame: 20 weeks

Secondary Outcomes

8OHdG Levels

ng/ml. Negative value signifies an decrease in 8OHdG levels

Time frame: change from baseline to 20 weeks

CoQ10 Levels

ng/ml

Time frame: change from baseline to 20 weeks

Data from ClinicalTrials.gov

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