Clinical Trials for Gaucher Disease

Genetic Studies of Lysosomal Storage Disorders

NCT ID: NCT00001215

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT ID: NCT05487599

The PROCEED trial is studying a new treatment called PR001 (also known as LY3884961) for adults with Gaucher Disease, specifically those experiencing peripheral symptoms. This study aims to find ou...

The GBA Multimodal Study in Parkinson's Disease

NCT ID: NCT04101968

The GBA Multimodal Study in Parkinson's Disease is a clinical trial that aims to understand how mutations in the GBA gene are connected to Parkinson's disease. Researchers will use advanced imaging...

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

NCT ID: NCT05669729

This clinical trial is designed to understand how well patients with Gaucher disease, their caregivers, and nurses understand and use educational materials related to a treatment called VPRIV, whic...

Longitudinal Study of Neurodegenerative Disorders

NCT ID: NCT03333200

This clinical trial is focused on understanding the progression of various rare genetic disorders that affect the brain, such as Krabbe Disease and Tay-Sachs Disease, among others. Researchers are ...

Drug Discovery for Parkinson's With Mutations in the GBA Gene

NCT ID: NCT05536388

The clinical trial titled "Drug Discovery for Parkinson's With Mutations in the GBA Gene" is being conducted by the New York Stem Cell Foundation (NYSCF) Research Institute. The main goal of this r...

Gaucher Disease Outcome Survey (GOS)

NCT ID: NCT03291223

The Gaucher Outcomes Survey (GOS) is a research study focused on understanding Gaucher Disease, a genetic condition that affects how the body processes certain fats. This study does not involve any...

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

NCT ID: NCT05843552

This clinical trial is studying Gaucher Disease, a condition caused by problems in lysosomes, which are tiny structures in our cells responsible for breaking down waste. The researchers want to und...

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

NCT ID: NCT00358943

The ICGG Gaucher Disease Registry is a global program designed to learn more about Gaucher disease, a genetic disorder that affects how the body processes certain fats. This registry collects infor...

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT ID: NCT06539169

The FLOWER trial is a nationwide study that aims to gather important information about rare diseases, such as Alpha-Thalassemia, Beta-Thalassemia, and many others. This study is completely virtual,...

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT ID: NCT05368038

A Long-term Follow-up Study of Gaucher Disease

NCT ID: NCT03190837

This clinical trial is focused on understanding Gaucher disease, a genetic condition that affects how your body processes a type of fat. The study aims to learn about the long-term effects of the d...

Institutional Registry of Rare Diseases

NCT ID: NCT06573723

This clinical trial, called the Institutional Registry of Rare Diseases, aims to gather information about various rare diseases, including conditions like amyloidosis, sarcoidosis, and Wilson disea...

Clinical Study of LY-M001 Injection in the Treatment of Adolescents With Type I Gaucher Disease

NCT ID: NCT06528080

This clinical trial is studying a new treatment called LY-M001 injection for adolescents aged 12 to 18 with Type I Gaucher Disease, a genetic disorder that affects how the body breaks down certain ...

Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3

NCT ID: NCT06523517

This clinical trial is focused on studying the effectiveness and safety of a medication called eliglustat in Chinese children and teenagers aged 12 to 17 who have Gaucher disease type 1 or type 3. ...

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

NCT ID: NCT05992532

The GammaGA trial is studying two rare genetic conditions called Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in patients who have certain blood disorders known as monoclonal...

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

NCT ID: NCT06258577

This clinical trial is looking into screening for two rare genetic conditions: Gaucher Disease and Acid Sphingomyelinase Deficiency. It will focus on patients in Taiwan who have specific health iss...

Biomarkers Related to Bone in Pediatric Gaucher Disease

NCT ID: NCT06116071

This clinical trial is studying how certain blood markers can help doctors understand bone issues in children and young adults with Gaucher disease, which is a genetic disorder affecting how the bo...