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Results for “Very Long Chain Acyl Coa Dehydrogenase Deficiency”

26 trials

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Showing 20 of 26 results

Not applicableUnknownNCT02635269
What this trial is testing

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Who this might be right for
Metabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more
Rigshospitalet, Denmark 60
Testing effectiveness (Phase 2)Study completedNCT00983788
What this trial is testing

Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects

Who this might be right for
Carnitine Palmitoyltransferase II DeficiencyVery Long Chain Acyl Coa Dehydrogenase Deficiency
Rigshospitalet, Denmark 12
Not applicableStudy completedNCT00328159
What this trial is testing

Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids

Who this might be right for
Inborn Errors of Metabolism
Assistance Publique - Hôpitaux de Paris 20
Not applicableStudy completedNCT02517307
What this trial is testing

Fatty Acid Oxidation Defects and Insulin Sensitivity

Who this might be right for
Very Long-chain Acyl-CoA Dehydrogenase DeficiencyTrifunctional Protein DeficiencyLong-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency+3 more
Oregon Health and Science University 41
Not applicableUnknownNCT05910151
What this trial is testing

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Who this might be right for
Propionic/Methylmalonic AcidemiasMaple Syrup Urine DiseaseCitrullinemia+26 more
West Kazakhstan Medical University 2,250
Not applicableEnded earlyNCT04812106
What this trial is testing

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

Who this might be right for
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Ultragenyx Pharmaceutical Inc 8
Testing effectiveness (Phase 2)Study completedNCT01494051
What this trial is testing

High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders

Who this might be right for
Very Long-chain Acyl-CoA Dehydrogenase DeficiencyTrifunctional Protein DeficiencyCarnitine Palmitoyltransferase 2 Deficiency+1 more
Oregon Health and Science University 13
Testing effectiveness (Phase 2)Looking for participantsNCT06017869
What this trial is testing

Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)

Who this might be right for
Mitochondrial DiseasesPearson Syndrome
Minovia Therapeutics Ltd. 6
Not applicableLooking for participantsNCT05554835
What this trial is testing

Global Registry and Natural History Study for Mitochondrial Disorders

Who this might be right for
Mitochondrial DiseasesKearns-Sayre SyndromeMIDD+15 more
LMU Klinikum 6,000
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableUnknownNCT05029843
What this trial is testing

SLSMDS Natural History Study

Who this might be right for
Pearson SyndromeSingle Large Scale Mitochondrial DNA Deletion Syndromes (SLSMDS)
The Champ Foundation 30
Testing effectiveness (Phase 2)Study completedNCT02214160
What this trial is testing

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

Who this might be right for
Carnitine Palmitoyltransferase (CPT I or CPT II) DeficiencyVery Long Chain Acyl-CoA Dehydrogenase (VLCAD) DeficiencyLong-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency+2 more
Ultragenyx Pharmaceutical Inc 94
Not applicableStudy completedNCT02327364
What this trial is testing

Natural History of Pearson Syndrome

Who this might be right for
Pearson Syndrome
Sumit Parikh 11
Testing effectiveness (Phase 2)Ended earlyNCT02104336
What this trial is testing

Phase 2 Study of EPI-743 in Children With Pearson Syndrome

Who this might be right for
Pearson Syndrome
Edison Pharmaceuticals Inc 2
Not applicableStudy completedNCT03531554
What this trial is testing

Acute Nutritional Ketosis in VLCAD Deficiency

Who this might be right for
VLCAD DeficiencyFatty Acid Oxidation Defects
University Medical Center Groningen 5
Not applicableLooking for participantsNCT04602325
What this trial is testing

Systemic Biomarkers of Brain Injury From Hyperammonemia

Who this might be right for
Urea Cycle DisorderOrganic AcidemiaMaple Syrup Urine Disease+3 more
Children's National Research Institute 24
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableStudy completedNCT00499070
What this trial is testing

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Who this might be right for
Dyskeratosis CongenitaFanconi AnemiaMyelodysplastic Syndromes+2 more
University Hospital Freiburg 119
Large-scale testing (Phase 3)Active Not RecruitingNCT05933200
What this trial is testing

Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

Who this might be right for
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Ultragenyx Pharmaceutical Inc 69
Not applicableNo Longer AvailableNCT01461304
What this trial is testing

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

Who this might be right for
Very Long-chain acylCoA Dehydrogenase (VLCAD) DeficiencyCarnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)Mitochondrial Trifunctional Protein Deficiency+5 more
Jerry Vockley, MD, PhD
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