Mitochondrial disorders and/or cerebellar ataxias
Conditions
Brief summary
Evaluation of adverse effects related to Ubiquinol based on subject reporting for 18 months. Subject interviews during each visit assessing the presence or absence of adverse events., Change from baseline in the Vineland Adaptive Behaviour Scale, III edition (VABS-III) at month 18
Detailed description
Effect on (a) cognitive function, (b) language skills, (c) quality of life, (d) symptom severity, treatment response and efficacy (e) movement disorder severity of 18 months of treatment with Ubiquinol evaluated by the change from baseline in the following assessments at month 18, Usefulness of CoQ10 plasma monitoring during treatment determined by change from baseline in the levels of plasma CoQ10, assessed at Visits V1, V3, V4, V5, and V6, Change from baseline in other biomarkers measured by blood analytical assessments (Visits V1, V3, V5, V6): FGF21, GDF15 analysis in plasma.
Interventions
DRUGQuinol 10 polvo estabilizado
DRUGsobres dispersables de 150mg
Sponsors
Fundacio Sant Joan De Deu
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluation of adverse effects related to Ubiquinol based on subject reporting for 18 months. Subject interviews during each visit assessing the presence or absence of adverse events., Change from baseline in the Vineland Adaptive Behaviour Scale, III edition (VABS-III) at month 18 | — |
Secondary
| Measure | Time frame |
|---|---|
| Effect on (a) cognitive function, (b) language skills, (c) quality of life, (d) symptom severity, treatment response and efficacy (e) movement disorder severity of 18 months of treatment with Ubiquinol evaluated by the change from baseline in the following assessments at month 18, Usefulness of CoQ10 plasma monitoring during treatment determined by change from baseline in the levels of plasma CoQ10, assessed at Visits V1, V3, V4, V5, and V6, Change from baseline in other biomarkers measured by blood analytical assessments (Visits V1, V3, V5, V6): FGF21, GDF15 analysis in plasma. | — |
Countries
Spain
Outcome results
None listed