Search clinical trials for: Lysosomale Störungen

Total 100014 results

Shire

Completed

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

Hunter Syndrome

United States, United Kingdom, Canada
Shire

Completed

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Syndrome

United States, Spain, Canada, United Kingdom, Mexico, Australia, France
University of Pittsburgh
National Institute of Neurological Disorders and Stroke (NINDS)

Active, not recruiting

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Krabbe Disease

United States
Sangamo Therapeutics

Active, not recruiting

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (LTFU)

Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II

United States
Vastra Gotaland Region
Karolinska University Hospital; Göteborg University; Uppsala University Hospital

Completed

Studying Lipids as Potential Biomarkers in Patients With Fabry Disease

Fabry Disease

Sweden
Denali Therapeutics Inc.

Active, not recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Mucopolysaccharidosis II

United States, Netherlands, Canada, United Kingdom
Massachusetts General Hospital

Active, not recruiting

A Natural History of Late Onset Tay-Sachs Disease

GM2 Gangliosidosis

United States
University Health Network, Toronto
Ozmosis Research Inc.

Completed

Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

Fabry Disease

Canada
Shoshana Vilk
Genzyme, a Sanofi Company

Completed

Digital Health Platform Customized for Patients With Gaucher Disease

Gaucher Disease | Patient Reported Outcome

Israel
Green Cross Corporation

Completed

A Study of GC1111 in Hunter Syndrom Patients

Hunter Syndrome

Korea, Republic of
Sangamo Therapeutics

Enrolling by invitation

Long-Term Follow-up of Subjects Who Were Treated With ST-920

Fabry Disease | Fabry Disease, Cardiac Variant

United States, Australia, United Kingdom
Genzyme, a Sanofi Company

Terminated

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Fabry Disease | Alpha Galactosidase A Deficiency

United States, Austria, United Kingdom
4D Molecular Therapeutics

Active, not recruiting

4D-310 in Adults With Fabry Disease and Cardiac Involvement

Fabry Disease

Taiwan, Australia
4D Molecular Therapeutics

Active, not recruiting

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

Fabry Disease

United States
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
Amicus Therapeutics
Engage Health Inc.

Completed

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Fabry Disease

United States
Takeda

Completed

A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease (PEDS)

Gaucher Disease

United States
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
Prevail Therapeutics
Eli Lilly and Company

Active, not recruiting

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

Gaucher Disease, Type 2

United States, United Kingdom
JCR Pharmaceuticals Co., Ltd.

Active, not recruiting

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Mucopolysaccharidosis II

Japan
Bio Sidus SA

Completed

PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion

Fabry Disease

Argentina
Caroline Michaela Kistorp
Rigshospitalet, Denmark; Amicus Therapeutics

Active, not recruiting

Real World Evidence Study of Danish Fabry Patients (RWE-FABRY)

Fabry Disease

Denmark
IntraBio Inc

Completed

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

GM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease

United States, Germany, Spain, United Kingdom
Sanofi

Completed

Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)

Fabry Disease

United Arab Emirates
Azafaros A.G.

Active, not recruiting

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease (RAINBOW)

Niemann-Pick Disease, Type C | GM2 Gangliosidosis

Brazil
Takeda

Active, not recruiting

A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Gaucher Disease

China
Sanofi

Active, not recruiting

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (ELIKIDS)

Gaucher's Disease Type III | Gaucher's Disease Type I

Canada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
Shire

Completed

Hunter Outcome Survey (HOS) (HOS)

Hunter Syndrome

United States
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
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
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

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