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Pyruvate Dehydrogenase Complex Deficiency

0
Pipeline Programs
4
Companies
4
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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0
0
0
0
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Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Saol Therapeutics
Saol TherapeuticsGA - Roswell
2 programs
DichloroacetateN/A1 trial
DichloroacetatePHASE_3
Active Trials
NCT06931262Available
Freya Biosciences
Freya BiosciencesDenmark - Copenhagen
1 program
Natural History Study of Pyruvate Dehydrogenase DeficiencyN/A1 trial
Active Trials
NCT05257005Unknown50Est. Aug 2024
Ultragenyx Pharmaceutical
1 program
TriheptanoinPHASE_11 trial
Active Trials
NCT06340685Recruiting6Est. Jun 2029
Medosome Biotec
Medosome BiotecFL - Alachua
1 program
DichloroacetatePHASE_31 trial
Active Trials
NCT02616484Active Not Recruiting34Est. Aug 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Medosome BiotecDichloroacetate
Ultragenyx PharmaceuticalTriheptanoin
Freya BiosciencesNatural History Study of Pyruvate Dehydrogenase Deficiency

Clinical Trials (4)

Total enrollment: 90 patients across 4 trials

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

Start: Jul 2020Est. completion: Aug 202534 patients
Phase 3Active Not Recruiting

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Start: Jul 2024Est. completion: Jun 20296 patients
Phase 1Recruiting

Expanded Access Treatment Protocol With DCA for Patients With PDCD

N/AAvailable
NCT05257005Freya BiosciencesNatural History Study of Pyruvate Dehydrogenase Deficiency

Natural History Study of Pyruvate Dehydrogenase Deficiency

Start: Nov 2020Est. completion: Aug 202450 patients
N/AUnknown

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 actively recruiting trials targeting 90 patients
4 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.