Expanded Access Treatment Protocol With DCA for Patients With PDCD
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age0-17
SponsorSaol Therapeutics Inc
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.-
Disqualifiers
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
Trial design
Treatments tested in this trial
Dichloroacetate (DCA)
DrugStudy 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.
Treatment groups
No treatment groups listed
Trial outcomes
This trial has no outcomes listed.Locations
Sponsors and contacts
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Saol Therapeutics Inc
Lead sponsor
AnovoRx
Collaborator
Source ClinicalTrials.gov