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ClinicalTrials.gov public records Last synced Oct 5, 2026, 9:22 PM EDT

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Showing 1–8 of 8 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Jerry Vockley, MD, PhD Clear all
Enrolling by invitation Phase 1 Interventional

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

NCT07125066
View directory record Official record
Conditions
Combined D,L-2-hydroxyglutaric Aciduria
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Oct 5, 2026, 9:22 PM EDT
Withdrawn Phase 2 Interventional

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

NCT07097311
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 5, 2026, 9:22 PM EDT
Withdrawn Phase 2 Interventional

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

NCT06067802
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
16 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 5, 2026, 9:22 PM EDT
Recruiting Phase 2 Interventional

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

NCT06773026
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
4 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Oct 5, 2026, 9:22 PM EDT
Enrolling by invitation Not applicable Interventional

Genome Sequencing in the Intensive Care Unit Population

NCT04848090
View directory record Official record
Conditions
Infant, Newborn, Disease, Genetic Disease
Interventions
Neonate WGS Testing
Diagnostic Test
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
Up to 1 Year
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 5, 2026, 9:22 PM EDT
No Longer Available No phase listed Expanded access

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

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 5, 2026, 9:22 PM EDT
Suspended Phase 2 Interventional

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

NCT06069375
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
10 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 5, 2026, 9:22 PM EDT
No Longer Available No phase listed Expanded access

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

NCT02968953
View directory record Official record
Conditions
Glucose Transporter 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
3 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 8, 2019 · Synced Oct 5, 2026, 9:22 PM EDT