Clinical trials: Fatty Acid Oxidation
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
Fatty Acid Oxidation Disorders
A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders
The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.
- Trial name
- NCT03833128
- Testing
- Low Dose REN001, High Dose REN001
- Where
- 7 sites in United States, France and 1 more
- Volunteers
- 24 people
- Sponsor
- Reneo Pharma Ltd
Fatty Acid Oxidation Disorder
A Prospective Study of the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders
The purpose of the study is to collect information on disease characteristics from adult patients diagnosed with fatty acid oxidation disorders (FAOD).
- Trial name
- FORWARD (NCT04482049)
- Where
- 15 sites in United States, Austria and 9 more
- Volunteers
- 61 people
- Sponsor
- Reneo Pharma Ltd
Ketosis, Epilepsy
Market Research - Acceptability Study for New MCT Fat Products
This is a market research, observational study to evaluate the tolerability and acceptability of MCT supplements for young children, young people and adults with intractable epilepsy, GLUT-1 or PDHD from 3 years to adulthood.
- Conditions
- Ketosis, Epilepsy, Fatty Acid Oxidation Disorder, Malabsorption
- Trial name
- NCT04309214
- Testing
- MCT fats
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Dr. Schär AG / SPA
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
The primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program.
- Trial name
- NCT03768817
- Testing
- No Intervention
- Where
- 15 sites in United States
- Volunteers
- 20 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Fatty Acid Oxidation Disorder
Experience of Using Lipano in Children With Fatty Acid Oxidation Disorders
This is a market research, observational study to evaluate the tolerability and acceptability of an MCT based formula for infants and young people with long chain fatty acid oxidation disorder from birth to 18 months.
- Trial name
- NCT04400422
- Testing
- Lipano formula
- Where
- Russia
- Volunteers
- 5 people
- Sponsor
- Dr. Schär AG / SPA
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
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
Obesity
A Study to Test Whether Survodutide Improves How the Body Uses Energy and Breaks Down Fat in People With Obesity
This study is open to adults between 18 and 65 years of age who have obesity. People can join the study if they have a body mass index (BMI) between 30 and 45 km/m².
- Trial name
- NCT06745284
- Testing
- Survodutide, Semaglutide
- Where
- 2 sites in United States
- Volunteers
- 64 people
- Sponsor
- Boehringer Ingelheim
Urea Cycle Disorders, Organic Acidemias
At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism
The goal of this observational study is to learn if people with certain ammonia metabolism disorders will measure their ammonia levels at home.
- Conditions
- Urea Cycle Disorders, Organic Acidemias, Fatty Acid Oxidation Disorder, Ammonia; Metabolic Disorder
- Trial name
- NCT06953505
- Testing
- Ammonia Study Device
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Sequitur Health Corp.
Diabetes
A Comparison of Measurements of Ketone Body Flux Versus Ketone Body Concentration as a Pharmacodynamic Marker of Hepatic Fatty Acid Oxidation (0000-110)
This study will compare two approaches to the measurement of fatty acid oxidation (ketone body flux and ketone body concentration) and determine which method demonstrates superior sensitivity and variability in the detection of…
- Trial name
- NCT00860392
- Testing
- Stable isotope tracer (13-C(4)-BHB)
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Ketosis, Epilepsy
Market Research - Acceptability Study for a Range of MCT Products
This is a market research, observational study to evaluate the tolerability and acceptability of MCT supplements for young children and young people with intractable epilepsy, GLUT-1 or PDHD from 3 years to adulthood.
- Conditions
- Ketosis, Epilepsy, Ketogenic Diet, Fatty Acid Oxidation Disorder
- Trial name
- NCT04309149
- Testing
- MCT fats
- Where
- 2 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Dr. Schär AG / SPA
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
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
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