AVAILABLENot Applicable

Expanded Access Treatment Protocol With DCA for Patients With PDCD

Plain-language 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 unavailable.

Who can participate

Age range0 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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 criteria

✕. 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

Trial details

NCT IDNCT06931262

SponsorSaol Therapeutics Inc

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

Contact for this trial

Kyle Ashton, Ph.D.

770-274-2500 eap@saolrx.com

View on ClinicalTrials.gov More Pyruvate Dehydrogenase Complex Deficiency trials