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Dietary Supplement

CoQ10 for Prader-Willi Syndrome

Phase 2
Waitlist Available
Led By Ingrid Tein, MD
Research Sponsored by The Hospital for Sick Children
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Weight > 35.0 kg
Patients with genetically confirmed PWS aged 13 to 18 years (n=14)
Must not have
Presence of cardiac disease with cardiac insufficiency/CHF
Presence of diabetes
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)

Summary

This trial is testing CoQ10 in patients with Prader-Willi Syndrome to see if it can improve their muscle function, reduce fatigue, and enhance their quality of life by helping cells produce energy more efficiently and protecting them from damage. Coenzyme Q10 (CoQ10) is a potent antioxidant that has been studied for its potential to improve muscle function and reduce oxidative stress in various conditions.

Who is the study for?
This trial is for teens aged 13-18 with genetically confirmed Prader-Willi Syndrome, weighing over 35 kg and in good health. They must be able to do exercise tests and take oral meds. Girls must use effective birth control, and boys need to ensure contraception during the study.
What is being tested?
The trial is testing Coenzyme Q10 against a placebo in patients with PWS to see if it improves muscle weakness and energy metabolism issues associated with the syndrome. Participants will either receive CoQ10 or a placebo without knowing which one they are taking.
What are the potential side effects?
While not specified here, common side effects of Coenzyme Q10 may include upset stomach, loss of appetite, nausea, vomiting, diarrhea; allergic reactions can occur but are rare.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I weigh more than 35 kilograms.
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I am between 13 and 18 years old with confirmed Prader-Willi Syndrome.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have heart disease with heart failure.
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I have diabetes.
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I have a long-term lung condition that is not asthma.
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I am currently taking CoQ10 supplements.
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I have thyroid disease or am on thyroid medication.
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I have a bleeding disorder.
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I have a skin condition.
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I have liver disease or a blockage in my bile duct.
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I am on medication for heart rhythm, blood pressure, or heart failure.
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I have a stomach disorder.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Three minute step test
Secondary study objectives
6 Minute walk test
Childhood Health Assessment Questionnaire (CHAQ)
Hand Grip Test
+6 more
Other study objectives
Magnetic Resonance Spectroscopy
Oxygen
Ergometry

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Coenzyme QExperimental Treatment1 Intervention
Each patient will be asked to take part in a 6 wk trial of pharmaceutical grade CoQ10 and will be randomly assigned to a start time. There will be a 6 wk washout period between treatment and placebo arms. At baseline, if this is the first arm, testing will include determination of muscle function based on our 3 min step test, muscle power (maximal jump, handgrip), strength (Queens' Square), endurance (6MWT), fatigue (PedsQL fatigue scale), physical activity level (3DPAR), attention (ADHDT scale), cognition (MoCA), physical function (CHAQ).and quality of life (PedQL). Following the 6 wk CoQ10 trial, testing will include repeat determination of all of the above as well as determination of total aerobic capacity (maximum cycle ergometry) and muscle metabolism (31P-MRS ergometry).
Group II: PlaceboPlacebo Group1 Intervention
Each patient will be asked to take part in a 6 wk trial of placebo and will be randomly assigned to a start time. There will be a 6 wk washout period between treatment and placebo arms. At baseline, if this is the first arm, testing will include determination of muscle function based on our 3 min step test, muscle power (maximal jump, handgrip), strength (Queens' Square), endurance (6MWT), fatigue (PedsQL fatigue scale), physical activity level (3DPAR), attention (ADHDT scale), cognition (MoCA), physical function (CHAQ).and quality of life (PedQL). Following the 6 wk placebo trial, testing will include repeat determination of all of the above as well as determination of total aerobic capacity (maximum cycle ergometry) and muscle metabolism (31P-MRS ergometry).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Coenzyme Q10
2016
Completed Phase 4
~1190

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Prader-Willi Syndrome (PWS) involves symptoms like muscle weakness and exercise intolerance, which may be linked to defects in energy metabolism. Coenzyme Q10, which enhances cellular energy metabolism by improving mitochondrial function and ATP production, is used to potentially address these issues. This treatment could help alleviate muscle weakness and improve exercise tolerance in PWS patients, although its full impact on cellular metabolism and clinical outcomes is not yet fully understood.
Treatment of Cognitive Deficits in Genetic Disorders: A Systematic Review of Clinical Trials of Diet and Drug Treatments.

Find a Location

Who is running the clinical trial?

The Hospital for Sick ChildrenLead Sponsor
704 Previous Clinical Trials
6,956,877 Total Patients Enrolled
2 Trials studying Prader-Willi Syndrome
58 Patients Enrolled for Prader-Willi Syndrome
Foundation for Prader-Willi ResearchOTHER
13 Previous Clinical Trials
1,174 Total Patients Enrolled
12 Trials studying Prader-Willi Syndrome
994 Patients Enrolled for Prader-Willi Syndrome
Ingrid Tein, MDPrincipal InvestigatorThe Hospital for Sick Children
2 Previous Clinical Trials
37 Total Patients Enrolled

Media Library

Coenzyme Q10 (Dietary Supplement) Clinical Trial Eligibility Overview. Trial Name: NCT03831425 — Phase 2
Prader-Willi Syndrome Research Study Groups: Coenzyme Q, Placebo
Prader-Willi Syndrome Clinical Trial 2023: Coenzyme Q10 Highlights & Side Effects. Trial Name: NCT03831425 — Phase 2
Coenzyme Q10 (Dietary Supplement) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03831425 — Phase 2
~0 spots leftby Oct 2025