Huntington's DiseasePhase 2PREQUELNCT00920699
Study in PRE-manifest Huntington's Disease of Coenzyme Q10 (UbiquinonE) Leading to Preventive Trials (PREQUEL)
A Phase 2 study testing CoQ10 in people with Huntington's Disease, run by Johns Hopkins University at 13 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Huntington's Disease
- What is being tested?
- CoQ10
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 13 sitesUnited States
- Study dates
- Feb 2010to Mar 2012
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
Albany Medical College
Albany, New York, 12208
Emory University School of Medicine
Atlanta, Georgia, 30329
Johns Hopkins University
Baltimore, Maryland, 21287
Colorado Neurological Institute
Englewood, Colorado, 80120
Baylor College of Medicine
Houston, Texas, 77030
Indiana University School of Medicine
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
University of Miami School of Medicine
Miami, Florida, 33136
Hennepin County Medical Center
Minneapolis, Minnesota, 55415
University of Rochester
Rochester, New York, 14618
University of California Davis
Sacramento, California, 95817
Washington University
St Louis, Missouri, 63110
Hereditary Neurological Disease Centre (HNDC)
Wichita, Kansas, 67206
About this study
Official title: A Multi-Center, Double-Blind, Randomized, Parallel Group Tolerability Study of Coenzyme Q10 (UbiquinonE)in PRE-manifest Huntington's Disease
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).
Read the detailed description
secondary objectives:
- 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.
- To assess the dose-response relationship between CoQ10 at dosages of 600, 1200 or 2400 mg per day and 8OHdG/8OHrG and OGG1.
- 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.
- To assess the feasibility of implementing a preventive therapeutic trial in a pre-manifest population.
- 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
- Being tested
- CoQ10
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CoQ10Drug
Also called -Ubiquinone, -coenzyme Q10
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Drug: CoQ10 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment.
No dosage modifications, reported as a %
Measured: 20 weeks
Secondary outcomes (2)
8OHdG Levels
ng/ml. Negative value signifies an decrease in 8OHdG levels
Measured: change from baseline to 20 weeks
CoQ10 Levels
ng/ml
Measured: change from baseline to 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2009 (estimated)
- Study startFeb 2010
- Primary completionMar 2012
- Study completionMar 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Huntington's DiseaseStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christopher A Ross, MD, PhD, Principal investigator, Johns Hopkins UniversityKevin M Biglan, MD, MPH, Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00920699
- Sponsor's protocol ID
- PREQUEL-01.00
- Other IDs
- NIH grant: 1 R01 NS060118-01A1
- Acronym
- PREQUEL
- Status last verified
- Aug 2017
- First submitted
- 9 Jun 2009
- Last updated
- 25 Sep 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Double-Blind, Randomized, Parallel Group Tolerability Study of Coenzyme Q10 (UbiquinonE)in PRE-manifest Huntington's Disease
- ClinicalTrials.gov ID
- NCT00920699
- Acronym / sponsor ID
- PREQUEL / PREQUEL-01.00
- Conditions
- Huntington's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Feb 2010
- Primary completion
- Mar 2012 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 15 Jun 2009 (estimated)
- Results
- Posted 25 Sep 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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.
- To assess the dose-response relationship between CoQ10 at dosages of 600, 1200 or 2400 mg per day and 8OHdG/8OHrG and OGG1.
- 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.
- To assess the feasibility of implementing a preventive therapeutic trial in a pre-manifest population.
- 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 600 mg per day of CoQ10 | Experimental | Drug: CoQ10All 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. |
| 1200 mg per day of CoQ10 | Experimental | Drug: CoQ10All 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. |
| 2400 mg per day of CoQ10 | Experimental | Drug: CoQ10All 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CoQ10Also: -Ubiquinone, -coenzyme Q10Capsules 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. | Drug | 1200 mg per day of CoQ10, 2400 mg per day of CoQ10, 600 mg per day of CoQ10 |
Primary outcome
Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment.
Time frame: 20 weeks
No dosage modifications, reported as a %
2 secondary outcomes
8OHdG Levels
Time frame: change from baseline to 20 weeks
CoQ10 Levels
Time frame: change from baseline to 20 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Huntington Study GroupLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christopher A Ross, MD, PhD principal investigator
Johns Hopkins University
Kevin M Biglan, MD, MPH principal investigator
University of Rochester
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CoQ10 in people with Huntington's Disease, run by Johns Hopkins University at 13 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Sep 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment. (time frame: 20 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2017.