Lactic AcidosisPhase 3NCT00004490
Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis
A Phase 3 study testing sodium dichloroacetate in people with Lactic Acidosis, run by University of Florida.
Trial summary
OBJECTIVES: I. Compare the safety of sodium dichloroacetate (DCA) vs placebo in children with congenital lactic acidosis. II. Determine the quality of life of these patients. III. Determine the pharmacokinetics and metabolic fate of DCA over the course of drug administration in these patients. Read more about this study
- Who is it for?
- Ages 3 months to 18 years
- Conditions the study is for
- Lactic Acidosis
- What is being tested?
- sodium dichloroacetate
- How many people
- 45 participants
- Where
- No sites listed yet
- Study dates
- Oct 1998to Sep 2002
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of congenital lactic acidosis (CLA) meeting the following criteria: Three basal venous lactates at least 2.5 mM, arterial lactates at least 2.0 mM, or CSF lactates at least 2.5 mM OR any combination of these, obtained over at least 1 month and within 6 months OR Increase in blood lactate at least 1.0 mM over basal following a carbohydrate meal challenge
AND
Enzymatic or molecular genetic proof of a defect of pyruvate dehydrogenase complex, one or more respiratory chain enzymes, or a Krebs cycle enzyme OR Over production of C14-lactate from C14-glucose by cultured skin fibroblasts
AND
Ability to withstand an 8 hour (if 2 years and under) or 12 hour (if over 2 years) fast without developing hypoglycemia (blood glucose less than 50 mg/dL)
No secondary lactic acidosis due to impaired oxygenation or circulation
No hyperlactatemia associated with proven biotinidase deficiency (biotin responsive CLA) or with enzyme deficiencies of gluconeogenesis
No primary, defined organic acidurias other than lactic acidosis, for which effective therapy is available (e.g., propionic aciduria)
No primary disorders of amino acid metabolism or fatty acid oxidation
No malabsorption syndromes associated with D-lactic acidosis
--Prior/Concurrent Therapy--
No chronic dialysis
--Patient Characteristics--
Hepatic: No primary hepatic disease unrelated to CLA
Renal: Creatinine less than 1.2 mg/dL OR Creatinine clearance at least 60 mL/min
Other: No concurrent infection or fever
About this study
OBJECTIVES:
- Compare the safety of sodium dichloroacetate (DCA) vs placebo in children with congenital lactic acidosis.
II. Determine the quality of life of these patients.
III. Determine the pharmacokinetics and metabolic fate of DCA over the course of drug administration in these patients.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, crossover study. Patients are stratified according to age (3 months to 2 years vs over 2 to 18 years).
All patients receive at least 12 months of sodium dichloroacetate (DCA) during a 2 year period of double blind, crossover evaluation of DCA and placebo by mouth.
Quality of life is assessed before treatment and periodically during treatment.
Completion date provided represents the completion date of the grant per OOPD records
- Conditions
- Lactic Acidosis
- Being tested
- sodium dichloroacetate
- Keywords
- disease-related problem/condition, inborn errors of metabolism, lactic acidosis, quality of life, rare disease
- Medical subject headings
- Acidosis, Lactic, Metabolism, Inborn Errors, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sodium dichloroacetateDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startOct 1998
- Study completionSep 2002
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lactic AcidosisStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Enrolment
- 45 participants
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Investigators
- Peter W. Stacpoole, Study chair, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004490
- Sponsor's protocol ID
- 199/14271
- Other IDs
- UF-G-FDR001500, UF-G-183-92
- Status last verified
- Apr 2000
- First submitted
- 18 Oct 1999
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis
- ClinicalTrials.gov ID
- NCT00004490
- Acronym / sponsor ID
- 199/14271
- Conditions
- Lactic Acidosis
- Keywords
- disease-related problem/condition, inborn errors of metabolism, lactic acidosis, quality of life, rare disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Study start
- Oct 1998
- Study completion
- Sep 2002
- First posted
- 19 Oct 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES:
- Compare the safety of sodium dichloroacetate (DCA) vs placebo in children with congenital lactic acidosis.
II. Determine the quality of life of these patients.
III. Determine the pharmacokinetics and metabolic fate of DCA over the course of drug administration in these patients.
Detailed description
PROTOCOL OUTLINE: This is a randomized, double blind, crossover study. Patients are stratified according to age (3 months to 2 years vs over 2 to 18 years).
All patients receive at least 12 months of sodium dichloroacetate (DCA) during a 2 year period of double blind, crossover evaluation of DCA and placebo by mouth.
Quality of life is assessed before treatment and periodically during treatment.
Completion date provided represents the completion date of the grant per OOPD records
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 3 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| sodium dichloroacetate | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
Related trials for Lactic Acidosis
RecruitingPhase 3
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
Not yet recruitingPhase 1/2
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
RecruitingObservational study
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Mitochondrial Disorders, Mitochondrial Genetic Disorders
RecruitingObservational study
Lactic Acidosis and Prior Metformin Treatment
Type 2 Diabetes
RecruitingObservational study
Clinical and Biological Signs of Dapagliflozin Overdose in ICU Patients With Metformin Poisoning
Drug Overdose, Metformin
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter W. Stacpoole study chair
University of Florida
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing sodium dichloroacetate in people with Lactic Acidosis, run by University of Florida.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2015.