Pyruvate Dehydrogenase Complex DeficiencyNCT06931262
Expanded Access Treatment Protocol With DCA for Patients With PDCD
A study testing Dichloroacetate (DCA) in people with Pyruvate Dehydrogenase Complex Deficiency, run by Saol Therapeutics Inc.
Trial summary
Expanded Access (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet the eligibility criteria to also have access to therapy that would be otherwise… Read more about this study
- Who is it for?
- Ages 0 to 17 years
- Conditions the study is for
- Pyruvate Dehydrogenase Complex Deficiency
- What is being tested?
- Dichloroacetate (DCA)
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- 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 0 to 17 years
- All sexes
- Child
Who may be able to join
- Ages 0 through adulthood
- Presence of characteristic clinical or metabolic features of PDCD and
- Presence of a known pathogenic mutation of a gene that is specifically associated with PDC (PDHA1, PDHB, DLAT, PDHX, DLD).
- Females of reproductive age must be willing to use an effective method of barrier contraception for the duration of the study.-
Who cannot join
- A genetic mitochondrial disease other than those stipulated under inclusion criteria
- Primary, defined organic acidurias other than lactic acidosis (e.g., propionic aciduria)
- Primary disorders of amino acid metabolism
- Primary disorders of fatty acid oxidation
- Secondary lactic acidosis due to impaired oxygenation or circulation (e.g., due to severe cardiomyopathy or congenital heart defects)
- Malabsorption syndromes associated with D-lactic acidosis
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- Renal insufficiency, defined as 1) a requirement for chronic dialysis or 2) serum creatinine ≥ 1.2 mg/dl or creatinine clearance <60 ml/min
- Primary hepatic disease unrelated to PDCD
- Pregnancy or breast feeding -
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Expanded Access Treatment Protocol With Dichloroacetate Sodium for Patients With Pyruvate Dehydrogenase Complex Deficiency
Expanded Access (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet the eligibility criteria to also have access to therapy that would be otherwise unavailable.
Read the detailed description
Expanded Access (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet the eligibility criteria to also have access to therapy that would be otherwise unavailable. The Expanded Access program (EAP) will also allow the sponsor to extend the collection of long term safety data for the current open label patients, while also expanding to an additional population of patients beyond those currently in the SL 1009-01 study.
- Being tested
- Dichloroacetate (DCA)
- Keywords
- PDCD
- Medical subject headings
- Pyruvate Dehydrogenase Complex Deficiency Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dichloroacetate (DCA)Drug
Study medication DCA is an oral solution mixed with an artificial sweetener containing aspartame and strawberry extract (50mg/mL) Participants will be genotyped to determine GSTZ1 (glutathione S-transferase Zeta-1) haplotype status, which will stratify this group into 1 of 2 dose regimens: EGT carriers will receive 12 mg/kg/12hr DCA. EGT non-carriers will receive 6 mg/kg/12 hr DCA.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pyruvate Dehydrogenase Complex DeficiencyStudies of the same condition, open and under way.
- 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
- Saol Therapeutics Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- AnovoRx
- Responsible party
- Sponsor
- Investigators
- Kiki Diorgu, M.D., Study chair, Saol Therapeutics Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06931262
- Sponsor's protocol ID
- SL 1009 EA-01
- Status last verified
- May 2025
- First submitted
- 1 Apr 2025
- Last updated
- 8 May 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Treatment Protocol With Dichloroacetate Sodium for Patients With Pyruvate Dehydrogenase Complex Deficiency
- ClinicalTrials.gov ID
- NCT06931262
- Acronym / sponsor ID
- SL 1009 EA-01
- Keywords
- PDCD
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Saol Therapeutics IncIndustry (pharma, biotech, devices)
- Collaborators
- AnovoRx
- First posted
- 17 Apr 2025 (actual)
- 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 (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet the eligibility criteria to also have access to therapy that would be otherwise unavailable.
Detailed description
Expanded Access (EA) will provide a transition to continue therapy for those patients who are currently in the open label extension of the Phase III study, SL 1009-01, while also allowing new patients diagnosed with PDCD who meet the eligibility criteria to also have access to therapy that would be otherwise unavailable. The Expanded Access program (EAP) will also allow the sponsor to extend the collection of long term safety data for the current open label patients, while also expanding to an additional population of patients beyond those currently in the SL 1009-01 study.
Study design
Who may qualify
- Ages
- 0 to 17 years
- Sex
- All
- Age groups
- Child
Key inclusion criteria (4 listed)
- Ages 0 through adulthood
- Presence of characteristic clinical or metabolic features of PDCD and
- Presence of a known pathogenic mutation of a gene that is specifically associated with PDC (PDHA1, PDHB, DLAT, PDHX, DLD).
- Females of reproductive age must be willing to use an effective method of barrier contraception for the duration of the study.-
Key exclusion criteria (9 listed)
- A genetic mitochondrial disease other than those stipulated under inclusion criteria
- Primary, defined organic acidurias other than lactic acidosis (e.g., propionic aciduria)
- Primary disorders of amino acid metabolism
- Primary disorders of fatty acid oxidation
- Secondary lactic acidosis due to impaired oxygenation or circulation (e.g., due to severe cardiomyopathy or congenital heart defects)
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 |
|---|---|---|
| Dichloroacetate (DCA)Study medication DCA is an oral solution mixed with an artificial sweetener containing aspartame and strawberry extract (50mg/mL) Participants will be genotyped to determine GSTZ1 (glutathione S-transferase Zeta-1) haplotype status, which will stratify this group into 1 of 2 dose regimens: EGT carriers will receive 12 mg/kg/12hr DCA. EGT non-carriers will receive 6 mg/kg/12 hr DCA. | 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 May 2025.
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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.
Kiki Diorgu, M.D. study chair
Saol Therapeutics Inc
Questions about this trial
What is this clinical trial studying?
A study testing Dichloroacetate (DCA) in people with Pyruvate Dehydrogenase Complex Deficiency, run by Saol Therapeutics Inc.
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 4 inclusion and 9 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 May 2025. 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.
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?
Contact Kyle Ashton, Ph.D. (770-274-2500, eap@saolrx.com), or a site near the patient. 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 May 2025; AzTrials last refreshed its copy on 28 Oct 2026.