Expanded Access Treatment Protocol With DCA for Patients With PDCD

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

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.

Study Overview

• Status: Available
• Conditions: Pyruvate Dehydrogenase Complex Deficiency
• Intervention / Treatment: Drug: Dichloroacetate (DCA)

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.

• Study Type: Expanded Access
• Expanded Access Type: Treatment IND/Protocol

Contacts and Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: N/A

Description

Inclusion Criteria:

1. Ages 0 through adulthood
2. Presence of characteristic clinical or metabolic features of PDCD and
3. Presence of a known pathogenic mutation of a gene that is specifically associated with PDC (PDHA1, PDHB, DLAT, PDHX, DLD).
4. Females of reproductive age must be willing to use an effective method of barrier contraception for the duration of the study.-

Exclusion Criteria:

1. A genetic mitochondrial disease other than those stipulated under inclusion criteria
2. Primary, defined organic acidurias other than lactic acidosis (e.g., propionic aciduria)
3. Primary disorders of amino acid metabolism
4. Primary disorders of fatty acid oxidation
5. Secondary lactic acidosis due to impaired oxygenation or circulation (e.g., due to severe cardiomyopathy or congenital heart defects)
6. Malabsorption syndromes associated with D-lactic acidosis
7. Renal insufficiency, defined as 1) a requirement for chronic dialysis or 2) serum creatinine ≥ 1.2 mg/dl or creatinine clearance <60 ml/min
8. Primary hepatic disease unrelated to PDCD
9. Pregnancy or breast feeding -

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Saol Therapeutics Inc
• Collaborators: AnovoRx
• Investigators: Study Chair: Kiki Diorgu, M.D., Saol Therapeutics Inc

Study record dates

Study Registration Dates

• First Submitted: April 1, 2025
• First Submitted That Met QC Criteria: April 8, 2025
• First Posted (Actual): April 17, 2025

Study Record Updates

• Last Update Posted (Actual): May 8, 2025
• Last Update Submitted That Met QC Criteria: May 5, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: PDCD
• Additional Relevant MeSH Terms: Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Neurobehavioral Manifestations; Heredodegenerative Disorders, Nervous System; Mental Retardation, X-Linked; Intellectual Disability; Genetic Diseases, X-Linked; Carbohydrate Metabolism, Inborn Errors; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Pyruvate Metabolism, Inborn Errors; Mitochondrial Diseases; Pyruvate Dehydrogenase Complex Deficiency Disease
• Other Study ID Numbers: SL 1009 EA-01