MedTrace Logo

Expanded Access Treatment Protocol With DCA for Patients With PDCD

NCT06931262 · Status: AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2025-05-08

No results posted yet for this study

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.

Conditions

  • Pyruvate Dehydrogenase Complex Deficiency

Interventions

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.

Sponsors & Collaborators

  • AnovoRx

    collaborator UNKNOWN

  • Saol Therapeutics Inc

    lead INDUSTRY

Principal Investigators

  • Kiki Diorgu, M.D. · Saol Therapeutics Inc

Eligibility

Min Age
0 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06931262 on ClinicalTrials.gov