Clinical Trials on Cholesterol Ester Storage Disease (CESD) in Italy

Total 303 results

Alexion Pharmaceuticals

Completed

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

Lysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)

United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
Alexion

Recruiting

Lysosomal Acid Lipase (LAL) Deficiency Registry (ALX-LALD-501)

Wolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-Deficiency

France, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
Fondazione SISA (Societa Italiana per lo Studio...

Recruiting

Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)

Lysosomal Acid Lipase Deficiency

Italy
Alexion Pharmaceuticals

Completed

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency

Spain, Germany, Italy, United States, Croatia, Canada, Russian Federation, Denmark, United Kingdom, Belgium, Mexico, Australia, Netherlands, Brazil, Turkey
University Of Perugia

Recruiting

Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity

Obesity | Ketosis | Lysosomal Acid Lipase Deficiency | Ketogenic Dieting

Italy
Alexion

Completed

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

Wolman Disease | Lysosomal Acid Lipase Deficiency

United States, Canada, United Kingdom, Italy, France
Alexion Pharmaceuticals

Completed

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency (ARISE)

Lysosomal Acid Lipase Deficiency

France, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
University of Roma La Sapienza
Alexion Pharmaceuticals

Recruiting

Lysosomal Acid Lipase Activity in Nonalcoholic Fatty Liver Disease (NAFLD LAL)

Non-Alcoholic Fatty Liver Disease

Italy
Alexion Pharmaceuticals

Terminated

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency

United Kingdom, Finland, United States, Italy
Azienda Ospedaliera Brotzu
University of Cagliari

Completed

Cheese Intake,CLA and Hypercholesterolemia (CASU)

Modifications of LDL Cholesterol | Modifications of Enocannabinoid Levels

Italy
University of Padova

Unknown

Effects of Parmigiano Reggiano on Muscle and Inflammatory Response to Eccentric Resistance Training in Older Adults

Inflammation | Aging | Atrophy, Muscular | Cholesterol Deposition

Italy
Sanofi

Completed

A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting (HomERT)

Pompe Disease | Mucopolysaccharidosis Type I (MPS I)

Italy
University of Lausanne
University of Modena and Reggio Emilia

Completed

Sialic Acid Supplementation in N-Acetylneuraminic Acid Synthase (NANS) Deficiency

N-Acetylneuraminic Acid Storage Disease

Switzerland, Italy
Fondazione Don Carlo Gnocchi Onlus
Associazione Italiana Glicogenosi (AIG); Associazione Riabilitatori Insufficienza... and other collaborators

Recruiting

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease (LOPD01)

Glycogen Storage Disease Type II | Pompe Disease (Late-onset)

Italy
IRCCS Eugenio Medea
University of Pisa; University of Messina

Completed

McArdle Disease Treatment by Ketogenic Diet

Glycogen Storage Disease

Italy
LMU Klinikum
Genzyme, a Sanofi Company

Completed

Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study) (STIG)

Pompe Disease

Germany, Taiwan, Italy
Orchard Therapeutics
Ospedale San Raffaele

Active, not recruiting

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Lysosomal Storage Disease | Metachromatic Leukodystrophy

Italy
Orchard Therapeutics
Ospedale San Raffaele

Active, not recruiting

Gene Therapy for Metachromatic Leukodystrophy (MLD)

Lysosomal Storage Disease | Metachromatic Leukodystrophy

Italy
Amicus Therapeutics

Available

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Pompe Disease Infantile-Onset

Taiwan, United States, Italy
University of Siena
Careggi Hospital

Completed

Evaluation of Antioxidant Activity of Oral Lutein in Preterm and Term Newborn

Infant, Newborn, Diseases | Other Lipid Storage Disorders

Italy
Amicus Therapeutics

Recruiting

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (ROSSELLA)

Glycogen Storage Disease Type II Infantile Onset

United States, Taiwan, France, Germany, Italy, United Kingdom
Orchard Therapeutics
Ospedale San Raffaele

Active, not recruiting

OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

Lysosomal Storage Diseases | Metachromatic Leukodystrophy

Italy
University of Roma La Sapienza

Completed

Matrilysin Expression in Different Stages of Colorectal Tumors (MMP7)

Colorectal Cancer Stage II | Colorectal Cancer Stage III | Colorectal Cancer Stage IV | Colorectal Cancer Stage 0 | Colorectal Cancer Stage I

Italy
Ultragenyx Pharmaceutical Inc

Terminated

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Glycogen Storage Disease Type III

United States, Spain, Italy
Translational Research Center for Medical Innovation...

Terminated

Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M

Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) | Neutral Lipid Storage Disease With Myopathy (NLSD-M)

Italy, Japan
Ultragenyx Pharmaceutical Inc

Active, not recruiting

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Glycogen Storage Disease Type IA

United States, Brazil, Canada, Denmark, Germany, Italy, Netherlands, Spain
Azienda Ospedaliera di Padova

Recruiting

Selection Protocol for Liver Transplantation in Patients Aged Over 70 Years (Over70-LT)

Liver Transplantation | Old Age | End Stage Liver DIsease

Italy
Spark Therapeutics

Completed

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Lysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Italy, United Kingdom, Netherlands, France, Germany
Astellas Gene Therapies

Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Pompe Disease (Late-onset)

United States, Spain, France, Taiwan, Italy
Mario Negri Institute for Pharmacological Research

Terminated

Enzyme Replacement Therapy in Fabry Disease

Fabry Disease

Italy
Genzyme, a Sanofi Company

Completed

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Glycogen Storage Disease Type II

United States, France, Germany, Israel, Italy, Netherlands, Taiwan
Azienda Ospedaliero, Universitaria Pisana
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico; Fondazione C.N.R...

Recruiting

Comparison of Hypothermic Versus Normothermic Ex-vivo Preservation. (DCDNet)

End Stage Liver DIsease

Italy
Federico II University

Completed

Paricalcitol in Fabry Disease

Proteinuria | Fabry Disease

Italy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

FASHION Fabry Disease Hypertrophic Cardiomyopathy and Infammation (FASHION)

Fabry Disease

Italy
Iperboreal Pharma Srl

Completed

L-Carnitine in Peritoneal Dialysis

End-Stage Renal Disease

Italy
Iperboreal Pharma Srl

Completed

L-Carnitine as an Osmo-metabolic Agent for Peritoneal Dialysis

End-Stage Renal Disease

Italy
University of Turin, Italy

Unknown

Analysis of Respiratory System Mechanics and Inflammatory Response During Orthotopic Liver Transplantation (SPPLIT Trial) (SPPLIT)

End Stage Liver Diseases

Italy
Ospedale San Donato
Amicus Therapeutics; Institute of Biomedicine and Molecular Immunology - CNR

Completed

Effect of Migalastat on Cardiac Involvement in Fabry Disease (MAIORA)

Heart Diseases | Fabry Disease

Italy
Spark Therapeutics

Active, not recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Lysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
sigma-tau i.f.r. S.p.A.

Completed

A Pilot Study to Evaluate ZADAXIN's® (Thymalfasin) Ability to Enhance Immune Response to the H1N1sw Influenza Vaccine

END STAGE RENAL DISEASE

Italy
Genzyme, a Sanofi Company

Completed

Alglucosidase Alfa Pompe Safety Sub-Registry

Pompe Disease

Belgium, United States, Czechia, Germany, Italy, Taiwan
Sanofi

Recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Glycogen Storage Disease Type II

Belgium, United States, Italy, Spain, Taiwan, United Kingdom, Netherlands, France, Germany
Sanofi

Recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (Baby-COMET)

Glycogen Storage Disease Type II

Belgium, United States, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom
Genzyme, a Sanofi Company

Recruiting

Pompe Pregnancy Sub-Registry

Pompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Glycogenesis 2 Acid Maltase Deficiency

Croatia, United States, Italy, Belgium, Czechia
IRCCS San Raffaele

Recruiting

Study on the Quality of Life and Pathological State in Patients Who Underwent Radical Cystectomy

Bladder Cancer | Muscle-Invasive Bladder Carcinoma | Radical Cystectomy | Urinary Diversion | Bladder Urothelial Carcinoma | Progression, Disease | Bladder Cancer Stage II | Bladder Cancer Stage IV | Muscle Invasive Bladder Urothelial Carcinoma | Neobladder | Bladder Cancer TNM Staging | Bladder Cancer Stage... and other conditions

Italy
University of Chicago
National Cancer Institute (NCI)

Withdrawn

Irinotecan Hydrochloride With FOLFIRI and Cetuximab as First-Line Therapy in Treating Patients With RAS Wild-Type Colorectal Cancer

Stage IVA Colorectal Cancer | Stage IVB Colorectal Cancer

Italy
BioMarin Pharmaceutical

Terminated

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Late-onset Pompe Disease

United States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
IRCCS San Raffaele
Fondazione Telethon

Active, not recruiting

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant (TigetT10_MPSIH)

Mucopolysaccharidosis IH

Italy
Gruppo Italiano Studio Linfomi

Unknown

Prospective Collection of Biological Data of Prognostic Relevance in Patients With B-Cell Chronic Lymphocytic Leukemia

Chronic Lymphocytic Leukemia Stage A(0) | Chronic Lymphocytic Leukemia Stage A(I) | Chronic Lymphocytic Leukemia Stage A(II)

Italy
Emoled

Completed

EmoLED Medical Device Treatment of Second and Third Stage Pressure Ulcers (RISE_UP)

Pressure Ulcer | Pressure Ulcers Stage II | Pressure Ulcers Stage III | Pressure Sore

Italy

Clinical Trials on Cholesterol Ester Storage Disease (CESD) in Italy

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