Search clinical trials for: Lysosomale Speicherstörungen

Total 923 results

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
Genzyme, a Sanofi Company

Active, not recruiting

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

Pompe's Disease

China
Sanofi

Completed

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

Fabry Disease

United Arab Emirates
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
Genzyme, a Sanofi Company

Completed

Avalglucosidase Alfa Extension Study (NEO-EXT)

Glycogen Storage Disease Type II Pompe Disease

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
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
Lundquist Institute for Biomedical Innovation at...
Swedish Orphan Biovitrum; Cure Sanfilippo Foundation

Completed

Open-label Study of Anakinra in MPS III

Mucopolysaccharidosis III

United States
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
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Terminated

A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

Mucopolysaccharidosis I | Hurler's Syndrome | Hurler-Scheie Syndrome | Scheie

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 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
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

Search clinical trials for: Lysosomale Speicherstörungen

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