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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 9 months

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

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Food and Drug Administration (FDA)FED

171 Previous Clinical Trials

1,331,572 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there other hospitals in North America conducting this same clinical trial?

"The trial is based out of 10 locations, the primary ones being University of Utah, Children's Hospital of Orange County, and Children's Hospital of Philadelphia."

Answered by AI

How does this treatment's safety profile look to patients?

"This particular Phase 3 trial has garnered enough evidence to support its efficacy, and there is also data suggesting that it is safe. Our team at Power rates this treatment as a 3 on our safety scale."

Answered by AI

Is this research study open to people who are younger than 75 years old?

"The age group that this clinical trial is recruiting from are children that are above 6 months old and below the age of 17."

Answered by AI

Are there any patients that can still sign up for this trial?

"The clinicaltrials.gov website says that this trial is not currently open for recruitment. The earliest posting date was July 14th, 2020 and the most recent update was on September 12th, 2022. There are 4 other trials recruiting patients right now though."

Answered by AI

Are there other instances where this treatment has been tried before in a clinical setting?

"There are 3 clinical trials underway for this treatment, 1 of which is in Phase 3. Many of the trials are based at Stanford University in California, but there are also 12 other locations running trials for this treatment."

Answered by AI

What are the other investigations like this one that have been conducted in the past?

"Research for this treatment began in 2020 with the first clinical trial taking place that year. That study was sponsored by Medosome Biotec LLC and included 34 participants. After Phase 3 approval was granted, there are now three ongoing studies involving 10 cities across one country."

Answered by AI

Who meets the standards to be a test subject for this clinical trial?

"This study is looking for 34 young patients, aged 6 months to 17 years old, that currently have pyruvate dehydrogenase complex deficiency. In order to be eligible, these individuals must also meet the following requirements: characteristic clinical or metabolic features of pyruvate dehydrogenase complex deficiency (PDCD), a known pathogenic mutation of a gene associated with PDCD."

Answered by AI

Recent research and studies

Molecular Genetics and MetabolismJournal

A Combined Therapeutic Approach for Pyruvate Dehydrogenase Deficiency Using Self-complementary Adeno-associated Virus Serotype-specific Vectors and Dichloroacetate

Han, Zongchao, Kristen Berendzen, Li Zhong, Ira Surolia, Nitin Chouthai, Weihong Zhao, Njeri Maina, Arun Srivastava, and Peter W. Stacpoole. 2008. “A Combined Therapeutic Approach for Pyruvate Dehydrogenase Deficiency Using Self-complementary Adeno-associated Virus Serotype-specific Vectors and Dichloroacetate”. Molecular Genetics and Metabolism. Elsevier BV. doi:10.1016/j.ymgme.2007.10.131.

Molecular Genetics and MetabolismJournal

Long-term Safety of Dichloroacetate in Congenital Lactic Acidosis

Abdelmalak, Monica, Alicia Lew, Ryan Ramezani, Albert L. Shroads, Bonnie S. Coats, Taimour Langaee, Meena N. Shankar, Richard E. Neiberger, S.H. Subramony, and Peter W. Stacpoole. 2013. “Long-term Safety of Dichloroacetate in Congenital Lactic Acidosis”. Molecular Genetics and Metabolism. Elsevier BV. doi:10.1016/j.ymgme.2013.03.019.

The Journal of Clinical PharmacologyJournal

Human Polymorphisms in the Glutathione Transferase Zeta 1/maleylacetoacetate Isomerase Gene Influence the Toxicokinetics of Dichloroacetate

Shroads, Albert L., Taimour Langaee, Bonnie S. Coats, Tracie L. Kurtz, John R. Bullock, David Weithorn, Yan Gong, et al.. 2012. “Human Polymorphisms in the Glutathione Transferase Zeta 1/maleylacetoacetate Isomerase Gene Influence the Toxicokinetics of Dichloroacetate”. The Journal of Clinical Pharmacology. Wiley. doi:10.1177/0091270011405664.

The Journal of Clinical PharmacologyJournal