Search clinical trials for: Lysosomal Storage Diseases

Total 375434 results

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 Manchester
University College, London; Manchester University NHS Foundation Trust; Orchard... and other collaborators

Active, not recruiting

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis Type IIIA

United Kingdom
Shire

Completed

Hunter Outcome Survey (HOS) (HOS)

Hunter Syndrome

United States
Recordati Rare Diseases

Completed

Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) (SCOB2)

Cystinosis

Belgium, France, United Kingdom, Germany, Italy
Shire

Terminated

A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

Gaucher Disease

Canada
Takeda

Active, not recruiting

Early Detection of Fabry Disease

Fabry Disease

Germany
Genzyme, a Sanofi Company

Active, not recruiting

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 (AMETHIST)

Tay-Sachs Disease | Sandhoff Disease

United States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
Sanofi

Completed

Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ (SEED)

Gaucher's Disease

China
Takeda

Completed

A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease

Fabry Disease

China
Passage Bio, Inc.

Suspended

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease (GALax-C)

Leukodystrophy, Globoid Cell

United States, Brazil, Netherlands, United Kingdom, Canada, Israel
AVROBIO

Terminated

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Gaucher Disease

United States, Canada
Ain Shams University

Completed

Role of Vitamin E in Gaucher Disease Patients

Gaucher Disease

Egypt
Orchard Therapeutics
Ospedale San Raffaele

Active, not recruiting

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

Lysosomal Storage Diseases | Metachromatic Leukodystrophy

Italy
Takeda

Active, not recruiting

A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition

Gaucher Disease

Spain
Mandos LLC

Terminated

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Niemann-Pick Type C Disease

United States
Amicus Therapeutics

Active, not recruiting

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Fabry Disease

United States, United Kingdom
Genzyme, a Sanofi Company

Active, not recruiting

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension (LEAP)

Gaucher Disease Type 1 | Gaucher Disease Type 3

Germany, United States, Japan, United Kingdom
Takeda

Withdrawn

A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain (mECHromatic)

Metachromatic Leukodystrophy (MLD)

Spain
Idorsia Pharmaceuticals Ltd.

Active, not recruiting

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

Fabry Disease

United States, United Kingdom, Norway, Germany, Australia, Canada, Spain, Switzerland, Austria, Belgium, France, Netherlands, Poland
Sanofi

Completed

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

Gaucher's Disease

United States
Sanofi

Completed

A First-in-human Single and Repeated Dose Escalation Study of SAR442501 in Healthy Adults Subjects

Osteochondrodysplasia

United States
AVROBIO

Terminated

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Fabry Disease

United States, Australia, Brazil
Sanofi

Completed

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth (Acronym)

Gaucher's Disease

United States
Mandos LLC

Terminated

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Niemann-Pick Disease, Type C

Costa Rica
Takeda

Completed

A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy (SWITCH)

Gaucher Disease

United States
JCR Pharmaceuticals Co., Ltd.

Enrolling by invitation

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

Mucopolysaccharidosis II

United States
Terence Flotte
Massachusetts General Hospital; University of Massachusetts, Worcester

Active, not recruiting

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Tay-Sachs Disease | Sandhoff Disease

United States
Freeline Therapeutics

Completed

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Lysosomal Storage Diseases | Fabry Disease

Germany, United Kingdom
REGENXBIO Inc.

Active, not recruiting

RGX-111 Gene Therapy in Patients With MPS I

Hurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)

United States, Brazil, Israel
University Children's Hospital, Zurich
Sanofi

Completed

Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease (Pompe_HrQol)

Pompe Disease

Switzerland
Genzyme, a Sanofi Company

Completed

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (ASCEND)

Sphingomyelin Lipidosis

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom
Chiesi Italia

Completed

Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease (GROUND)

Fabry Disease

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
IntraBio Inc

Completed

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Niemann-Pick Disease, Type C

United States, United Kingdom, Germany, Slovakia, Spain
Sanofi

Completed

A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants

Gaucher's Disease Type I
REGENXBIO Inc.

Active, not recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Brazil
REGENXBIO Inc.

Active, not recruiting

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Canada
Resverlogix Corp

Withdrawn

Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease

Fabry Disease

Canada
ZevraDenmark

Available

Early Access Program With Arimoclomol in US Patients With NPC

Niemann-Pick Disease, Type C

United States
National and Kapodistrian University of Athens

Completed

Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients

Fabry Disease

Greece
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
agnes chen
University of California, Los Angeles; National Institute of Neurological Disorders... and other collaborators

Terminated

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Mucopolysaccharidosis I | Cognitive Decline

United States
Duke University
Genzyme, a Sanofi Company

Active, not recruiting

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

Pompe Disease (Late-onset) | Pompe Disease | GAA Deficiency

United States
REGENXBIO Inc.

Enrolling by invitation

Long Term Follow-Up for RGX-111

Mucopolysaccharidosis I

United States, Brazil
Azafaros A.G.

Active, not recruiting

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) (PRONTO)

GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease

United States, Brazil, France, Germany, Italy, United Kingdom
Genzyme, a Sanofi Company

Completed

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

Mucopolysaccharidosis I

China
Genzyme, a Sanofi Company

Completed

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Fabry Disease

China
Genzyme, a Sanofi Company

Active, not recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Glycogen Storage Disease Type II

France
Genzyme, a Sanofi Company

Completed

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Sphingomyelin Lipidosis

United States, Belgium, Brazil, France, Germany, Italy, United Kingdom
Chiesi Farmaceutici S.p.A.

Available

Expanded Access to Velmanase Alfa

Alpha-Mannosidosis

Search clinical trials for: Lysosomal Storage Diseases

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