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Investigating a New Treatment: Clinical Study on Coenzyme Q (Ubiquinol) for Patients with Mitochondrial Disorders and Cerebellar Ataxias
EU CTIS ID: 2023-505244-18-01
What this study is testing
To determine the safety and efficacy of Ubiquinol in patients with 1) a confirmed genetic diagnosis of mitochondrial disease or 2) cerebellar ataxia with or without molecular diagnosis.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Minimum age 1 year at the time of Screening Visit. Both pediatric and adult patients will be included, with maximum age of 90 years.
- Diagnosis of at least one of the following: i. Genetic diagnosis of mitochondrial disease, ii. Cerebellar ataxia (with or without molecular diagnosis).
- Gross Motor Function Classification System (GMFCS) level <4 at baseline
- Consent to comply with study procedures. For subjects under the age of 18 (or age of consent), parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including the need for frequent and prolonged follow-up; parent(s)/legal guardian(s) with custody of the subject must give their consent for subject to enrol in the study. Patients >12 years and < 18 years old should sign the approved assent document
- Males and females of childbearing potential must be willing to use an effective method of contraception (i.e., implants, injectable, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after last dose of study drug or early termination visit. Male subjects must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or early termination visit. Note: Double-barrier method (i.e., condom with spermicide) is required if no other methods of contraception are in use.
- Must be able to abstain from other forms of Coenzyme Q10 and / or statins for 30 days prior to the Baseline Visit and for the duration of the study.
You likely can't join if
- Non-Progressive cerebellar ataxia.
- Allergy to Coenzyme Q (Ubiquinone/Ubiquinol) or to excipients such as L-ascorbic acid (E414), gum Arabic (E414), dextrin (E1400) or oat/soy lethitin
- Pregnant or lactating subjects or those sexually active subjects who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at Screening and during the Baseline Visit.
- International normalized ratio ≥1.5×ULN at time of screening or clinically significant bleeding, as determined by the investigator.
- Comorbidities that may confound study results (e.g., fat malabsorption syndrome) in the opinion of the investigator
- Concomitant use of other form of CoQ10 and/or statins within 30 days prior to the initiation of treatment. This prohibited medication can be discontinued at the Screening Visit; if this is the case, the GMFCS assessment must be repeated to confirm inclusion eligibility after a minimum of 30 days post discontinuation, and there must be no more than a Level 4.
See the full eligibility criteria
Who can join
- Minimum age 1 year at the time of Screening Visit. Both pediatric and adult patients will be included, with maximum age of 90 years.
- Diagnosis of at least one of the following: i. Genetic diagnosis of mitochondrial disease, ii. Cerebellar ataxia (with or without molecular diagnosis).
- Gross Motor Function Classification System (GMFCS) level <4 at baseline
- Consent to comply with study procedures. For subjects under the age of 18 (or age of consent), parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including the need for frequent and prolonged follow-up; parent(s)/legal guardian(s) with custody of the subject must give their consent for subject to enrol in the study. Patients >12 years and < 18 years old should sign the approved assent document
- Males and females of childbearing potential must be willing to use an effective method of contraception (i.e., implants, injectable, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after last dose of study drug or early termination visit. Male subjects must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or early termination visit. Note: Double-barrier method (i.e., condom with spermicide) is required if no other methods of contraception are in use.
- Must be able to abstain from other forms of Coenzyme Q10 and / or statins for 30 days prior to the Baseline Visit and for the duration of the study.
What rules you out
- Non-Progressive cerebellar ataxia.
- Allergy to Coenzyme Q (Ubiquinone/Ubiquinol) or to excipients such as L-ascorbic acid (E414), gum Arabic (E414), dextrin (E1400) or oat/soy lethitin
- Pregnant or lactating subjects or those sexually active subjects who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at Screening and during the Baseline Visit.
- International normalized ratio ≥1.5×ULN at time of screening or clinically significant bleeding, as determined by the investigator.
- Comorbidities that may confound study results (e.g., fat malabsorption syndrome) in the opinion of the investigator
- Concomitant use of other form of CoQ10 and/or statins within 30 days prior to the initiation of treatment. This prohibited medication can be discontinued at the Screening Visit; if this is the case, the GMFCS assessment must be repeated to confirm inclusion eligibility after a minimum of 30 days post discontinuation, and there must be no more than a Level 4.
- Illicit drug use 30 days prior to screening and during the study is prohibited
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.