What side effects are associated with this type of intervention?

Coenzyme Q10 is being studied for its potential to enhance cellular energy metabolism in Prader-Willi Syndrome (PWS). While specific side effects of Coenzyme Q10 in PWS patients are not detailed, common side effects of Coenzyme Q10 supplementation in general may include gastrointestinal symptoms such as nausea, diarrhea, and stomach upset. Additionally, some individuals may experience headaches, dizziness, or allergic skin reactions. It is important for patients to discuss these potential side effects with their healthcare provider to ensure safe and effective use.

What prior FDA approvals exist?

Prader-Willi Syndrome (PWS) is a complex genetic disorder with no specific FDA-approved treatments targeting the underlying cause. Management typically focuses on symptom relief, including growth hormone therapy to address growth delay and muscle weakness. Coenzyme Q10, which enhances cellular energy metabolism, has been used in some PWS patients, but its efficacy and FDA approval status for this specific use remain unclear. Other treatments are generally supportive and symptomatic.

What other types of research exist?

Currently, there are no specific novel alternatives to Coenzyme Q10 mentioned for Prader-Willi Syndrome in the provided context. However, exploring treatments that enhance mitochondrial function or energy metabolism, such as other mitochondrial-targeted therapies or metabolic modulators, could be promising. Consulting with a healthcare provider for the latest research and clinical trials is recommended.

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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 weeks3 visits

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)

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

0 / 12Eligibility criteria met

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.