Combined D,L-2-hydroxyglutaric Aciduria

1
Pipeline Programs
1
Companies
1
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
1
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Zevra Therapeutics
Zevra TherapeuticsDenmark - Frederiksberg
1 program
1
Sodium phenylbutyratePhase 11 trial
Active Trials
NCT07125066Enrolling By Invitation1Est. Dec 2027

Trial Timeline

Clinical trial activity over time

2025
2026
2027
Zevra TherapeuticsSodium phenylbutyrate

Clinical Trials (1)

Total enrollment: 1 patients across 1 trials

NCT07125066Zevra TherapeuticsSodium phenylbutyrate

An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)

Start: Jul 2025Est. completion: Dec 20271 patients
Phase 1Enrolling By Invitation

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

1 companies competing in this space