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Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

NCT02616484 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 34

Last updated 2025-05-04

No results posted yet for this study

Summary

The objective of this research study is to conduct a pivotal phase 3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital lactic acidosis and is a frequently fatal metabolic disease of childhood for which no proven treatment exists. The investigators predict that DCA represents targeted potential therapy for PDCD because of its ability to increase both the catalytic activity and stability of the enzyme complex. The conclusions of numerous laboratory and clinical investigations are consistent with this postulate and have led to the designation of DCA as an Orphan Product for congenital lactic acidosis by the Food and Drug Administration.

A novel Observer reported outcome (ObsRO) survey that is completed by study participant's parent/caregiver, is the efficacy outcome measure.

Funding Source - FDA OOPD

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.

OTHER

Placebo

Participants will receive the same volume of placebo in liquid form given during DCA treatment arm. Liquid will be an exact replication of DCA formulation with no DCA added.

GENETIC

Genotype

Participants will be genotyped to determine GSTZ1 haplotype status.

Sponsors & Collaborators

  • Medosome Biotec LLC

    collaborator INDUSTRY

  • Saol Therapeutics Inc

    lead INDUSTRY

Principal Investigators

  • Richard Neibeger, MD · University of Florida

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
6 Months
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-07-14
Primary Completion
2025-08-27
Completion
2025-08-27
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT02616484 on ClinicalTrials.gov