Phase 1 N=4 Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Primary: Metabolic Stress
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
N/A N=12 Effect of Medium Chain Fatty Acids on Cognitive Function During Acute Hypoglycemia in Patients With Type 1 Diabetes
Diabetes Mellitus, Type 1
Primary: Immediate Verbal Memory — 15.85; 17.36; 14.97; 13.28 units on a scale
Phase 2 N=32 Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency · Carnitine Palmitoyltransferase 2 (CPT2) Deficiency · Mitochondrial Trifunctional Protein (TFP) Deficiency
Primary: Energy Expenditure — -73; 107 kcal/day
N/A N=41 Fatty Acid Oxidation Defects and Insulin Sensitivity
Very Long-chain Acyl-CoA Dehydrogenase Deficiency · Trifunctional Protein Deficiency · Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
Primary: Glucose Disposal Rate (Rd)- the Rate of Glucose Infusion to Maintain Euglycemia During Steady State Insulin Infusion in mg/Min — 709; 429; 842; 497 mg/min — p=0.136
Phase 2 N=13 High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
Very Long-chain Acyl-CoA Dehydrogenase Deficiency · Trifunctional Protein Deficiency · Carnitine Palmitoyltransferase 2 Deficiency
Primary: Energy Expenditure — 2045; 2343 kcal
N/A N=10 Effect of MCT Emulsification on Ketogenesis in Human Adults
Healthy
Primary: Plasma Ketone Concentration — 136.97; 177.43; 270.61; 249.3 µmol/L
N/A N=20 Medium Chain Triglycerides and Aerobic Exercise on Ketone Production in Women With or Without Prediabetes
Prediabetic State
Primary: Plasma Ketone Concentrations — -52; 12; 493; 627 (µmol/h)/L
Phase 1 N=14 Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
N/A N=98 Dietary Ketosis: Fatty Acids Activate AMPK Energy Circuits Modulating Global Methylation
Mild Cognitive Impairment · Metabolic Syndrome
Primary: MoCA (Montreal Cognitive Assessment) — 28.38; 20.22 score on a scale — p=<0.0001
Phase 2 N=94 Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency · Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency · Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
Primary: Annualized LC-FAOD Major Clinical Events (MCEs) Rate: 18 Months Pre- and Entire UX007 Period Comparison for UX007-CL201-Rollover Cohort — 1.76; 1.00 events/year — p=0.0347
Phase 3 N=24 Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
Mitochondrial Diseases
Primary: McMaster Gross Motor Function (GMFM 88) — -0.002; -0.12 units on a scale — p=0.95
Phase 2 N=11 Fat Malabsorption in Chronic Pancreatitis
Chronic Pancreatitis
Primary: Malabsorption Blood Test: Difference in Mean HA AUC Between Groups: Subjects With CP Compared to Healthy Subjects — 8.3; 17.7 mg*h/dL — p=<0.001
Phase 1 N=12 Effect of Medium Chain Triglyceride Intake on Colonization of Preterm Infants With Candida
Candida Infection · Premature Infant
Primary: Stool Fungal Burden — 500; 55000; 1671; 622.5 cfu per gram — p=0.9
Phase 2 N=38 CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Spinal Muscular Atrophy Type I
Primary: Laboratory Safety Data
N/A N=15 High-fat Meal Challenge in Pediatrics
Pediatric Obesity · Insulin Resistance
Primary: Palatability of the High Fat Agent. — 13 Participants — p=0.15
Phase 3 N=41 A Double-blind, Randomized, Multicenter, Cross-over Study to Compare the Effect of Creon N and Creon® on Fat Digestion in Subjects ≥ 12 Years of Age With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Primary: Coefficient of Fat Absorption (CFA) — 88.1; 89.5 percentage of fat intake
Phase 3 N=8 The Impact of Free Fatty Acid (FFA-) Suppression on Myocardial Lipids and Function in Patients With Type 2 Diabetes
Type 2 Diabetes
Primary: MYCL — 0.54; 0.44; 0.35; 0.36 percentage of water signal
Phase 1 N=16 Acute Effects of Medium Chain Triglyceride (MCT) Nutritional Ketosis on Parkinson's Disease (PD) Symptoms and Biomarkers (MCT-PD)
Parkinson's Disease
Primary: Feasibiilty of Ketogenic Diet - Retention (Co-primary Endpoint) — 15 Participants
Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
N/A N=26 Fatty Acid Oxidation Disorders & Body Weight Regulation Grant
Trifunctional Protein Deficiency
Primary: An Outcome of This Study is the Difference in Percent Body Fat (%BF) Between Subjects With a Long-chain Fatty Acid Oxidation Disorder and Normal Controls. — 31.8; 27.2…
Phase 2 N=29 A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) · Carnitine Palmitoyltransferase (CPT II) Deficiency · Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
Primary: Change From Baseline in Time Adjusted-Area Under the Curve (AUC/Time) for Workload During Cycle Ergometry at Week 24 — 423.594 watts — p=0.1518
N/A N=10 Comparing the Ketogenic Effect of Coconut Oil and Different MCTs
Healthy Adults
Primary: Plasma Ketone Concentrations — 69; 294; 109; 363 µmol/L
N/A N=15 Ketone Supplementation in Cystic Fibrosis
Cystic Fibrosis
Primary: Pulmonary Function Testing — 1.9; 2.0 L/sec
N/A N=6 Testing Glial Pathways to HAAF in Human Subjects Using Carbon 13 Magnetic Resonance Spectroscopy
Diabetes Complications · Hypoglycemia
Primary: Percent Enrichment of Bicarbonate as Measured by Magnetic Resonance Spectroscopy (MRS) — 61.9 % enrichment — p=0.0053
Phase 2 N=72 Fatty Acid Supplementation in Children With ASD
Autism Spectrum Disorder
Primary: Bioavailability — 1.95; 1.80; 3.00; 0.80 mol%
Phase 2 N=20 An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Primary: Reported Change in Seizures Frequency From Baseline at 13 Weeks — 4.4; 189.5 seizures/two weeks
Phase 3 N=5 GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis
Bile Acid Synthesis Defect · Inborn Error of Bile Acid Metabolism · Inborn Error of Bile Acid Conjugation
Primary: Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation. — 5 Participants
N/A N=46 Neurologic Injuries in Adults With Urea Cycle Disorders
Brain Diseases, Metabolic, Inborn · Urea Cycle Disorder · Ornithine Transcarbamylase Deficiency
Primary: Concentration of Glutamine and Myoinositol by MRS — 4.97; 3.66; 3.78; 4.50 mM — p=0.003
Phase 2 N=10 Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
GLUT1DS1
Primary: Change in Ketosis (Beta-hydroxybutyrate Levels) — 3.82 mM
Early Phase 1 N=10 Study of Modified Atkins Diet in Kabuki Syndrome
Kabuki Syndrome
Primary: Benton Judgement of Line Orientation — 8.6; 11.6 score on a scale