Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency
1
Pipeline Programs
2
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
0
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Ultragenyx PharmaceuticalNOVATO, CA
1 programtriheptanoinN/A1 trial
Active Trials
NCT01461304No Longer Available
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Oregon TherapeuticsTriheptanoin
Clinical Trials (2)
Total enrollment: 32 patients across 2 trials
Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Start: Sep 2011Est. completion: Apr 201532 patients
Phase 2Completed
Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
N/ANo Longer Available
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
2 companies competing in this space
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