Gaucher Disease Clinical Trials

A listing of Gaucher Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Filter By
Clear all Advanced
I am/have/had
added new label for I am/have/had
I am looking for
added new label for I am looking for
Advanced Filters
Found 24 clinical trials
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

PRV-GD2-101 is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose PR001 in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the

type 1 gaucher disease
  • 19 Jul, 2021
  • 5 locations
Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3

adult Gaucher disease Type 1 (GD1) patients. Screen adult and pediatric GD3 patients who qualify for treatment with venglustat in Parts 2 and 3. Parts 2 and 3: Cohort 2

  • 13 Jun, 2021
  • 7 locations
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Primary Objective: Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (2 to <18 years old). Secondary Objective: Evaluate the efficacy of eliglustat and quality of life in pediatric patients (2 to <18 years old).

platelet count
  • 27 Jan, 2021
  • 37 locations
Phase 1/2a Clinical Trial of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

patients have mutations in at least one chromosomal copy of GBA1, while Gaucher disease patients have mutations in both chromosomal copies. Without enough GCase, glycolipids accumulate

  • 18 Sep, 2020
  • 4 locations
A Multicenter Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are …

  • 27 Mar, 2021
  • 3 locations
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means

type 1 gaucher disease
replacement therapy
velaglucerase alfa
substrate reduction therapy
  • 22 May, 2021
  • 1 location
Phase 1/2 Lentiviral Vector Gene Therapy - The GuardOne Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

postpubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in plasma and PBLs).

type 1 gaucher disease
platelet count
a hemoglobin
  • 24 Jan, 2021
  • 2 locations
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type

Primary Objective To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type To

  • 21 Mar, 2021
  • 1 location
Getting Global Rare Disease Insights Through Technology Study

metabolic disorders such as Fabry disease, Pompe disease, and Gaucher disease, will provide access to Zamplo to patients free-of-charge to evaluate its utility in managing the symptoms of their disease

  • 19 Apr, 2021
  • 1 location
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

  • 18 Apr, 2021
  • 1 location