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

NCT00920699 | Completed Phase 2 Results DMC

• 2 other identifiers: PREQUEL-01.00NIH grant: 1 R01 NS060118-01A1
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 13

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).

Conditions

Huntington's Disease

Outcome Measures

Primary Outcomes (1)

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

  No dosage modifications, reported as a %

  20 weeks

Secondary Outcomes (2)

• 8OHdG Levels

  change from baseline to 20 weeks

• CoQ10 Levels

  change from baseline to 20 weeks

Study Arms (3)

600 mg per day of CoQ10

EXPERIMENTAL

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

1200 mg per day of CoQ10

EXPERIMENTAL

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

2400 mg per day of CoQ10

EXPERIMENTAL

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

Interventions

CoQ10 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.

Also known as: -Ubiquinone, -coenzyme Q10

1200 mg per day of CoQ10; 2400 mg per day of CoQ10; 600 mg per day of CoQ10

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• years of age or older.
• Concomitant medications are permitted with the exception of CoQ10, creatine \> 5g/day and warfarin.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Johns Hopkins University: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (13)

University of California Davis

Sacramento, California, 95817, United States

Location

Colorado Neurological Institute

Englewood, Colorado, 80120, United States

Location

University of Miami School of Medicine

Miami, Florida, 33136, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30329, United States

Location

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Hereditary Neurological Disease Centre (HNDC)

Wichita, Kansas, 67206, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Hennepin County Medical Center

Minneapolis, Minnesota, 55415, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Albany Medical College

Albany, New York, 12208, United States

Location

University of Rochester

Rochester, New York, 14618, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Links

• Huntington Study Group

MeSH Terms

Conditions

Huntington Disease

Interventions

coenzyme Q10; Ubiquinone

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Benzoquinones; Quinones; Organic Chemicals; Coenzymes; Enzymes and Coenzymes

Results Point of Contact

• Title: Christopher A. Ross M.D. Ph.D.
• Organization: Johns Hopkins University

caross@jhmi.edu

410-614-0011

Study Officials

• Christopher A Ross, MD, PhD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

• Kevin M Biglan, MD, MPH

  University of Rochester

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2009
• First Posted: June 15, 2009
• Study Start: February 1, 2010
• Primary Completion: March 1, 2012
• Study Completion: March 1, 2012
• Last Updated: September 25, 2017
• Results First Posted: September 25, 2017

Record last verified: 2017-08

Locations

US (13)