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NCT02616484 · ClinicalTrials.gov registry record · Phase 3
Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
A Phase 3 study of Pyruvate Dehydrogenase Complex Deficiency, sponsored by Saol Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 34
- Enrollment target
- 10
- Study locations
NCT02616484: Active Phase 3 study of Pyruvate Dehydrogenase Complex Deficiency, sponsored by Saol Therapeutics.
NCT02616484 is a Phase 3 study of Pyruvate Dehydrogenase Complex Deficiency that is active but no longer recruiting, run by Saol Therapeutics. The registered enrollment target is 34 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower). The trial reports 10 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02616484, a Phase 3 study of Pyruvate Dehydrogenase Complex Deficiency, is active but no longer recruiting, sponsored by Saol Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 34 participants
- Enrollment target
- 10
- Study locations
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
Primary Outcome
The efficacy of dichloroacetate will be determined by applying a novel Observer Reported Outcome (ObsRO) measure of health. The ObsRO scores will be graded on a Likert Scale from 0-4. (0 being absent and 4 being extremely severe).
Conditions Studied
Interventions
- OTHER Placebo
- DRUG Dichloroacetate (DCA)
- GENETIC Genotype
Study Locations (10)
California
- Children's Hospital of Orange County - Orange
- Stanford University - Stanford
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
- Children's Hospital of Pittsburgh - Pittsburgh
District of Columbia
- Children's National Medical Center - Washington D.C.
Florida
- University of Florida - Gainesville
Ohio
- University Hospitals Cleveland Medical Center - Cleveland
Texas
- Baylor College of Medicine - Houston
Utah
- University of Utah - Salt Lake City
Washington
- Seattle Children's Hospital - Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 34 participants |
| Start Date | 2020-07-14 |
| Est. Completion | 2025-08-27 |
| Phase | Phase 3 |
What the registry record for NCT02616484 still lists
NCT02616484 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 34 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower).
The record links to 1 condition, with Pyruvate Dehydrogenase Complex Deficiency appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.
NCT02616484 lists 10 locations in 8 states (California, Pennsylvania, District of Columbia).
Frequently Asked Questions
What is clinical trial NCT02616484 about?
NCT02616484 is a clinical study titled "Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:". 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 ...
What is the current status of trial NCT02616484?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 34 participants. The study started on 2020-07-14. Estimated completion is 2025-08-27.
What conditions does trial NCT02616484 study?
This clinical trial studies the following conditions: Pyruvate Dehydrogenase Complex Deficiency.
What interventions are being tested in trial NCT02616484?
The interventions under investigation include: Placebo (OTHER), Dichloroacetate (DCA) (DRUG), Genotype (GENETIC).
Who is sponsoring clinical trial NCT02616484?
This trial is sponsored by Saol Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02616484 being conducted?
This trial has 10 study locations across California, District of Columbia, Florida, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT02616484's enrollment target sits among peer trials
34 1905th of 2000 higher than 94 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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