Status:
WITHDRAWN
Mitochondrial Complex I Dysfunction in PWS
Lead Sponsor:
The Hospital for Sick Children
Collaborating Sponsors:
Foundation for Prader-Willi Research
Conditions:
Prader-Willi Syndrome
Eligibility:
All Genders
13-18 years
Phase:
PHASE2
Brief Summary
Prader-Willi Syndrome (PWS) is characterized by profound infantile hypotonia, growth delay, cognitive impairment, muscle weakness and exercise intolerance. Studies have suggested that a defect in ener...
Detailed Description
Investigators will determine the clinical efficacy of CoQ10 in a prospective, randomized, double-blind, controlled, cross-over study. Primary outcome will include determination of muscle function base...
Eligibility Criteria
Inclusion
- Consent provided
- patients with genetically confirmed PWS aged 13 to 18 years (n=14)
- ability to cooperate with exercise testing
- weight \> 35.0 kg
- in good general health as evidenced by medical history
- able to take oral medications
- for females of reproductive potential, use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 8 weeks after the end of study intervention administration
- for males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner during study participation and for an additional 8 weeks after the end of study intervention administration
Exclusion
- unable to perform exercise tests,
- already taking CoQ10
- having liver disease or bile duct blockage,
- having thyroid disease or taking thyroid medications
- presence of diabetes
- taking antiarrhythmics or antihypertensives or anti-failure medications
- presence of gastric disorders
- presence of skin disorders
- pregnancy or lactation
- lactose intolerance
- known allergic reaction to CoQ10 or components of preparation.
- treatment with another investigational drug or other intervention
- current smoker or tobacco use within 6 months
- current cannabis user or use within 6 months
- presence of chronic respiratory disease other than asthma
- presence of cardiac disease with cardiac insufficiency/CHF
- presence of MR-incompatible metal in body, metal devices or tattoos
- presence of a bleeding disorder
- gelatin intolerance
- clinically significant findings in laboratory tests at screening
Key Trial Info
Start Date :
June 1 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2024
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT03831425
Start Date
June 1 2023
End Date
June 1 2024
Last Update
June 18 2024
Active Locations (1)
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1
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8