Status:
AVAILABLE
Expanded Access Treatment Protocol With DCA for Patients With PDCD
Lead Sponsor:
Saol Therapeutics Inc
Collaborating Sponsors:
AnovoRx
Conditions:
Pyruvate Dehydrogenase Complex Deficiency
Eligibility:
All Genders
Up to 17 years
Brief 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...
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...
Eligibility Criteria
Inclusion
- 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.-
Exclusion
- 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
- 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 -
Key Trial Info
Start Date :
Trial Type :
EXPANDED_ACCESS
End Date :
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT06931262
Last Update
May 8 2025
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