NCT06931262

Expanded Access Treatment Protocol With DCA for Patients With PDCD

Expanded Access Treatment Protocol With Dichloroacetate Sodium for Patients With Pyruvate Dehydrogenase Complex Deficiency

• Status: Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Saol Therapeutics Inc · Industry
• Sex: All
• Age: 0 Years – 17 Years
• Healthy volunteers: —

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.

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.

Conditions

• Pyruvate Dehydrogenase Complex Deficiency

Interventions

Type	Name	Description
DRUG	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.

Timeline

• First posted: 2025-04-17
• Last updated: 2025-05-08