Search clinical trials for: lysosomal storage diseases

Total 374566 results

GeneCradle Inc

Recruiting

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Pompe Disease (Late-onset)

China
Lundquist Institute for Biomedical Innovation at...

Not yet recruiting

Study of Cannabidiol in Sanfilippo Syndrome

Mucopolysaccharidosis III | Sanfilippo Syndrome

United States
Chiesi Farmaceutici S.p.A.
ICON plc

Not yet recruiting

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease (FLY)

Fabry Disease
Caroline Michaela Kistorp
Sanofi

Not yet recruiting

Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease (FORTIFY)

Chronic Kidney Diseases | Fabry Disease

Denmark
National Taiwan University Hospital

Recruiting

The Natural History of Sialidosis Type I

Observational Study

Taiwan
Forge Biologics, Inc

Not yet recruiting

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Krabbe Disease

United States
Ondokuz Mayıs University

Recruiting

The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence

Quality of Life | Urinary Incontinence | Menopause | Wolman Disease | Psychological Well-Being

Turkey
Xinhua Hospital, Shanghai Jiao Tong University...
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Type II Gaucher Disease

China
UniQure Biopharma B.V.

Recruiting

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Fabry Disease

United States
Brunel University
Royal Free Hospital NHS Foundation Trust

Recruiting

A Co-designed Physical Activity Intervention in Fabry Disease

Fabry Disease

United Kingdom
Chung-Hsing Wang
Sanofi

Not yet recruiting

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Gaucher Disease

Taiwan
University of Cambridge
Sanofi

Recruiting

Molecular Imaging in Fabry Disease of the Heart

Fabry Disease, Cardiac Variant

United Kingdom
Children's Hospital of Fudan University

Not yet recruiting

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Fabry Disease

China
Sanofi
Pulse Infoframe Ltd.

Recruiting

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

Niemann-Pick Diseases | Acid Sphingomyelinase Deficiency

United States
Chiesi Farmaceutici S.p.A.

Not yet recruiting

Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3 (LAMPO(0-3))

Alpha-Mannosidosis
Denali Therapeutics Inc.

Recruiting

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Mucopolysaccharidosis Type IIIA

United States
Huashan Hospital

Not yet recruiting

Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)

Pompe Disease (Late-onset)

China
Qilu Hospital of Shandong University

Recruiting

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy (SEARCH)

Fabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular Hypertrophy

China
He Huang
Lingyi Biotech Co., Ltd.

Recruiting

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I (LY-M001)

Gaucher Disease Type I

China
Astellas Gene Therapies

Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Pompe Disease (Late-onset)

United States, Spain, France, Taiwan, Italy
Orchard Therapeutics

Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT (HURCULES)

MPS-IH (Hurler Syndrome)

Netherlands, United States, Italy, United Kingdom
McMaster University

Not yet recruiting

Nutritional Therapy in Late-onset Pompe Disease (PDT-MIS)

Obesity | Lysosomal Storage Diseases | Muscle Loss | Glycogen Storage Disease Type II | Pompe Disease | Nutrition Poor | Glycogen Storage Disease Type II, Adult | Glycogen Storage Disease Type II Late Onset
Amicus Therapeutics

Recruiting

A Global Prospective Observational Registry of Patients With Pompe Disease

Pompe Disease

United States
Lysosomal and Rare Disorders Research and Treatment...

Recruiting

Biomarkers Related to Bone in Pediatric Gaucher Disease

Bone Diseases | Gaucher Disease

United States
AceLink Therapeutics, Inc.
Tigermed Consulting Co., Ltd

Recruiting

Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease

Fabry Disease

China
Rocket Pharmaceuticals Inc.

Recruiting

A Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Danon Disease

United States
Westfälische Wilhelms-Universität Münster
Chiesi GmbH

Recruiting

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa (GoPEG)

Fabry Disease

Germany
JCR Pharmaceuticals Co., Ltd.

Recruiting

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis III-A

Germany
ISU Abxis Co., Ltd.

Recruiting

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

Fabry Disease

Korea, Republic of
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Sanofi

Recruiting

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia (upreACH-2)

Osteochondrodysplasia

Australia, Spain, Korea, Republic of, China, Italy
Lysosomal and Rare Disorders Research and Treatment...

Recruiting

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Fabry Disease

United States
Sanofi

Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

Fabry Disease

Taiwan
Institut National de la Santé Et de la Recherche...

Recruiting

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multiple Sulfatase Deficiency Disease | Mucopolysaccharidosis IX

France
Takeda

Recruiting

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Hunter Syndrome

Canada, France
Sanofi

Recruiting

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (SHORTEN)

Fabry's Disease

United States
Fundacion para la Investigacion Biomedica del Hospital...
Fundación Mutua Madrileña

Recruiting

Autoimmune and Inflammatory Response Biomarkers in Fabry Disease (Bio-FAIR)

Fabry Disease

Spain
Nacuity Pharmaceuticals, Inc.

Recruiting

PK and PD Study of NPI-001 and Cysteamine Bitartrate (INCA)

Cystinosis

Australia
Fundación Española de Hematología y Hemoterapía

Recruiting

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

Gaucher Disease | Acid SphingoMyelinase Deficiency

Spain
Cystinose Stiftung
Cystinosis Research Network; Leben eben! Cystinose-Selbsthilfe e.V.

Recruiting

Development of Health-related Quality of Life Instrument for Patients With Cystinosis (QUALIFY)

Cystinosis

Germany
Fondazione Don Carlo Gnocchi Onlus
Associazione Italiana Glicogenosi (AIG); Associazione Riabilitatori Insufficienza... and other collaborators

Recruiting

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease (LOPD01)

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

Italy
Medical University of Vienna

Recruiting

Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study. (POMPE)

Pompe Disease | McArdle Disease

Austria
Hospices Civils de Lyon

Recruiting

T1 Mapping in Fabry Disease (MAP-FAB)

Fabry Disease

France
Eline C. B. Eskes

Recruiting

Biomarkers Of Lung InVolvement In ASMD (BOLIVIA)

Asmd, Visceral Type

Netherlands
Sanofi

Recruiting

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

Gaucher Disease

United States
Eline C. B. Eskes

Recruiting

Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency (MONACO)

Asmd, Visceral Type

Netherlands
Institut National de la Santé Et de la Recherche...

Recruiting

European Cystinosis Cohort (RaDiCo-ECYSCO)

Cystinosis

France
University of California, San Francisco

Recruiting

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

MPS IVA | MPS VI

United States
Bio Sidus SA

Recruiting

Switch Over Study of Biosimilar AGA for Fabry Disease (SMILE)

Fabry Disease

Argentina
University of Minnesota

Recruiting

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

Gaucher Disease

United States

Search clinical trials for: lysosomal storage diseases

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