Mitochondrial Enzyme DeficienciesCLANCT01797276
Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
A study testing Dichloroacetate in people with Mitochondrial Enzyme Deficiencies, run by University of Florida at 1 site in United States.
Trial summary
Expanded access to DCA as continued treatment for congenital lactic acidosis. Read more about this study
- Who is it for?
- Ages 18 to 50 years, women only
- Conditions the study is for
- Mitochondrial Enzyme Deficiencies
- What is being tested?
- Dichloroacetate
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 18 to 50 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Diagnosis of congenital lactic acidosis
- Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria:
- Intolerance to DCA
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Florida Box 100226
Gainesville, Florida, 32610-0226
About this study
Expanded access to DCA as continued treatment for congenital lactic acidosis.
Read the detailed description
Subjects who participated in a Phase 3 Randomized Controlled Trial of DCA for treatment of Congenital Lactic Acidosis (CLA) are eligible to continue treatment with investigational medication DCA at the same dose of 25mg/kg/day for expanded access. Study participants must travel to the study site for bi-annual evaluation by the study investigator. Bi-annual evaluation will include an interim medical history review, physical exam, blood collection for DCA trough level, and urine pregnancy testing (if indicated).
- Conditions
- Mitochondrial Enzyme Deficiencies
- Being tested
- Dichloroacetate
- Keywords
- Dichloroacetate (DCA), Congenital Lactic Acidosis (CLA), acidosis, orphan disease
- Medical subject headings
- Acidosis, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DichloroacetateDrug
Also called DCA
Subjects are given DCA 25mg/kg in a divided dose twice a day.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial Enzyme DeficienciesStudies 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Peter W. Stacpoole, PhD, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01797276
- Sponsor's protocol ID
- IRB#420-2005
- Acronym
- CLA
- Status last verified
- Aug 2017
- First submitted
- 15 Feb 2013
- Last updated
- 8 Aug 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
- ClinicalTrials.gov ID
- NCT01797276
- Acronym / sponsor ID
- CLA / IRB#420-2005
- Conditions
- Mitochondrial Enzyme Deficiencies
- Keywords
- Dichloroacetate (DCA), Congenital Lactic Acidosis (CLA), acidosis, orphan disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- University of FloridaUniversity, hospital or other
- First posted
- 22 Feb 2013 (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.
Expanded access to DCA as continued treatment for congenital lactic acidosis.
Detailed description
Subjects who participated in a Phase 3 Randomized Controlled Trial of DCA for treatment of Congenital Lactic Acidosis (CLA) are eligible to continue treatment with investigational medication DCA at the same dose of 25mg/kg/day for expanded access. Study participants must travel to the study site for bi-annual evaluation by the study investigator. Bi-annual evaluation will include an interim medical history review, physical exam, blood collection for DCA trough level, and urine pregnancy testing (if indicated).
Study design
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Diagnosis of congenital lactic acidosis
- Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria:
- Intolerance to DCA
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| DichloroacetateAlso: DCASubjects are given DCA 25mg/kg in a divided dose twice a day. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Aug 2017.
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, PhD, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Dichloroacetate in people with Mitochondrial Enzyme Deficiencies, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 0 exclusion criteria. 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: status not confirmed, as of the sponsor's last update on 8 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
Are results available for this study?
Results are not available in the source record.
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 8 Aug 2017.