Clinical trials: Fatty Acid Deficiency
Long Chain Fatty Acid Oxidation Disorders
Expanded Access to Triheptanoin
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
- Trial name
- NCT03773770
- Testing
- Triheptanoin
- Sponsor
- Ultragenyx Pharmaceutical Inc
Essential Fatty Acid Deficiency (EFAD)
Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion (Part A)
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care (Intralipid) from 7 up to 90 days to develop Essential Fatty Acid…
- Trial name
- NCT04555044
- Testing
- Clinolipid, Intralipid
- Where
- 9 sites in United States
- Volunteers
- 101 people
- Sponsor
- Baxter Healthcare Corporation
Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency
Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population
- Conditions
- Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency, Malnutrition, Pediatric ALL
- Trial name
- NCT06274788
- Testing
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Where
- 10 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fresenius Kabi
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
- Trial name
- NCT05933200
- Testing
- Triheptanoin, MCT Oil
- Where
- 15 sites in Czechia, Germany and 5 more
- Volunteers
- 69 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in…
- Conditions
- 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, Trifunctional Protein (TFP) Deficiency
- Trial name
- NCT02214160
- Testing
- UX007
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 94 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
The primary objective of the study was to evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD following 24 weeks of treatment.
- Conditions
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- NCT01886378
- Testing
- UX007
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 29 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Non Alcoholic Fatty Liver
Evaluation of MF4637 for Correcting the Omega-3 Nutritional Deficiency in NAFLD Patients
This study is a prospective, randomized, placebo-controlled, double-blind trial to determine the effect of high concentrate omega-3 capsules on the omega-3 status of patients with non-alcoholic fatty liver.
- Trial name
- NCT02923804
- Testing
- Omega-3, Olive oil
- Where
- 7 sites in United States
- Volunteers
- 172 people
- Sponsor
- Pronova BioPharma
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.
- Trial name
- NCT04632953
- Testing
- No Intervention
- Where
- 16 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency
Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
This study will demonstrate Short-, mid-, and long-term safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this…
- Conditions
- Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency, Neurocognitive Deficit, Malnutrition
- Trial name
- NCT04807478
- Testing
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Sponsor
- Fresenius Kabi
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
The objectives of this study are to characterize the disease impact, the disease management and resource utilization, and provide benefits to the LC-FAOD community, by use of a convenient online platform for participants (or…
- Trial name
- NCT04812106
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Malnutrition, Child, Malnutrition
Safety Study of SMOFlipid to Evaluate the Risk of Developing EFAD and/or PNAC in Pediatric and Adult Patients
Evaluate the risk of developing EFAD and/or PNAC in adult and pediatric patients 1 month of age and older, who are anticipated to need 8 weeks or longer of parenteral nutrition treatment with SMOFlipid.
- Conditions
- Malnutrition, Child, Malnutrition, Essential Fatty Acid Deficiency (EFAD), Parenteral Nutrition Associated Cholestasis
- Trial name
- NCT06049680
- Testing
- SMOFlipid® (lipid injectable emulsion)
- Where
- 6 sites in United States
- Volunteers
- 100 people
- Sponsor
- Fresenius Kabi
Lysosomal Acid Lipase Deficiency
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
- Trial name
- NCT02193867
- Testing
- Sebelipase Alfa
- Where
- 5 sites in United States, Finland and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Essential Fatty Acid Deficiency (EFAD)
Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care for up to 90 days to develop Essential Fatty Acid Deficiency (EFAD).
- Trial name
- NCT02476994
- Testing
- Clinolipid, Intralipid
- Where
- 4 sites in United States
- Volunteers
- 1 people
- Sponsor
- Baxter Healthcare Corporation
Lysosomal Acid Lipase Deficiency
A Study to Identify and Characterize LAL-D Patients in High-risk Populations
The objective of this study is to determine the frequency of Lysosomal Acid Lipase Deficiency (LAL D) by lysosomal acid lipase (LAL) enzyme activity assay in patients who are considered to be at risk.
- Trial name
- NCT02345421
- Where
- 15 sites in United States
- Volunteers
- 640 people
- Sponsor
- Alexion Pharmaceuticals, Inc.