In short
Clinical trials are investigating Ubiquinol in people with mitochondrial disorders and cerebellar ataxias. These studies are looking at safety and possible benefit, including changes in daily function, in selected patient groups.
Key points
- Clinical trials of Ubiquinol are being studied in people with mitochondrial disorders and cerebellar ataxias. The main trial listed is a Phase 2 interventional study planned for 90 participants. It was designed to check safety by tracking adverse effects and to look for changes in daily function using the Vineland Adaptive Behaviour Scale, III edition. The target groups included people with a confirmed genetic diagnosis of mitochondrial disease and people with cerebellar ataxia, with or without a molecular diagnosis. The trial status is Withdrawn, so it did not continue as planned.
Trial overview
This clinical study investigated Ubiquinol in patients with mitochondrial disorders and cerebellar ataxias. 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.
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. This means the study was aimed at selected patients with specific nerve and muscle-related conditions, not at the general population.
Mitochondrial disorders are illnesses linked to problems in the cell structures that help make energy, and cerebellar ataxia affects balance and coordination. The source data does not list other eligibility details such as age limits or laboratory requirements.
Trial design and phase
This was an interventional study, which means researchers planned to give a treatment and then observe the results. The trial was in Phase 2, a stage that often looks at early signs of benefit while also continuing safety checks.
The planned intervention was listed as Quinol 10 polvo estabilizado, sobres dispersables de 150mg, given orally at 30 mg/kg. The source identifies this intervention in the trial record, but it does not provide more detailed study procedures in the available data.
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. In simple terms, the researchers planned to ask participants about any unwanted health problems during each visit.
The other main outcome was the change from baseline in the Vineland Adaptive Behaviour Scale, III edition (VABS-III) at month 18. This scale measures how well a person manages everyday life skills, so the study was also looking at possible changes in daily function.
The trial record does not list additional secondary outcomes in the provided data.
Status and enrollment
The study status is Withdrawn, meaning it was stopped before completion and did not continue as planned. The planned enrollment was 90 participants.
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.