Coenzyme Q10 in Huntington's Disease (HD)
This study is currently recruiting participants.
Verified by National Institute of Neurological Disorders and Stroke (NINDS), January 2009
First Received: February 4, 2008 Last Updated: January 14, 2009
This study is currently recruiting participants.
Verified by National Institute of Neurological Disorders and Stroke (NINDS), January 2009
First Received: February 4, 2008 Last Updated: January 14, 2009
University of Rochester
Information provided by:
National Institute of Neurological Disorders and Stroke (NINDS)
ClinicalTrials.gov Identifier:
NCT00608881
Purpose
The goals of this trial are to determine if coenzyme Q10 is effective in slowing the worsening symptoms of Huntington's disease and to learn about the safety and acceptability of long-term coenzyme Q10 use by determining its effects on people with Huntington's disease.
Condition
Intervention
Phase
Huntington's Disease
Drug: coenzyme Q10Other: placebo
Phase III
Study Type:
Interventional
Study Design:
Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Parallel Assignment, Efficacy Study
Official Title:
Coenzyme Q10 in Huntington's Disease (HD)
Further study details as provided by National Institute of Neurological Disorders and Stroke (NINDS):
Primary Outcome Measures:
Change in total functional capacity [ Time Frame: over 5 years ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
Change in other UHDRS scores; Tolerability - proportion of subjects completing the study at the assigned dosage level; Safety - frequency of adverse events; Times to decline in TFC by 2 and 3 points [ Time Frame: duration of the trial ] [ Designated as safety issue: Yes ]
Estimated Enrollment:
608
Study Start Date:
March 2008
Estimated Study Completion Date:
April 2014
Estimated Primary Completion Date:
April 2014 (Final data collection date for primary outcome measure)
Arms
Assigned Interventions
A: Active Comparator
Randomized to active treatment (coenzyme Q10 2400 mg/day)
Drug: coenzyme Q10
4 - 300 mg CoQ chewable wafers taken orally twice a day
B: Placebo Comparator
Randomized to placebo
Other: placebo
an inactive substance Detailed Description:
Huntington's disease (HD) is a slowly progressive disorder that devastates the lives of those affected and their families. There are no treatments that slow the progression of HD, only mildly effective symptomatic therapies are available.
The purpose of this trial is to find out if coenzyme Q10 (CoQ) is effective in slowing the worsening symptoms of HD. In this study, researchers also will learn about the safety and acceptability of long-term CoQ use by determining its effects on people with HD.
Participants in this trial will be randomly chosen to one of two groups. Group 1 will receive CoQ (2400 mg/day), and group 2 will receive a placebo (an inactive substance). Researchers will compare the change in total functional capacity (TFC)—a measure of functional disability—in the two groups. The TFC is a valid and reliable measure of disease progression and is particularly responsive to change in the early and mid-stages of HD. Researchers will also compare the changes in other components of the Unified Huntington's Disease Rating Scale '99 (UHDRS) including: the total motor score, total behavioral frequency score, total behavior frequency X severity score, verbal fluency test, symbol digit modalities test, Stroop, interference test, functional checklist, and independence scale scores. The groups will also be compared with respect to tolerability, adverse events, vital signs, and laboratory test results as measures of safety.
Eligibility
Ages Eligible for Study:
16 Years and older
Genders Eligible for Study:
Both
Accepts Healthy Volunteers:
No
Criteria
Inclusion Criteria:
To be eligible for enrollment into this study, subjects must meet the following eligibility criteria within 28 days prior to randomization:
Subjects must have clinical features of HD and a confirmed family history of HD, OR a CAG repeat expansion ≥ 36.
TFC > 9.
Must be ambulatory and not require skilled nursing care.
Age ≥ 16 years.
Women must not be able to become pregnant (e.g., post menopausal, surgically sterile or using adequate birth control methods for the duration of the study).
If psychotropic medications are taken (e.g., anxiolytics, hypnotics, benzodiazepines, antidepressants), they must be at a stable dosage for four weeks prior to randomization and should be maintained at a constant dosage throughout the study, as possible. Any changes to these medications mandated by clinical conditions will be systematically recorded and the subject will be permitted to remain in the trial.
Able to give informed consent and comply with trial procedures
Able to take oral medication.
Able to identify an informant or caregiver who will be willing and able to supervise the daily dosing of study medications and to maintain control of study medications in the home.
A designated individual will be identified by the subject to participate in the ongoing consent process should the subject's cognitive capacity to consent become compromised during participation in the study.
Exclusion Criteria:
History or known sensitivity of intolerability to CoQ.
Exposure to any investigational drug within 30 days of the Baseline visit.
Clinical evidence of unstable medical illness in the investigator's judgment.
Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression or suicidal ideation within 90 days of the Baseline visit.
Substance (alcohol or drug) abuse within one year of the Baseline visit.
Women who are pregnant or breastfeeding.
Use of supplemental coenzyme Q10 within 120 days prior to the Baseline visit
Clinically serious abnormalities in the screening laboratory studies (Screening creatinine greater than 2.0, alanine aminotransferase (ALT) or total bilirubin greater than 3 times the upper limit of normal, absolute neutrophil count of ≤1000/ul, platelet concentration of <100,000/ul,> 1.5 time upper limit of normal).
Known allergy to FD&C yellow #5 or any other ingredient in the study drug (active and placebo)
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00608881
Contacts
Contact: Huntington Study Group
1-800-487-7671
Show 43 Study Locations
Sponsors and Collaborators
Massachusetts General Hospital
National Institute of Neurological Disorders and Stroke (NINDS)
University of Rochester
Investigators
Principal Investigator:
Merit Cudkowicz, MD MSc
Massachusetts General Hospital
Principal Investigator:
Michael McDermott, PhD
University of Rochester, Biostatistics
Principal Investigator:
Karl Kieburtz, MD MPH
Director, Clinical Trials Coordination Center, University of Rochester
Information provided by:
National Institute of Neurological Disorders and Stroke (NINDS)
ClinicalTrials.gov Identifier:
NCT00608881
Purpose
The goals of this trial are to determine if coenzyme Q10 is effective in slowing the worsening symptoms of Huntington's disease and to learn about the safety and acceptability of long-term coenzyme Q10 use by determining its effects on people with Huntington's disease.
Condition
Intervention
Phase
Huntington's Disease
Drug: coenzyme Q10Other: placebo
Phase III
Study Type:
Interventional
Study Design:
Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Parallel Assignment, Efficacy Study
Official Title:
Coenzyme Q10 in Huntington's Disease (HD)
Further study details as provided by National Institute of Neurological Disorders and Stroke (NINDS):
Primary Outcome Measures:
Change in total functional capacity [ Time Frame: over 5 years ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
Change in other UHDRS scores; Tolerability - proportion of subjects completing the study at the assigned dosage level; Safety - frequency of adverse events; Times to decline in TFC by 2 and 3 points [ Time Frame: duration of the trial ] [ Designated as safety issue: Yes ]
Estimated Enrollment:
608
Study Start Date:
March 2008
Estimated Study Completion Date:
April 2014
Estimated Primary Completion Date:
April 2014 (Final data collection date for primary outcome measure)
Arms
Assigned Interventions
A: Active Comparator
Randomized to active treatment (coenzyme Q10 2400 mg/day)
Drug: coenzyme Q10
4 - 300 mg CoQ chewable wafers taken orally twice a day
B: Placebo Comparator
Randomized to placebo
Other: placebo
an inactive substance Detailed Description:
Huntington's disease (HD) is a slowly progressive disorder that devastates the lives of those affected and their families. There are no treatments that slow the progression of HD, only mildly effective symptomatic therapies are available.
The purpose of this trial is to find out if coenzyme Q10 (CoQ) is effective in slowing the worsening symptoms of HD. In this study, researchers also will learn about the safety and acceptability of long-term CoQ use by determining its effects on people with HD.
Participants in this trial will be randomly chosen to one of two groups. Group 1 will receive CoQ (2400 mg/day), and group 2 will receive a placebo (an inactive substance). Researchers will compare the change in total functional capacity (TFC)—a measure of functional disability—in the two groups. The TFC is a valid and reliable measure of disease progression and is particularly responsive to change in the early and mid-stages of HD. Researchers will also compare the changes in other components of the Unified Huntington's Disease Rating Scale '99 (UHDRS) including: the total motor score, total behavioral frequency score, total behavior frequency X severity score, verbal fluency test, symbol digit modalities test, Stroop, interference test, functional checklist, and independence scale scores. The groups will also be compared with respect to tolerability, adverse events, vital signs, and laboratory test results as measures of safety.
Eligibility
Ages Eligible for Study:
16 Years and older
Genders Eligible for Study:
Both
Accepts Healthy Volunteers:
No
Criteria
Inclusion Criteria:
To be eligible for enrollment into this study, subjects must meet the following eligibility criteria within 28 days prior to randomization:
Subjects must have clinical features of HD and a confirmed family history of HD, OR a CAG repeat expansion ≥ 36.
TFC > 9.
Must be ambulatory and not require skilled nursing care.
Age ≥ 16 years.
Women must not be able to become pregnant (e.g., post menopausal, surgically sterile or using adequate birth control methods for the duration of the study).
If psychotropic medications are taken (e.g., anxiolytics, hypnotics, benzodiazepines, antidepressants), they must be at a stable dosage for four weeks prior to randomization and should be maintained at a constant dosage throughout the study, as possible. Any changes to these medications mandated by clinical conditions will be systematically recorded and the subject will be permitted to remain in the trial.
Able to give informed consent and comply with trial procedures
Able to take oral medication.
Able to identify an informant or caregiver who will be willing and able to supervise the daily dosing of study medications and to maintain control of study medications in the home.
A designated individual will be identified by the subject to participate in the ongoing consent process should the subject's cognitive capacity to consent become compromised during participation in the study.
Exclusion Criteria:
History or known sensitivity of intolerability to CoQ.
Exposure to any investigational drug within 30 days of the Baseline visit.
Clinical evidence of unstable medical illness in the investigator's judgment.
Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression or suicidal ideation within 90 days of the Baseline visit.
Substance (alcohol or drug) abuse within one year of the Baseline visit.
Women who are pregnant or breastfeeding.
Use of supplemental coenzyme Q10 within 120 days prior to the Baseline visit
Clinically serious abnormalities in the screening laboratory studies (Screening creatinine greater than 2.0, alanine aminotransferase (ALT) or total bilirubin greater than 3 times the upper limit of normal, absolute neutrophil count of ≤1000/ul, platelet concentration of <100,000/ul,> 1.5 time upper limit of normal).
Known allergy to FD&C yellow #5 or any other ingredient in the study drug (active and placebo)
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00608881
Contacts
Contact: Huntington Study Group
1-800-487-7671
Show 43 Study Locations
Sponsors and Collaborators
Massachusetts General Hospital
National Institute of Neurological Disorders and Stroke (NINDS)
University of Rochester
Investigators
Principal Investigator:
Merit Cudkowicz, MD MSc
Massachusetts General Hospital
Principal Investigator:
Michael McDermott, PhD
University of Rochester, Biostatistics
Principal Investigator:
Karl Kieburtz, MD MPH
Director, Clinical Trials Coordination Center, University of Rochester
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