# Phase 3 Trial of DCA in PDC Deficiency IND 028,625 (02/04/2015)

> **NIH FDA R01** · UNIVERSITY OF FLORIDA · 2022 · $496,401

## Abstract

Abstract
 Congenital pyruvate dehydrogenase complex (PDC) deficiency (PDCD) is a life-threatening
mitochondrial disease of metabolic integration in which the efficient conversion of carbohydrate fuel into
energy is perturbed. There are no proven therapies for PDCD. However, dichloroacetate (DCA) represents
targeted potential therapy for this disease by stimulating residual PDC activity, decreasing hyperlactatemia
and improving mitochondrial energetics. This background and our preliminary data suggest that DCA may
benefit the clinical status of PDCD children. We have worked closely with the FDA and a parent-driven
organization to develop an appropriate study design and identify prospective subjects. We now propose to
undertake a 4 year phase 3 clinical trial to test the hypotheses that oral DCA will improve at-home
functionality of affected children compared to placebo and will be well-tolerated and safe for chronic use.
Our primary endpoint will be based on an improved clinical status, as determined by a novel, Observer
Reported Outcome (ObsRO) measure. Our secondary endpoints will include 1) confirmation of better
clinical outcome by the Karnofsky/Lansky performance scale of home functionality in children with life-
threatening disease; and 2) decreased lactatemia.

## Key facts

- **NIH application ID:** 10436136
- **Project number:** 5R01FD005407-04
- **Recipient organization:** UNIVERSITY OF FLORIDA
- **Principal Investigator:** Peter Wallace Stacpoole
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2022
- **Award amount:** $496,401
- **Award type:** 5
- **Project period:** 2016-06-01 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10436136

## Citation

> US National Institutes of Health, RePORTER application 10436136, Phase 3 Trial of DCA in PDC Deficiency IND 028,625 (02/04/2015) (5R01FD005407-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10436136. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*