FDA Sponsored Phase 3 Trial of LIQ-NOL® in Mitochondrial Diseases

This study is sponsored by the FDA Orphan Products Division, Tishcon Corp., & General Clinical Research Centers funded by the National Institutes of Health.

Study Information

The study will help provide information to determine if Coenzyme Q10 (CoQ10) is a safe and effective treatment for children with mitochondrial diseases. There are no cures for mitochondrial diseases at this time. Treatments have sought to help reduce the symptoms or delay the progression of the disease. CoQ10 is a mitochondrial cofactor and antioxidant that is a part of the process by which food and oxygen are converted into energy by cells.


Children age 12 months to 17 years with biochemical or genetic proof of a mitochondrial disease. There are factors that could exclude some children with a mitochondrial disorder from participating. Ask the study coordinator if your child is eligible to participate.
If your child is not eligible, that dose not mean that he/she does not have a mitochondrial disorder, as there are many specific factors that are part of the selection process.

ParticipationYour participation is important because: It will contribute to medical knowledge about mitochondrial diseases, help determine the benefit of CoQ10 in treating mitochondrial diseases, and help future clinical research trials look for specific treatments for people with mitochondrial deficiencies.

Potential Benefits

  1. Developmental evaluation of your child
  2. Evaluation of his/her motor skills
  3. Objective evaluation of effectiveness of CoQ10

Participating Centers

  • General Clinical Research Center, University of Florida, Gainesville, Florida
  • William T. Dahms Clinical Research Unit, University Hospitals Case Medical Center, Cleveland, Ohio
  • Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
  • Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada