Home | | | Search | | | Browse | | | Resources | | | Help | | | What's New | | | About |
---|
Prevention of Dichloroacetate Toxicity
This study is currently recruiting patients.
Sponsored by: | FDA Office of Orphan Products Development |
---|---|
Information provided by: | FDA Office of Orphan Products Development |
Purpose
This is a study to determine the safety of dichloroacetate (DCA) with a low-tyrosine diet given with or without nitisinone (NTBC) in children with chronic lactic acidosis (CLA).
Condition | Treatment or Intervention |
---|---|
Acidosis, Lactic Chronic Disease |
Drug: Nitisinone (NTBC) Drug: Dichloroacetate Behavior: Low-tyrosin diet |
MedlinePlus related topics: Metabolic Disorders
Study Type: Interventional
Study Design: Prevention, Randomized, Double-Blind, Placebo Control, Safety/Efficacy Study
Expected Total Enrollment: 30
Study start: September 2001;
Study completion: August 2004
DCA is being studied for the treatment of patients with CLA, which is a rare collection of mitochondrial metabolism errors causing cellular energy failure and early death. DCA causes reversible liver and peripheral nerve toxicity and it interrupts both tyrosine and heme metabolism. The inhibitory effect of DCA on mammalian tyrosine metabolism elicits biochemical changes similar to those observed in hereditary tyrosinemia type I (HT). However, some reports and studies indicate substantial reduction in the biochemical and clinical consequences of HT may occur when patients are treated concomitantly with a low-tyrosine diet (LTD) and the chemical NTBC, which inhibits an early step in tyrosine catabolism. Possibly, the same dietary and pharmacologic interventions may mitigate or prevent toxicity associated with chronic DCA exposure.
Patients visit the Center 5 times over a 1-year period, usually for 2 to 3 days per visit, for an extensive series of clinical and biochemical tests. Visit 1 is for baseline examinations and blood and urine chemistries and to educate the patient on an LTD. This visit lasts approximately 7 days to determine acceptable circulating tyrosine concentrations for LTD formula at discharge. Patients are provided with tubes to take to local laboratories every 2 weeks for blood work. Patients are readmitted in 1 month to determine adherence to diet and serum tyrosine levels. Patients who evidence dietary compliance, no adverse effects, and a willingness to continue are placed in 1 of 2 treatment arms: DCA plus an LTD plus placebo or DCA plus an LTD plus NTBC. Thereafter, patients return during Months 5, 9, and 13, which completes their 1-year treatment phase.
Eligibility
Ages Eligible for Study: 3 Months - 21 Years, Genders Eligible for Study: Both
Criteria
Inclusion criteria:
Exclusion criteria:
Location and Contact Information
More Information
U.S. National Library of Medicine, Contact NLM Customer Service | ||||||||||||||
National Institutes of Health, Department of Health & Human Services | ||||||||||||||
Copyright, Privacy, Accessibility, Freedom of Information Act |