Mitochondrial Complex I Dysfunction in PWS

• Register: ClinicalTrials.gov
• Last refreshed on: 24 April 2023
• Main ID: NCT03831425
• Date of registration: 22/01/2019
• Prospective Registration: Yes
• Primary sponsor: The Hospital for Sick Children
• Public title: Mitochondrial Complex I Dysfunction in PWS
• Scientific title: Mitochondrial Complex I Dysfunction in Prader Willi Syndrome: A New Therapeutic Target
• Date of first enrolment: June 1, 2023
• Target sample size: 14
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/show/NCT03831425
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada

Contacts

• Name: Bushra Momtaz

• Name: Ingrid Tein, MD
• Affiliation: The Hospital for Sick Children

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Consent provided

2. patients with genetically confirmed PWS aged 13 to 18 years (n=14)

3. ability to cooperate with exercise testing

4. weight > 35.0 kg

5. in good general health as evidenced by medical history

6. able to take oral medications

7. 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

8. 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 Criteria:

1. unable to perform exercise tests,

2. already taking CoQ10

3. having liver disease or bile duct blockage,

4. having thyroid disease or taking thyroid medications

5. presence of diabetes

6. taking antiarrhythmics or antihypertensives or anti-failure medications

7. presence of gastric disorders

8. presence of skin disorders

9. pregnancy or lactation

10. lactose intolerance

11. known allergic reaction to CoQ10 or components of preparation.

12. treatment with another investigational drug or other intervention

13. current smoker or tobacco use within 6 months

14. current cannabis user or use within 6 months

15. presence of chronic respiratory disease other than asthma

16. presence of cardiac disease with cardiac insufficiency/CHF

17. presence of MR-incompatible metal in body, metal devices or tattoos

18. presence of a bleeding disorder

19. gelatin intolerance

20. clinically significant findings in laboratory tests at screening



Age minimum: 13 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Prader-Willi Syndrome

Intervention(s)

Dietary Supplement: Coenzyme Q10

Other: Placebo

Primary Outcome(s)

Three minute step test [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Secondary Outcome(s)

Vertical Jump Test [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Hand Grip Test [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Three Day Physical Activity Record (3DPAR) [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

6 Minute walk test [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Childhood Health Assessment Questionnaire (CHAQ) [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Montreal Cognitive Assessment (MOCA) [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Pediatric Quality of Life Index (PedQL) [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

SWAN Rating Scale for Attention Deficit Hyperactivity Disorder (ADHD) [Time Frame: Baseline, week 8 (after 6 weeks on study drug or placebo), [6 week washout], week 20 (after 6 weeks study drug or placebo)]

Secondary ID(s)

1000053066

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Foundation for Prader-Willi Research