Expanded Access Treatment Protocol With DCA for Patients With PDCD

May 5, 2025 updated by: Saol Therapeutics Inc

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

Intervention / Treatment

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

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Saol Therapeutics Inc

Collaborators

AnovoRx

Investigators

  • Study Chair: Kiki Diorgu, M.D., Saol Therapeutics Inc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

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

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SL 1009 EA-01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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