Prevention of Dichloroacetate Toxicity

Status Completed

Clinical Trial Summary

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).

Clinical Trial Description

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.

Completion date provided represents the completion date of the grant per OOPD records ;

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Masking: Double-Blind, Primary Purpose: Prevention

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00031161
Study type	Interventional
Source	FDA Office of Orphan Products Development
Contact
Status	Completed
Phase	N/A
Start date	September 2001
Completion date	September 2005

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT03153644` - Improving Contraceptive Care for Women With Medical Conditions
Recruiting	`NCT06058754` - Group-based [ADAPT] Versus One-to-one [Usual] Occupational Therapy (Go:OT Trial)	N/A
Completed	`NCT04082585` - Total Health Improvement Program Research Project
Recruiting	`NCT05558085` - Biomarker Cost-Benefit Analysis of EFNEP	N/A
Completed	`NCT04037436` - Functional Exercise and Nutrition Education Program for Older Adults	N/A
Not yet recruiting	`NCT05622422` - A Chronic Disease Self Care Management Pilot Study	N/A
Not yet recruiting	`NCT06016101` - Usefulness of the Medissimo Nurse Application for Supporting Medication Compliance in Elderly People With Chronic Polypathologies
Not yet recruiting	`NCT04954209` - Comparative Study in Long-term Commitment to Physical Activity After Two Different Resumption Programs
Not yet recruiting	`NCT04090593` - Chronic Disease Mobile Educational Experience	N/A
Not yet recruiting	`NCT03628963` - Optimizing Patient Usability Experience for Chronic Care	N/A
Completed	`NCT02390570` - Incorporating Patient Capacity Into the Clinical Landscape	N/A
Completed	`NCT02072941` - Preparing Spanish-speaking Older Adults for Advance Care Planning and Medical Decision Making (PREPARE)	N/A
Completed	`NCT01933789` - Improving Communication About Serious Illness	N/A
Terminated	`NCT02115971` - Jumping Exercises Approach in Individuals With Chronic Ankle Instability	N/A
Completed	`NCT02292940` - Consumer Health IT Tools: Impact on Experience, Access, and Outcomes for Patients With Complex Chronic Conditions
Completed	`NCT02017262` - Group Self-Management of Depression and Medical Illness	N/A
Completed	`NCT02307929` - Evaluation of Quality of Care - Nurse Allied Health Clinic Programme, HA	N/A
Completed	`NCT01458184` - Study of PhoneCare System to Treat Patients With Chronic Diseases	N/A
Completed	`NCT00333710` - Evaluating a Telehealth Treatment for Veterans With Hepatitis C and PTSD	N/A
Completed	`NCT00380536` - Medical Self-Management for Improving Health Behavior Among Individuals in Community Mental Health Settings	N/A

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.