Ignite Creation Date:
2024-05-05 @ 11:25 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00031161
Status:
COMPLETED
Last Update Posted:
2015-03-25
First Post:
2002-02-26
Brief Title:
Prevention of Dichloroacetate Toxicity
Sponsor:
University of Florida
Organization:
FDA Office of Orphan Products Development
Study Overview
Official Title:
Prevention of Dichloroacetate Toxicity
Status:
COMPLETED
Status Verified Date:
2002-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
Detailed 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
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| FD-R-002013-01 | None | None | None |