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Metabolic Modulator

DCA for Pyruvate Dehydrogenase Deficiency (DCA/PDCD Trial)

Phase 3
Waitlist Available
Led By Richard Neibeger, MD
Research Sponsored by University of Florida
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Presence of a known pathogenic mutation of a gene that is specifically associated with PDCD
Age 6 months through 17 years
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 9 months
Awards & highlights

DCA/PDCD Trial Summary

This trial is a study to test if the drug dichloroacetate (DCA) can treat children with a deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency can cause a fatal metabolic disease of childhood, and there is no proven treatment currently. DCA is a potential targeted therapy for PDCD because it has the ability to increase the catalytic activity and stability of the enzyme complex. FDA has designated DCA as an Orphan Product for congenital lactic acidosis. The trial will use a novel Observer reported outcome (ObsRO) survey to measure efficacy, which will be completed by the study

Who is the study for?
This trial is for children aged 6 months to 17 years with Pyruvate Dehydrogenase Complex Deficiency (PDCD), evidenced by a known pathogenic gene mutation and clinical or metabolic features of PDCD. It excludes those with unrelated liver disease, amino acid metabolism disorders, pregnancy, fatty acid oxidation disorders, other genetic mitochondrial diseases besides PDCD, renal insufficiency, or secondary lactic acidosis.Check my eligibility
What is being tested?
The study tests Dichloroacetate (DCA) against a placebo in young children with PDCD. DCA could potentially stabilize the enzyme complex affected by this condition. The trial's effectiveness will be measured using an Observer reported outcome survey completed by the child's parent or caregiver.See study design
What are the potential side effects?
While specific side effects are not listed here, DCA may cause various reactions depending on individual tolerance and response to treatment. Parents/caregivers should monitor for any unusual symptoms or changes in their child’s health.

DCA/PDCD Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a genetic mutation linked to programmed cell death.
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I am between 6 months and 17 years old.

DCA/PDCD Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~9 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 9 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
The efficacy will be measured between the groups by using the Observer Reported Outcome (ObsRO) measure of health.
The number of participants with adverse events will be compared between the groups.
Secondary outcome measures
Blood lactate levels between the groups.
Karnofsky/Lansky Performance Status
Plasma β-hydroxybutyrate (β-OHB) concentrations between the groups.
Other outcome measures
The efficacy will be measured using the Observer Reported Outcome (ObsRO) scale of health based on the age at randomization between the groups
The efficacy will be measured using the Observer Reported Outcome (ObsRO) scale of health based on the dietary fat intake between the groups
The efficacy will be measured using the Observer Reported Outcome (ObsRO) scale of health based on the genetic-based dosing between the groups.
+2 more

DCA/PDCD Trial Design

2Treatment groups
Active Control
Placebo Group
Group I: Dichloroacetate, then PlaceboActive Control3 Interventions
This group will start on the Dichloroacetate (DCA) treatment which will last for 4 months. After 4 months a 1 month washout period will occur. After the 1 month the group will crossover to the placebo treatment for 4 months. 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
Group II: Placebo, then DichloroacetatePlacebo Group3 Interventions
This group will start on the placebo treatment which will last for 4 months. After 4 months a 1 month washout period will occur. After the 1 month the group will crossover to the Dichloroacetate (DCA) treatment for 4 months. 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

Find a Location

Who is running the clinical trial?

Food and Drug Administration (FDA)FED
174 Previous Clinical Trials
1,333,658 Total Patients Enrolled
Saol Therapeutics IncIndustry Sponsor
2 Previous Clinical Trials
164 Total Patients Enrolled
Medosome Biotec LLCIndustry Sponsor
1 Previous Clinical Trials
45 Total Patients Enrolled

Media Library

Dichloroacetate (DCA) (Metabolic Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT02616484 — Phase 3
Pyruvate Dehydrogenase Deficiency Research Study Groups: Placebo, then Dichloroacetate, Dichloroacetate, then Placebo
Pyruvate Dehydrogenase Deficiency Clinical Trial 2023: Dichloroacetate (DCA) Highlights & Side Effects. Trial Name: NCT02616484 — Phase 3
Dichloroacetate (DCA) (Metabolic Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02616484 — Phase 3
~5 spots leftby Mar 2025
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