Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Public ClinicalTrials.gov record NCT02616484. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT02616484
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 34 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Months to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 13, 2020
- Primary completion
- Aug 26, 2025
- Completion
- Aug 26, 2025
- Last update posted
- May 3, 2025
2020 – 2025
United States locations
- U.S. sites
- 10
- U.S. states
- 8
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital of Orange County | Orange | California | 92868 | — |
| Stanford University | Stanford | California | 94305 | — |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | — |
| University of Florida | Gainesville | Florida | 32611 | — |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 15224 | — |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| University of Utah | Salt Lake City | Utah | 84113 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02616484, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 3, 2025 · Synced Sep 1, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02616484 live on ClinicalTrials.gov.