Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

0.0%

0 terminated/withdrawn out of 8 trials

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 0 completed trials have results

Key Signals

2 recruiting

Enrollment Performance

Analytics

Phase 2
4(66.7%)
Phase 1
1(16.7%)
N/A
1(16.7%)
6Total
Phase 2(4)
Phase 1(1)
N/A(1)

Activity Timeline

Global Presence

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Clinical Trials (8)

Showing 8 of 8 trials
NCT06067802Phase 2Suspended

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

Role: lead

NCT07097311Phase 2Recruiting

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

Role: lead

NCT04848090Not ApplicableEnrolling By Invitation

Genome Sequencing in the Intensive Care Unit Population

Role: lead

NCT06069375Phase 2Suspended

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

Role: lead

NCT07125066Phase 1Enrolling By Invitation

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

Role: lead

NCT06773026Phase 2Recruiting

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

Role: lead

NCT01461304Unknown

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Role: lead

NCT02968953Unknown

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

Role: lead

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