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

Johns Hopkins University

Status and phase: Completed; Phase 2

Conditions: Huntington's Disease

Treatments: Drug: CoQ10

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT00920699

PREQUEL-01.00

NIH grant: 1 R01 NS060118-01A1

Details and patient eligibility

About

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).

Full 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.

Enrollment: 90 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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90 participants in 3 patient groups

600 mg per day of CoQ10

Experimental group

Description:

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.

Treatment:

Drug: CoQ10

1200 mg per day of CoQ10

Experimental group

Description:

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.

Treatment:

Drug: CoQ10

2400 mg per day of CoQ10

Experimental group

Description:

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.

Treatment:

Drug: CoQ10