Clinical trial NCT00920699: Study in PRE-manifest Huntington's Disease of Coenzyme Q10 (UbiquinonE) Leading to Preventive Trials (PREQUEL)
Official title: A Multi-Center, Double-Blind, Randomized, Parallel Group Tolerability Study of Coenzyme Q10 (UbiquinonE)in PRE-manifest Huntington's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Huntington's Disease
Lead sponsor: Johns Hopkins University
Sponsor class: OTHER
Collaborators: name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2009-06-15
Enrollment: 90
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).

Detailed description: 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.

Eligibility 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.

Primary outcomes:
measure: Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment.
timeFrame: 20 weeks
Secondary outcomes:
measure: 8OHdG Levels
timeFrame: change from baseline to 20 weeks
measure: CoQ10 Levels
timeFrame: change from baseline to 20 weeks
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: University of California Davis
state: California
city: Englewood
country: United States
facility: Colorado Neurological Institute
state: Colorado
city: Miami
country: United States
facility: University of Miami School of Medicine
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Wichita
country: United States
facility: Hereditary Neurological Disease Centre (HNDC)
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Minneapolis
country: United States
facility: Hennepin County Medical Center
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
Study officials:
affiliation: Johns Hopkins University
name: Christopher A Ross, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Rochester
name: Kevin M Biglan, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: