Search clinical trials for: Very Long Chain acyl-CoA Dehydrogenase Deficiency

Total 80644 results

Ultragenyx Pharmaceutical Inc

Recruiting

A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Turkey, United Kingdom, Spain, Saudi Arabia, Poland, Czechia, Germany
West Kazakhstan Medical University

Recruiting

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

Ornithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditions

Kazakhstan
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Ultragenyx Pharmaceutical Inc

Recruiting

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

United States, Canada
Children's National Research Institute
National Center for Advancing Translational Sciences (NCATS)

Recruiting

Systemic Biomarkers of Brain Injury From Hyperammonemia

Hypoxic-Ischemic Encephalopathy | Urea Cycle Disorder | Organic Acidemia | Fatty Acid Oxidation Disorder | Maple Syrup Urine Disease | Glutaric Acidemia I

United States
Ultragenyx Pharmaceutical Inc

Available

Expanded Access to Triheptanoin

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Oregon Health and Science University

Completed

Oral Ketones and Exercise Among Patients With Long-chain Fatty Acid Oxidation Disorders

Trifunctional Protein Deficiency | Very Long Chain Acyl Coa Dehydrogenase Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency | Carnitine Palmitoyltransferase Deficiency 2

United States
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
Oregon Health and Science University

Completed

Fatty Acid Oxidation Defects and Insulin Sensitivity

Normal Volunteers | Trifunctional Protein Deficiency | Very Long-chain Acyl-CoA Dehydrogenase Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency | Medium-chain Acyl-CoA Dehydrogenase Deficiency | Carnitine Palmitoyltransferase II Deficiency, Myopathic

United States
Ultragenyx Pharmaceutical Inc

Completed

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 | Trifunctional Protein (TFP) Deficiency | Carnitine-acylcarnitine Translocase (CACT) Deficiency

United States, United Kingdom
Ultragenyx Pharmaceutical Inc

Terminated

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

United States
Jerry Vockley, MD, PhD
Ultragenyx Pharmaceutical Inc

No longer available

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

Barth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditions

United States
Ultragenyx Pharmaceutical Inc

Completed

A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Trifunctional Protein (TFP) Deficiency | Carnitine Palmitoyltransferase (CPT II) Deficiency | Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency

United States, United Kingdom
Oregon Health and Science University

Completed

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

Trifunctional Protein Deficiency | Carnitine Palmitoyltransferase 2 Deficiency | Very Long-chain Acyl-CoA Dehydrogenase Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
Oregon Health and Science University
University of Pittsburgh

Completed

Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder (Triheptanoin)

Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase 2 (CPT2) Deficiency | Mitochondrial Trifunctional Protein (TFP) Deficiency | Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency

United States
Rigshospitalet, Denmark
Groupe Hospitalier Pitie-Salpetriere

Completed

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

Carnitine Palmitoyltransferase II Deficiency | Very Long Chain Acyl Coa Dehydrogenase Deficiency

Denmark
Assistance Publique - Hôpitaux de Paris

Completed

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

Inborn Errors of Metabolism

France
Rigshospitalet, Denmark

Unknown

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Carbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditions

Denmark
Oregon Health and Science University
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Oregon...

Terminated

Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy

Peripheral Neuropathy | Mitochondrial Trifunctional Protein Deficiency

United States
Jerry Vockley, MD, PhD
Ultragenyx Pharmaceutical Inc

Recruiting

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

Medium-chain Acyl-CoA Dehydrogenase Deficiency

United States
Jerry Vockley, MD, PhD
Acer Therapeutics Inc.

Recruiting

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

Medium-chain Acyl-CoA Dehydrogenase Deficiency

United States
Coordinación de Investigación en Salud, Mexico
Instituto Mexicano del Seguro Social

Recruiting

Effect of Supplementation With ω-3 Fatty Acids, Vitamin D and Calcium in Patients With Acute Lymphoblastic Leukemia.

Acute Lymphoblastic Leukemia | Vitamin d Deficiency

Mexico
University Hospital, Lille

Recruiting

Impact of a Strategy Based on Bacterial DNA Detection to Optimize Antibiotics in Immunocompromised Patients With Hospital-acquired Pneumonia Requiring Mechanical Ventilation (RESPIRE)

Pneumonia, Ventilator-Associated | Immunodeficiency | Pneumonia, Hospital-Acquired

France
First Affiliated Hospital, Sun Yat-Sen University

Recruiting

Safety and Efficacy of Aspirin in Stroke Patients With Glucose-6-phosphate Dehydrogenase Deficiency (SAST) (SAST)

Stroke | G6PD Deficiency

China
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
National Cancer Institute (NCI)

Active, not recruiting

Oxaliplatin, Leucovorin Calcium, and Fluorouracil With or Without Bevacizumab in Treating Patients Who Have Undergone Surgery for Stage II Colon Cancer

Lynch Syndrome | Colon Mucinous Adenocarcinoma | Colon Signet Ring Cell Adenocarcinoma | Stage IIA Colon Cancer AJCC v7 | Stage IIB Colon Cancer AJCC v7 | Stage IIC Colon Cancer AJCC v7

United States, Canada, Puerto Rico, Peru, South Africa
M.D. Anderson Cancer Center
National Cancer Institute (NCI); AstraZeneca

Active, not recruiting

Danvatirsen and Durvalumab in Treating Patients With Advanced and Refractory Pancreatic, Non-Small Cell Lung Cancer, and Mismatch Repair Deficient Colorectal Cancer

Stage IVA Lung Cancer AJCC v8 | Stage IVB Lung Cancer AJCC v8 | Refractory Pancreatic Carcinoma | Stage II Pancreatic Cancer AJCC v8 | Stage III Pancreatic Cancer AJCC v8 | Stage IV Pancreatic Cancer AJCC v8 | Lung Non-Small Cell Carcinoma | Stage III Lung Cancer AJCC v8 | Stage IV Lung Cancer AJCC v8 | Stage IIIA Lung Cancer AJCC... and other conditions

United States
Sanguine Biosciences

Completed

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Depression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditions

United States
Gilead Sciences

Terminated

Drug-Drug Interaction Study of Vesatolimod in Adults With HIV-1 Who Have Very Low or Undetectable Virus Levels

Human Immunodeficiency Virus Type 1 (HIV-1) Infection

United States
University of California, San Diego
The Hartwell Foundation

Completed

Omega-3 Long Chain Polyunsaturated Fatty Acid (LCPUFA) Supplementation in Very Low Birth Weight Infants for The Prevention Retinopathy of Prematurity

Retinopathy of Prematurity

United States
Oslo University Hospital

Active, not recruiting

Long-term Treatment With the Ketogenic Diet in Epilepsy

Epilepsy | GLUT1DS1 | PDH Deficiency

Norway
University of Maryland, Baltimore
Bill and Melinda Gates Foundation; Auro Vaccines LLC

Completed

A Safety and Immunogenicity Trial of IHV01 (FLSC-001)

HIV Infection

United States
Barcelona Institute for Global Health
Carlos III Health Institute

Completed

Walnuts, Long-Chain Polyunsaturated Fatty Acids and Adolescent Brain Development (WALNUTs)

Neurobehavioral Manifestations | Vitamin/Nutritional Deficiency

Spain
First Affiliated Hospital Xi'an Jiaotong University
Xi'an Gaoxin Hospital

Unknown

Effects of Fat-soluble Vitamins Supplementation on Common Complications and Neural Development in Very Low Birth Weight Infants

Sepsis | Anemia | Vitamin D Deficiency | Vitamin A Deficiency | Bronchopulmonary Dysplasia | Very Low Birth Weight Infants | Vitamin E Deficiency

China
University of Pittsburgh
Horizon Pharma Ireland, Ltd., Dublin Ireland

Completed

Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation

Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency

United States
Lv Yingjuan

Completed

Comparison of Vitamin D Status and Vitamin D Receptor Polymorphisms in Patients With High Myopia and Primary Open-angle Glaucoma

Vitamin D Deficiency | High Myopia | Primary Open-angle Glaucoma | Vitamin D Receptor Polymorphisms

China
University of Oslo
Oslo University Hospital; University Hospital, Akershus

Terminated

Nutrition, Growth and Development Among Very Preterm Infants (PRENU)

Low Birth Weight Infant

Norway
Maria Luz Couce Pico
Fundación Ramón Domínguez

Withdrawn

Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects

Long-chain Fatty Acid Transport Deficiency

Spain
National Cancer Institute (NCI)

Completed

Sunitinib Malate in Treating HIV-Positive Patients With Cancer Receiving Antiretroviral Therapy

HIV Infection | Clear Cell Renal Cell Carcinoma | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditions

United States
Academisch Medisch Centrum - Universiteit van Amsterdam...
The Stop ALD Foundation

Completed

The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) (BEZA)

Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy

Netherlands
University of Tennessee

Not yet recruiting

Enteral Zinc Supplementation in Very Low Birth Weight Infants

Nutritional Deficiency | Very Low Birth Weight Infant
Belgian Research on AIDS and HIV Consortium

Not yet recruiting

Use of Long-Acting Injectable Cabotegravir/Rilpivirine for the Treatment of HIV in Belgium

Human Immunodeficiency Virus
King's College Hospital NHS Trust
Chiesi Limited

Not yet recruiting

Less Invasive Surfactant Administration in Late Preterm or Early Term Born Infants

Respiratory Distress Syndrome | Surfactant Deficiency Syndrome Neonatal | Preterm Pregnancy
Chiang Mai University

Recruiting

Study Compares Pneumothorax Recurrence: Absorbable Mesh vs. Pleurectomy in Primary Spontaneous Pneumothorax.

Primary Spontaneous Pneumothorax

Thailand
University Medical Center, Kazakhstan
Nazarbayev University; Astana Medical University; Ministry of Science and Higher... and other collaborators

Recruiting

Effectiveness of Weight Loss Methods on Lifespan for Metabolic Syndrome

Metabolic Syndrome

Kazakhstan
Wave Life Sciences Ltd.

Not yet recruiting

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD (RestorAATion-2)

Alpha-1 Antitrypsin Deficiency
University of Zurich
Swiss HIV Cohort Study

Recruiting

Cabotegravir Plus Rilpivirine Long-acting Regimen in the Swiss HIV Cohort Study:Uptake, Outcome, and Risk Factors for Treatment Failures

HIV Infections

Switzerland
University of Copenhagen

Not yet recruiting

The Effect of Medium-chain Fatty Acids on Cardiac Function (MediHeart)

Heart Failure | Nutrition, Healthy

Denmark
Beam Therapeutics Inc.

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

Alpha 1-Antitrypsin Deficiency

United Kingdom
University of California, Los Angeles
University of North Carolina, Chapel Hill; University of California, Irvine; Florida... and other collaborators

Recruiting

Exploring, Predicting, and Intervening on Long-term Viral Suppression Electronically (EPI-LoVE)

HIV Infections

United States

Search clinical trials for: Very Long Chain acyl-CoA Dehydrogenase Deficiency

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