Search clinical trials for: lysosomal storage diseases

Total 377411 results

Pfizer

Completed

A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Gaucher Disease
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
Sanofi

Withdrawn

To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease (BCLEAR1)

Fabry Disease
Chiesi Farmaceutici S.p.A.
Chiesi USA, Inc.

Available

Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

Fabry Disease

United States
Massachusetts General Hospital

Completed

Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

Fabry's Disease

United States
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
Genzyme, a Sanofi Company

Completed

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

Pompe Disease | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
Centre Hospitalier Universitaire de Nice

Completed

Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness (POPS3)

Pompe Disease

France
Chiesi Farmaceutici S.p.A.
Iqvia Pty Ltd; Protalix

Completed

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion (PEOPLE)

Fabry Disease

United States, Belgium, Denmark, Italy, United Kingdom
Lundquist Institute for Biomedical Innovation at...

Active, not recruiting

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI

United States
ArmaGen, Inc

Completed

Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I

Mucopolysaccharidosis I

Brazil
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I

Mucopolysaccharidosis I

Brazil
HaEmek Medical Center, Israel

Terminated

Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

Liver Fibrosis | Gaucher Disease | Spleen; Fibrosis

Israel
Mandos LLC

Completed

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Niemann-Pick Disease, Type C

United States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

Mucopolysaccharidosis I

United States
Shaare Zedek Medical Center

Completed

Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

Gaucher Disease, Type 1

Israel
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Mucolipidosis Disorder Type I, II, III, IV (BioML) (BioML)

Skeletal Abnormalities | Psychomotor Retardation

Germany, India, Sri Lanka, Egypt
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Niemann Pick Type C Disease (BioNPC) (BioNPC)

Niemann-Pick Disease, Type C | Niemann-Pick Disease

Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2) (BioGM1/BioGM2)

Seizures | Mental Retardation | Hepato-splenomegaly | Dysostosis Multiplex

Egypt, Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Gaucher Disease: BioGaucher (BioGaucher) (BioGaucher)

Splenomegaly | Hepatomegaly

Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Patients With Fabry Disease (BioFabry) (BioFabry)

Chronic Kidney Disease | Hearing Loss | Angiokeratomas | Ocular Abnormalities

Sri Lanka, India, Germany
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Glycogen Storage Diseases (BioGlycogen) (BioGlycogen)

Fructose Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease | Glycogen Storage Disease Type V | Glycogen Storage Disease Type I | Glycogen Storage Disease Type III | Glycogen Storage Disease Type VII | Glycogen Storage Disease Type IV | Glycogen Storage Disease Type... and other conditions

Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Hurler Disease (BioHurler) (BioHurler)

Metabolism, Inborn Errors | Mucopolysaccharidosis Type I | Gargoylism

Germany, India, Sri Lanka, Egypt
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Farber Disease (BioFarber) (BioFarber)

Splenomegaly | Hepatomegaly | Ceramidase Deficiency | Farber's Lipogranulomatosis

Germany, India, Egypt
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Sly Disease (MPS VII) (BioSly) (BioSly)

Developmental Delay | Splenomegaly | Hepatomegaly | Skeletal Abnormalities

Germany, India, Sri Lanka, Egypt
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Krabbe Disease (BioKrabbe) (BIOKRABBE)

Krabbe Disease

Egypt, Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Metachromatic Leukodystrophy (BioMeta) Disease (BioMeta)

Muscle Weakness | Peripheral Neuropathy

Egypt, Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Wolman Disease (BioWolman) (BioWolman)

Cholesterol Ester Storage Disease | Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type

Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Mannosidosis Disease (BioMannosidosis) (BioMannosido)

Alpha-Mannosidase B Deficiency | Lysosomal Alpha B Mannosidosis | Alpha-Mannosidase Deficiency

India, Sri Lanka, Germany
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Maroteaux-Lamy Disease (BioMaroteaux) (BioMaroteaux)

Lung Diseases | Eye Abnormalities | Lysosomal Storage Disease | Macroglossia | Obstructive Sleep Apnoea

Germany, India, Sri Lanka, Egypt
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis) (BioCystinosis)

Hypophosphatemia | Photophobia | Renal Fanconi Syndrome

Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Morquio Disease (BioMorquio) (BioMorquio)

Morquio Syndrome A | Morquio Syndrome | Morquio B Disease | Accumulation of Mucopolysaccharides

Germany, India, Sri Lanka, Egypt
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Pompe Disease (BioPompe) (BioPompe)

Lung Disease | Cerebral Aneurysm | Macroglossia | Cardiac Diseases | Obstructive Sleep Apnoea | Muscular Weakness | Hepato-splenomegaly

Germany, India, Sri Lanka
CENTOGENE GmbH Rostock

Terminated

Biomarkers for Hunter Syndrome (BioHunter)

Mucopolysaccharidosis II | Hunter Syndrome | Hunter's Syndrome, Mild Form | Hunter's Canal Syndrome

Mexico
Sangamo Therapeutics

Terminated

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

MPS I

United States
Allievex Corporation

Active, not recruiting

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis Type IIIB | MPS III B

Germany, United States, Turkey, United Kingdom, Colombia, Spain, Taiwan
KemPharm Denmark A/S

Terminated

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Gaucher Disease, Type 1 | Gaucher Disease, Type 3

India
BioMarin Pharmaceutical

Terminated

A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

Late-onset Pompe Disease

United States, United Kingdom
Hospital de Clinicas de Porto Alegre
The Isaac Foundation

Active, not recruiting

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Mucopolysaccharidoses | Mucopolysaccharidosis VI | Morquio A Syndrome | Mucopolysaccharidosis IV A | MPS IV A | MPS VI | MPS - Mucopolysaccharidosis | Morquio Syndrome A | Morquio Syndrome

Brazil
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-171 in Patients With Mucopolysaccharidosis I

Mucopolysaccharidosis I

United States, Brazil, Japan
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Mucopolysaccharidosis II

Japan
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Brazil
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Japan
Orchard Therapeutics
Ospedale San Raffaele

Active, not recruiting

Gene Therapy for Metachromatic Leukodystrophy (MLD)

Lysosomal Storage Disease | Metachromatic Leukodystrophy

Italy
Shaare Zedek Medical Center
Shire

Completed

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Gaucher Disease, Type 1
Shaare Zedek Medical Center
Pfizer

Completed

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

Gaucher Disease, Type 1
Allievex Corporation

Active, not recruiting

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB

United States, Germany, Spain, Argentina, Australia, Brazil, Colombia, Taiwan, Turkey
Sangamo Therapeutics

Terminated

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Mucopolysaccharidosis II | MPS II

United States
New York Medical College

Completed

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Mucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditions

United States
Turkish Society of Pediatric Gastroenterology,...

Completed

Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease (VICTORIA)

Duchenne Muscular Dystrophy | Pompe Disease (Late-onset)

Turkey

Search clinical trials for: lysosomal storage diseases

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