Medium-chain Acyl-CoA Dehydrogenase Deficiency

3
Pipeline Programs
2
Companies
3
Clinical Trials
2 recruiting
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
3
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Ultragenyx Pharmaceutical
2 programs
2
TriheptanoinPhase 21 trial
TriheptanoinPhase 21 trial
Active Trials
NCT06067802Suspended8Est. Jul 2028
NCT07097311Recruiting24Est. Apr 2028
Zevra Therapeutics
Zevra TherapeuticsDenmark - Frederiksberg
1 program
1
Sodium phenylbutyratePhase 21 trial
Active Trials
NCT06773026RecruitingEst. Jul 2027

Trial Timeline

Clinical trial activity over time

2025
2026
2027
2028
Ultragenyx PharmaceuticalTriheptanoin
Ultragenyx PharmaceuticalTriheptanoin
Zevra TherapeuticsSodium phenylbutyrate

Clinical Trials (3)

Total enrollment: 32 patients across 3 trials

NCT07097311Ultragenyx PharmaceuticalTriheptanoin

Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Start: Jul 2026Est. completion: Apr 202824 patients
Phase 2Recruiting
NCT06067802Ultragenyx PharmaceuticalTriheptanoin

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Start: Feb 2026Est. completion: Jul 20288 patients
Phase 2Suspended
NCT06773026Zevra TherapeuticsSodium phenylbutyrate

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Start: Jun 2025Est. completion: Jul 2027
Phase 2Recruiting

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 actively recruiting trials targeting 32 patients
2 companies competing in this space