Ignite Creation Date:
2024-05-05 @ 5:19 PM
Last Modification Date:
2024-10-26 @ 9:30 AM
Study NCT ID:
NCT00432744
Status:
COMPLETED
Last Update Posted:
2017-09-11
First Post:
2007-02-06
Brief Title:
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
Sponsor:
University of Florida
Organization:
University of Florida
Study Overview
Official Title:
Phase 3 Trial of Coenzyme Q10 in Mitochondrial Disease
Status:
COMPLETED
Status Verified Date:
2017-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
To show that oral CoQ10 is a safe and effective treatment for children with inborn errors of mitochondrial energy metabolism due to defects in specific respiratory chain RC complexes or mitochondrial DNA mtDNA mutations and that this beneficial action is reflected in improved motor and neurobehavioral function
Detailed Description:
In many patients mitochondrial disease leads to progressive nerve and muscle damage that may be associated with problems with thinking talking remembering walking or balancing Sometimes it may also cause abnormal build up in the blood and spinal fluid of a substance called lactic acid This problem makes the blood and spinal fluid too acid which can be life-threatening There is no known specific or effective treatment for mitochondrial diseases Sometimes certain diets can improve the condition but seldom prevent the nerve or muscle damage or reduce the chance of developing life-threatening acidity of the blood
CoQ10 is not approved by the Food and Drug Administration FDA for the treatment of mitochondrial diseases It is an investigational drug that we believe may help people with certain mitochondrial diseases like the one you have both in terms of reducing the acidity of your blood and preventing or decreasing nerve and muscle damage Our belief is based on previous studies that have been conducted in children with mitochondrial diseases of various types Therefore a 12 month study has been designed to determine the safety and benefit of taking CoQ10 every day This will be done by comparing the subjects response to taking CoQ10 for 6 months to taking a placebo for 6 months A placebo looks smells and tastes like the drug being tested in this case CoQ10 but has no effect Placebo studies such as this one are very common in evaluating investigational drugs for any disease and are usually required by the FDA before a drug can be approved
CoQ10 or placebo will be given as a liquid once per day in the evening by mouth or feeding tube The CoQ10 dose will be 10 mgkg with a maximum dose of 400 mg a day Neither the subject nor the health care givers will know exactly when subjects are receiving CoQ10 or when subjects are receiving the placebo However subjects will receive CoQ10 for at least 6 months At each visit the subject will be given a seven month supply of CoQ10 nutritional cocktail and multivitamins to take home and they will be asked to bring back any unused medications at the next visit At each visit subjects will be given a medication diary to record the time and date that they take the medications that will be provided This diary should be returned to the study coordinator at the subjects scheduled visit During the 12 month period that subjects are on the study they will be expected to stop taking all medications and other supplements except for those provided by the study and those that the study doctor determines are medically needed Except in the case of an emergency the subject must contact the study doctor before taking any new medications or supplements In the case of an emergency subjects are required to inform the study doctor of the emergency and treatments as soon as possible
Subjects will be hospitalized on the General Clinical Research Center GCRC ward of Shands Hospital for 2-4 days every three to six months A parent or legal guardian will be expected to stay with the subject During that hospitalization physical and intellectual development will be measured by standard tests The GCRC dietician will ask questions about current diet at each visit and record answers A general medical history and physical examination including gross motor function andor strength tests will be performed during each visit as well as a six minute walking test While enrolled in this study a special nutritional cocktail and a Centrum-like multivitamin supplement will be provided for subjects to take daily The nutritional cocktail has vitamin C up to 10 mgkgday max 400 mgday riboflavin up to 25 mgkgday max 100 mgday thiamine up to 25 mgkgday 100 mgday carnitine up to 10 mgkgday max 400 mgday The nutritive cocktail is in capsule form and the number of capsules that the subject will take will be based on body weight for every 4 kg of body weight subjects will receive 1 capsule daily Each capsule contains Vitamin C 40 mg Riboflavin 10 mg thiamine 10 mg and carnitine 40 mg The maximum amount of capsules that will be given daily to anyone in this study is 10 capsules daily A parent or legal guardian will be asked to complete each of eight questionnaires regarding behavioral and developmental quality of life 5 and sleep The behavioral and developmental four of the quality of life QOL questionnaires and the sleep questionnaire should be completed at the 0 6 and 12 month visits One of the QOL questionnaires 46 questions will need to be completed monthly and mailed back to the study center after completion self-addressed stamped envelopes will be provided by the study and given to you by the coordinator We expect that it will take up to 20 minutes to complete the monthly QOL questionnaire and up to 3 hours at the 0 6 and 12 month visit to complete the rest of the questionnaires
About 15-20 ml of blood 3-4 teaspoons will be obtained during each hospitalization A urine sample will also be collected during each hospital visit In females of child-bearing age urine will also be collected and tested for the presence of HCG a hormone that determines pregnancy Within 24 hours of blood and urine collection test results will be assessed by the study physician
CoQ10 is not approved by the Food and Drug Administration FDA for the treatment of mitochondrial diseases It is an investigational drug that we believe may help people with certain mitochondrial diseases like the one you have both in terms of reducing the acidity of your blood and preventing or decreasing nerve and muscle damage Our belief is based on previous studies that have been conducted in children with mitochondrial diseases of various types Therefore a 12 month study has been designed to determine the safety and benefit of taking CoQ10 every day This will be done by comparing the subjects response to taking CoQ10 for 6 months to taking a placebo for 6 months A placebo looks smells and tastes like the drug being tested in this case CoQ10 but has no effect Placebo studies such as this one are very common in evaluating investigational drugs for any disease and are usually required by the FDA before a drug can be approved
CoQ10 or placebo will be given as a liquid once per day in the evening by mouth or feeding tube The CoQ10 dose will be 10 mgkg with a maximum dose of 400 mg a day Neither the subject nor the health care givers will know exactly when subjects are receiving CoQ10 or when subjects are receiving the placebo However subjects will receive CoQ10 for at least 6 months At each visit the subject will be given a seven month supply of CoQ10 nutritional cocktail and multivitamins to take home and they will be asked to bring back any unused medications at the next visit At each visit subjects will be given a medication diary to record the time and date that they take the medications that will be provided This diary should be returned to the study coordinator at the subjects scheduled visit During the 12 month period that subjects are on the study they will be expected to stop taking all medications and other supplements except for those provided by the study and those that the study doctor determines are medically needed Except in the case of an emergency the subject must contact the study doctor before taking any new medications or supplements In the case of an emergency subjects are required to inform the study doctor of the emergency and treatments as soon as possible
Subjects will be hospitalized on the General Clinical Research Center GCRC ward of Shands Hospital for 2-4 days every three to six months A parent or legal guardian will be expected to stay with the subject During that hospitalization physical and intellectual development will be measured by standard tests The GCRC dietician will ask questions about current diet at each visit and record answers A general medical history and physical examination including gross motor function andor strength tests will be performed during each visit as well as a six minute walking test While enrolled in this study a special nutritional cocktail and a Centrum-like multivitamin supplement will be provided for subjects to take daily The nutritional cocktail has vitamin C up to 10 mgkgday max 400 mgday riboflavin up to 25 mgkgday max 100 mgday thiamine up to 25 mgkgday 100 mgday carnitine up to 10 mgkgday max 400 mgday The nutritive cocktail is in capsule form and the number of capsules that the subject will take will be based on body weight for every 4 kg of body weight subjects will receive 1 capsule daily Each capsule contains Vitamin C 40 mg Riboflavin 10 mg thiamine 10 mg and carnitine 40 mg The maximum amount of capsules that will be given daily to anyone in this study is 10 capsules daily A parent or legal guardian will be asked to complete each of eight questionnaires regarding behavioral and developmental quality of life 5 and sleep The behavioral and developmental four of the quality of life QOL questionnaires and the sleep questionnaire should be completed at the 0 6 and 12 month visits One of the QOL questionnaires 46 questions will need to be completed monthly and mailed back to the study center after completion self-addressed stamped envelopes will be provided by the study and given to you by the coordinator We expect that it will take up to 20 minutes to complete the monthly QOL questionnaire and up to 3 hours at the 0 6 and 12 month visit to complete the rest of the questionnaires
About 15-20 ml of blood 3-4 teaspoons will be obtained during each hospitalization A urine sample will also be collected during each hospital visit In females of child-bearing age urine will also be collected and tested for the presence of HCG a hormone that determines pregnancy Within 24 hours of blood and urine collection test results will be assessed by the study physician
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| R01FD003032-01A1 | FDA | None | httpsreporternihgovquickSearchR01FD003032-01A1 |