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

NCT ID: NCT00920699

Last Updated: 2017-09-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 90 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-02-28
• Study Completion Date: 2012-03-31

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.

Conditions

Huntington's Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

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.

Group Type: EXPERIMENTAL

CoQ10

Intervention Type: DRUG

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.

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.

Group Type: EXPERIMENTAL

CoQ10

Intervention Type: DRUG

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.

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.

Group Type: EXPERIMENTAL

CoQ10

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

-Ubiquinone; -coenzyme Q10

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

Johns Hopkins University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Christopher A Ross, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Kevin M Biglan, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

University of Rochester

Locations

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

Emory University School of Medicine

Atlanta, Georgia, United States

Indiana University School of Medicine

Indianapolis, Indiana, United States

University of Iowa

Iowa City, Iowa, United States

Hereditary Neurological Disease Centre (HNDC)

Wichita, Kansas, United States

Johns Hopkins University

Baltimore, Maryland, United States

Hennepin County Medical Center

Minneapolis, Minnesota, United States

Washington University

St Louis, Missouri, United States

Albany Medical College

Albany, New York, United States

University of Rochester

Rochester, New York, United States

Baylor College of Medicine

Houston, Texas, United States

Countries

United States

Other Identifiers

NIH grant: 1 R01 NS060118-01A1

Identifier Type: -

Identifier Source: secondary_id

PREQUEL-01.00

Identifier Type: -

Identifier Source: org_study_id