The purpose of this clinical trial is to determine whether the drug NTBC(Nitisione, Orfadinin TM) reduces or prevents the undesirable side effects (toxicity), of the investigational drug, dichloroacetate (DCA), in patients with Congenital Lactic Acidosis.
We have designed a double blind, parallel clinical trial that has been approved by the FDA to assess the ability of NTBC to reduce the toxicity of DCA. During the trial, patients will not know which drugs they are receiving. There will be two groups of patients for the 13-month trial. For the first month of the trial, all patients will be placed on a weight-maintaining, low tyrosine diet (LTD). Afterwards, patients will be separated into two groups. One group will receive DCA + NTBC + LTD and the other group will receive DCA + Placebo + LTD for the remaining 12 months.
Patients are admitted 6 times to our General Clinical Research Center at the University of Florida. The first visit will require 5 days of baseline tests and dietary education, while the remaining admissions will take 2-3 days. A parent stays in the same room as his or her child throughout each visit.
The diet for this trial is similar to the patient's normal diet at home. However a special drink that is low in the amino acids tyrosine and phenylalanine will supplement it. Our dietician will work closely with you to create a diet that is specific to each patient's nutritional needs. There are some foods that will be restricted as part of the diet, including high protein foods such as meat, cheese and …