Search clinical trials for: Lysosomal Storage Disease

Total 383193 results

BioMarin Pharmaceutical

Completed

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV A

United States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
Shire

Completed

This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.

Fabry Disease

Canada
Genzyme, a Sanofi Company

Completed

Alglucosidase Alfa Pompe Safety Sub-Registry

Pompe Disease

Belgium, United States, Czechia, Germany, Italy, Taiwan
Duke University
Genzyme, a Sanofi Company

Completed

Identification of Tongue Involvement in Late-Onset Pompe Disease

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

United States
University of Florida
BioMarin Pharmaceutical

Completed

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease (DRMF)

Pompe Disease

United States
Shire

Completed

A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

Gaucher Disease, Type 1

Israel, Paraguay, Argentina, Russian Federation, Tunisia
Shire

Completed

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Shire

Completed

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Gaucher Disease

Japan
Shire

Completed

Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Metachromatic Leukodystrophy (MLD)

Denmark
CEN Biotech
Takeda; BioClinica, Inc.

Completed

Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa) (EIROS)

MRI | Gaucher Disease, Type 1

France
Shire

Completed

Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Gaucher Disease

Israel, Romania, Serbia
Cyclo Therapeutics, Inc.

Active, not recruiting

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Niemann-Pick Disease, Type C1

United States
Shire

Completed

Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) (IDEAMLD)

Metachromatic Leukodystrophy (MLD)

Australia, Denmark, Japan, France, Germany
Shire

Terminated

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome

Netherlands, United Kingdom
Shire

Completed

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Mucopolysaccharidosis (MPS)

United Kingdom, Netherlands
Shire

Completed

Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Late Infantile Metachromatic Leukodystrophy

Denmark
Shire

Completed

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Shire
Quintiles, Inc.

Completed

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

Gaucher Disease

Japan
Shire

Completed

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome Type A

United States
Shire

Completed

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Gaucher Disease, Type 3

Egypt, India, Tunisia
Shire

Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Hunter Syndrome

Spain, United States, Canada, United Kingdom, France, Australia, Mexico
Shire

Terminated

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Sanfilippo Syndrome | Mucopolysaccharidosis (MPS)

United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
Shire

Completed

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Hunter Syndrome | Mucopolysaccharidosis II (MPS II)

United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
Shire

Completed

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1

United States, Israel, Argentina, India, Korea, Republic of, Paraguay, Poland, Russian Federation, Spain, Tunisia, United Kingdom
Shire

Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Mucopolysaccharidosis II

United States, United Kingdom, Brazil, Germany
Shire

Completed

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Gaucher Disease

United States, Israel, United Kingdom, Poland, Spain
Shire

Completed

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

Fabry Disease

United States, United Kingdom, Australia, Czechia, Finland, Paraguay, Poland, Slovenia
Shire

Completed

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

Hunter Syndrome

United States, United Kingdom
Shire

Completed

Safety Study of Replagal® Therapy in Children With Fabry Disease

Fabry Disease

United States
Shire

Terminated

Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD

Late Infantile Metachromatic Leukodystrophy

Denmark
Shire

Completed

A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

Fabry Disease

Canada
Shire

Completed

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

Gaucher Disease, Type 1

Spain, Israel, United States, India, Paraguay, United Kingdom, Russian Federation, Tunisia, Argentina
Shire

Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Sanfilippo Syndrome

United States, Spain, United Kingdom, Argentina, France, Germany, Italy, Netherlands
Shire

Completed

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Hunter Syndrome

United Kingdom, United States, Brazil
Shire

Completed

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

Fabry Disease

United States, Slovenia, Australia, Finland, Poland, Czechia, United Kingdom
Shire
Covance; PRA Health Sciences; PharmaNet

Completed

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

Brazil, Poland, Taiwan
Melbourne Health
Sanofi

Withdrawn

Complement Activation in the Lysosomal Storage Disorders (CATALYST)

Lysosomal Storage Diseases | Fabry Disease | Niemann-Pick Disease, Type C | Gaucher Disease

Australia
Shire

Completed

Sophisticated Assessment of Disease Burden in Patients With Fabry Disease (SOPHIA)

Fabry Disease

Finland, Sweden, Germany, Belgium, Czechia, Denmark
Shire
PharmaNet; Nextrials, Inc.

Withdrawn

An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Leukodystrophy, Globoid Cell
CENTOGENE GmbH Rostock

Completed

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease (LYSO-PROOF)

Lysosomal Storage Diseases | Sphingolipidoses | Gaucher Disease

Albania, Greece, India, Israel, Morocco, Pakistan, Spain
Shire

No longer available

Treatment Protocol of Replagal for Patients With Fabry Disease

Fabry Disease

United States
Shire

Approved for marketing

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1

United States
University of Florida
Amicus Therapeutics

Completed

Diet and Exercise in Pompe Disease

Lysosomal Storage Diseases | Glycogen Storage Disease Type II | Pompe Disease

United States
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Terminated

Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)

Tay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 Gangliosidoses

United States
CENTOGENE GmbH Rostock
Shire Human Genetic Therapies, Inc.

Completed

Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation (sifap2)

Fabry Disease | Cerebrovascular Accident

Austria, Croatia, France, Germany, Poland, Portugal, Georgia
CENTOGENE GmbH Rostock
Shire Human Genetic Therapies, Inc.

Completed

Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients (sifap1)

Cerebrovascular Accident

Austria, Belgium, Croatia, Finland, France, Georgia, Germany, Ireland, Italy, Malta, Poland, Portugal, Spain, United Kingdom
CENTOGENE GmbH Rostock

Withdrawn

Fabry and Cardiomyopathy (FaCard) (FaCard)

Stroke, Acute | Cerebrovascular Accident | Cerebral Stroke

Germany
CENTOGENE GmbH Rostock

Completed

Establishment of Human Cellular Disease Models for Morquio Disease (IPSMORQUIO)

Morquio Disease

Pakistan
CENTOGENE GmbH Rostock

Completed

The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease (SWITCH)

Fabry Disease | Anderson-Fabry Disease | Fabry´s Disease

Argentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
CENTOGENE GmbH Rostock

Completed

Induced Pluripotent Stem Cells for Niemann Pick Disease (IPSNPABC)

Niemann-Pick Diseases

Pakistan

Search clinical trials for: Lysosomal Storage Disease

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