Table of Contents
Trial overview
This clinical study investigated Ubiquinol in patients with mitochondrial disorders and cerebellar ataxias.[1] The brief summary says the goal was to determine safety and efficacy in people with a confirmed genetic diagnosis of mitochondrial disease or with cerebellar ataxia, with or without a molecular diagnosis.[1]
Who was studied
The trial focused on two main patient groups: people with a confirmed genetic diagnosis of mitochondrial disease, and people with cerebellar ataxia whether or not they had a molecular diagnosis.[1] This means the study was aimed at selected patients with specific nerve and muscle-related conditions, not at the general population.[1]
Mitochondrial disorders are illnesses linked to problems in the cell structures that help make energy, and cerebellar ataxia affects balance and coordination.[1] The source data does not list other eligibility details such as age limits or laboratory requirements.[1]
Trial design and phase
This was an interventional study, which means researchers planned to give a treatment and then observe the results.[1] The trial was in Phase 2, a stage that often looks at early signs of benefit while also continuing safety checks.[1]
The planned intervention was listed as Quinol 10 polvo estabilizado, sobres dispersables de 150mg, given orally at 30 mg/kg.[1] The source identifies this intervention in the trial record, but it does not provide more detailed study procedures in the available data.[1]
What was measured
The main safety outcome was the evaluation of adverse effects related to Ubiquinol over 18 months, based on subject reporting during study visits.[1] In simple terms, the researchers planned to ask participants about any unwanted health problems during each visit.[1]
The other main outcome was the change from baseline in the Vineland Adaptive Behaviour Scale, III edition (VABS-III) at month 18.[1] This scale measures how well a person manages everyday life skills, so the study was also looking at possible changes in daily function.[1]
The trial record does not list additional secondary outcomes in the provided data.[1]
Status and enrollment
The study status is Withdrawn, meaning it was stopped before completion and did not continue as planned.[1] The planned enrollment was 90 participants.[1]
Because the trial was withdrawn, the available record is mainly useful for understanding what the researchers intended to study, rather than for showing final results.[1]
