449 studies found for:    "lipid metabolism, inborn errors" OR "farber lipogranulomatosis"
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Rank Status Study
21 Not yet recruiting Phase 2 Study of EPI-743 in Children With Pearson Syndrome
Condition: Pearson Syndrome
Intervention: Drug: EPI-743
22 Completed A 12-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05244)
Conditions: Hypercholesterolemia;   Familial Hypercholesterolemia;   Homozygous Sitosterolemia
Interventions: Drug: Ezetimibe;   Drug: Ezetimibe + other lipid-lowering medication(s)
23 Completed Cholesterol Metabolism in Heterozygous Phytosterolemia
Condition: Cardiovascular Disease
Intervention: Dietary Supplement: phytosterol ester
24 Not yet recruiting Effects of Colesevelam
Condition: Sitosterolemia
Interventions: Drug: Colesevelam;   Other: Placebo
25 Completed High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
Conditions: Very Long-chain Acyl-CoA Dehydrogenase Deficiency;   Trifunctional Protein Deficiency;   Carnitine Palmitoyltransferase 2 Deficiency;   Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
Intervention: Behavioral: Diet counseling
26 Unknown  Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
Conditions: Dyskeratosis Congenita;   Fanconi Anemia;   Myelodysplastic Syndromes;   Nonmalignant Neoplasm;   Pearson Marrow-pancreas Syndrome;   Shwachman-diamond Syndrome
Interventions: Genetic: polymerase chain reaction;   Other: flow cytometry;   Other: immunologic technique;   Procedure: biopsy
27 Recruiting Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
Condition: Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Intervention: Drug: Ravicti
28 Completed Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects
Conditions: Carnitine Palmitoyltransferase II Deficiency;   Very Long Chain Acyl Coa Dehydrogenase Deficiency
Interventions: Drug: Bezafibrate;   Other: Placebo
29 Completed Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia
Conditions: Heart Diseases;   Metabolism, Inborn Errors
Interventions: Drug: SCH-58235;   Drug: Ezetimibe
30 Unknown  Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease;   Fucosidosis;   Globoid Cell Leukodystrophy;   Adrenoleukodystrophy;   Mannosidosis;   Niemann-Pick Disease;   Pulmonary Complications;   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Metachromatic Leukodystrophy;   Gaucher's Disease;   Wolman Disease
Intervention:
31 Completed A Study of AT1001 in Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: AT1001 (migalastat hydrochloride)
32 Completed Lipid Efficacy and Safety in Patients With Mixed Hyperlipidemia (MK-0524B-024)
Condition: Mixed Hyperlipidemia
Interventions: Drug: Comparator: MK0524B;   Drug: Comparator: Atorvastatin
33 Active, not recruiting Study of Alirocumab (REGN727/ SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)
Condition: Heterozygous Familial Hypercholesterolemia
Interventions: Drug: LMT (atorvastatin, simvastatin, or rosuvastatin);   Drug: alirocumab (REGN727/ SAR236553);   Drug: placebo
34 Completed Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Condition: Fabry Disease
Interventions: Drug: migalastat hydrochloride;   Drug: Placebo
35 Suspended Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies
Condition: Hyperlipoproteinemia Type I
Intervention:
36 Enrolling by invitation An Extension of a Phase 1/2, Open Label, Dose Ranging Study of PRX-102 in Adult Fabry Patients
Condition: Fabry Disease
Intervention: Drug: PRX-102
37 Recruiting Efficacy and Safety of Fenofibrate Added on to Atorvastatin Compared With Atorvastatin in Mixed Hypercholesterolemic Patient(CKD-337)
Condition: Mixed Hyperlipidemia
Interventions: Drug: Atorvastatin 20mg;   Drug: Fenofibrate 160mg;   Other: Placebo (Fenofibrate 160 mg)
38 Recruiting Study of Inborn Errors of Cholesterol Synthesis and Related Disorders
Condition: Lipoidosis
Intervention:
39 Completed
Has Results
Effects of Ezetimibe With Simvastatin in the Therapy of Adolescents With HeFH (Study P02579)
Condition: Hypercholesterolemia
Interventions: Drug: ezetimibe with simvastatin;   Drug: simvastatin
40 Completed 48-wk Open Label Phase IIIb to Evaluate Efficacy and Safety
Condition: Dyslipidaemia
Interventions: Drug: Rosuvastatin;   Behavioral: Maintenance of specific diet

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Indicates status has not been verified in more than two years