Phase IIa non-randomized open single-arm study, multicentre clinical trial to determine the efficacy and safety of coenzyme Q (Ubiquinol) for patients with mitochondrial disorders and cerebellar ataxias
interventional
N/A
1 country
2
To determine the safety and efficacy of Ubiquinol in patients with 1) a confirmed genetic diagnosis of mitochondrial disease or 2) cerebellar ataxia with or without molecular diagnosis.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
Not reported
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
August 26, 2024
Actual (reported)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
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.
Hospital Universitario 12 De Octubre
Madrid, 28041, Spain
Sant Joan De Deu Barcelona Hospital
Esplugues De Llobregat, 08950, Spain
Clinical Trial Unit
First Submitted
August 26, 2024
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