Trial Outcomes & Findings for Study in PRE-manifest Huntington's Disease of Coenzyme Q10 (UbiquinonE) Leading to Preventive Trials (PREQUEL) (NCT NCT00920699)

NCT ID: NCT00920699

Last Updated: 2017-09-25

Results Overview

No dosage modifications, reported as a %

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

90 participants

Primary outcome timeframe

20 weeks

Results posted on

2017-09-25

Participant Flow

Participant milestones

Participant milestones
Measure
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. 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.
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. 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.
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. 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.
Overall Study
STARTED
29
30
32
Overall Study
COMPLETED
27
29
28
Overall Study
NOT COMPLETED
2
1
4

Reasons for withdrawal

Reasons for withdrawal
Measure
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. 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.
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. 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.
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. 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.
Overall Study
Withdrawal by Subject
2
0
2
Overall Study
no longer met eligibility
0
1
0
Overall Study
Lost to Follow-up
0
0
2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
600 mg Per Day of CoQ10
n=29 Participants
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. 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.
1200 mg Per Day of CoQ10
n=29 Participants
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. 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.
2400 mg Per Day of CoQ10
n=32 Participants
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. 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.
Total
n=90 Participants
Total of all reporting groups
Age, Continuous
39.2 years
STANDARD_DEVIATION 9.8 • n=5 Participants
39.3 years
STANDARD_DEVIATION 11.5 • n=7 Participants
39.8 years
STANDARD_DEVIATION 11.6 • n=5 Participants
39.5 years
STANDARD_DEVIATION 10.9 • n=4 Participants
Sex: Female, Male
Female
15 Participants
n=5 Participants
16 Participants
n=7 Participants
16 Participants
n=5 Participants
47 Participants
n=4 Participants
Sex: Female, Male
Male
14 Participants
n=5 Participants
13 Participants
n=7 Participants
16 Participants
n=5 Participants
43 Participants
n=4 Participants
Region of Enrollment
United States
29 Participants
n=5 Participants
29 Participants
n=7 Participants
32 Participants
n=5 Participants
90 Participants
n=4 Participants

PRIMARY outcome

Timeframe: 20 weeks

No dosage modifications, reported as a %

Outcome measures

Outcome measures
Measure
600 mg Per Day of CoQ10
n=29 Participants
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. 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.
1200 mg Per Day of CoQ10
n=29 Participants
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. 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.
2400 mg Per Day of CoQ10
n=32 Participants
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. 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.
Tolerability as Assessed by Ability to Complete the Study on the Originally Randomized Treatment Assignment.
90.0 percentage of participants
93.1 percentage of participants
81.3 percentage of participants

SECONDARY outcome

Timeframe: change from baseline to 20 weeks

Population: Lab data not available for all participants to compare baseline and 20 weeks

ng/ml. Negative value signifies an decrease in 8OHdG levels

Outcome measures

Outcome measures
Measure
600 mg Per Day of CoQ10
n=19 Participants
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. 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.
1200 mg Per Day of CoQ10
n=21 Participants
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. 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.
2400 mg Per Day of CoQ10
n=27 Participants
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. 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.
8OHdG Levels
0.15 ng/ml
Standard Deviation 3.26
-1.56 ng/ml
Standard Deviation 3.23
0.55 ng/ml
Standard Deviation 7.1

SECONDARY outcome

Timeframe: change from baseline to 20 weeks

Population: data was not available for all participants to compare baseline to 20 weeks

ng/ml

Outcome measures

Outcome measures
Measure
600 mg Per Day of CoQ10
n=24 Participants
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. 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.
1200 mg Per Day of CoQ10
n=25 Participants
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. 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.
2400 mg Per Day of CoQ10
n=28 Participants
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. 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.
CoQ10 Levels
1.82 ng/ml
Standard Deviation 1.78
1.92 ng/ml
Standard Deviation 1.66
2.33 ng/ml
Standard Deviation 1.38

Adverse Events

600 mg Per Day of CoQ10

Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths

1200 mg Per Day of CoQ10

Serious events: 0 serious events
Other events: 20 other events
Deaths: 0 deaths

2400 mg Per Day of CoQ10

Serious events: 0 serious events
Other events: 16 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
600 mg Per Day of CoQ10
n=29 participants at risk
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. 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.
1200 mg Per Day of CoQ10
n=29 participants at risk
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. 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.
2400 mg Per Day of CoQ10
n=32 participants at risk
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. 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.
Gastrointestinal disorders
Diarrhea
6.9%
2/29 • Number of events 2 • up to 20 weeks
24.1%
7/29 • Number of events 7 • up to 20 weeks
12.5%
4/32 • Number of events 4 • up to 20 weeks
Gastrointestinal disorders
Abdominal discomfort/pain; dyspepsia
6.9%
2/29 • Number of events 2 • up to 20 weeks
13.8%
4/29 • Number of events 4 • up to 20 weeks
3.1%
1/32 • Number of events 1 • up to 20 weeks
Nervous system disorders
Dizziness
3.4%
1/29 • Number of events 1 • up to 20 weeks
6.9%
2/29 • Number of events 2 • up to 20 weeks
3.1%
1/32 • Number of events 1 • up to 20 weeks
Gastrointestinal disorders
Abdominal distension
3.4%
1/29 • Number of events 1 • up to 20 weeks
3.4%
1/29 • Number of events 1 • up to 20 weeks
3.1%
1/32 • Number of events 1 • up to 20 weeks
Musculoskeletal and connective tissue disorders
increase creatinine phosphokinase (blood)
6.9%
2/29 • Number of events 2 • up to 20 weeks
0.00%
0/29 • up to 20 weeks
3.1%
1/32 • Number of events 1 • up to 20 weeks
Psychiatric disorders
Depression
0.00%
0/29 • up to 20 weeks
6.9%
2/29 • Number of events 2 • up to 20 weeks
3.1%
1/32 • Number of events 1 • up to 20 weeks
Gastrointestinal disorders
Flatulence
0.00%
0/29 • up to 20 weeks
6.9%
2/29 • Number of events 2 • up to 20 weeks
3.1%
1/32 • Number of events 1 • up to 20 weeks
Renal and urinary disorders
Hematuria
0.00%
0/29 • up to 20 weeks
0.00%
0/29 • up to 20 weeks
9.4%
3/32 • Number of events 3 • up to 20 weeks
Nervous system disorders
Headache
0.00%
0/29 • up to 20 weeks
3.4%
1/29 • Number of events 1 • up to 20 weeks
6.2%
2/32 • Number of events 2 • up to 20 weeks
Gastrointestinal disorders
Vomiting
3.4%
1/29 • Number of events 1 • up to 20 weeks
3.4%
1/29 • Number of events 1 • up to 20 weeks
3.1%
1/32 • Number of events 1 • up to 20 weeks

Additional Information

Christopher A. Ross M.D. Ph.D.

Johns Hopkins University

Phone: 410-614-0011

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place